0001628280-24-007205 | 10-K | XBRL Viewer | Biocryst Pharmaceuticals INC

Stock-Based Compensation - Option Activity (Details) - USD ($) $ / shares in Units, shares in Thousands, $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Outstanding [Roll Forward]
Options exercised (in shares)	(1,276)
Incentive Plan
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Additional Disclosures [Abstract]
Options, exercises in period, intrinsic value		$ 21,150	$ 25,484
Inducement Plan
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Additional Disclosures [Abstract]
Options, exercises in period, intrinsic value		$ 3,710	$ 6,700
Share-based Payment Arrangement, Option \| Incentive Plan
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Outstanding [Roll Forward]
Options outstanding, beginning balance (in shares)	31,179
Options, grants in period, gross (in shares)	6,625
Options exercised (in shares)	(935)
Options cancelled or forfeited (in shares)	(1,364)
Options outstanding, ending balance (in shares)	35,505	31,179
Options exercisable (in shares)	21,008
Number of stock option awards vested and expected to vest (in shares)	33,557
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Outstanding, Weighted Average Exercise Price [Abstract]
Weighted average exercise price, beginning balance (in dollars per share)	$ 8.56
Weighted average exercise price, stock option awards granted (in dollars per share)	6.67
Weighted average exercise price, stock option awards exercised (in dollars per share)	5.21
Weighted average exercise price, stock option awards cancelled (in dollars per share)	10.00
Weighted average exercise price, ending balance (in dollars per share)	8.24	$ 8.56
Weighted average exercise price, stock option awards, exercisable (in dollars per share)	7.87
Weighted average exercise price of stock option awards vested and expected to vest (in dollars per share)	$ 8.19
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Additional Disclosures [Abstract]
Weighted average remaining contractual life	6 years 10 months 6 days
Options, exercises in period, intrinsic value	$ 3,601
Options outstanding, aggregate intrinsic value	$ 12,280
Options, exercisable, weighted average remaining contractual term (year)	5 years 5 months 8 days
Stock option awards vested and expected to vest, weighted average remaining contractual life (year)	6 years 9 months 7 days
Options, aggregate intrinsic value	$ 12,280
Options, exercisable, aggregate intrinsic value	$ 12,280
Share-based Payment Arrangement, Option \| Inducement Plan
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Outstanding [Roll Forward]
Options outstanding, beginning balance (in shares)	5,341
Options, grants in period, gross (in shares)	1,196
Options exercised (in shares)	(344)
Options cancelled or forfeited (in shares)	(666)
Options outstanding, ending balance (in shares)	5,527	5,341
Options exercisable (in shares)	2,448
Number of stock option awards vested and expected to vest (in shares)	5,122
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Outstanding, Weighted Average Exercise Price [Abstract]
Weighted average exercise price, beginning balance (in dollars per share)	$ 8.80
Weighted average exercise price, stock option awards granted (in dollars per share)	8.15
Weighted average exercise price, stock option awards exercised (in dollars per share)	3.70
Weighted average exercise price, stock option awards cancelled (in dollars per share)	10.47
Weighted average exercise price, ending balance (in dollars per share)	8.77	$ 8.80
Weighted average exercise price, stock option awards, exercisable (in dollars per share)	7.17
Weighted average exercise price of stock option awards vested and expected to vest (in dollars per share)	$ 8.68
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Additional Disclosures [Abstract]
Weighted average remaining contractual life	7 years 9 months 3 days
Options, exercises in period, intrinsic value	$ 1,803
Options outstanding, aggregate intrinsic value	$ 4,463
Options, exercisable, weighted average remaining contractual term (year)	6 years 10 months 20 days
Stock option awards vested and expected to vest, weighted average remaining contractual life (year)	7 years 8 months 19 days
Options, aggregate intrinsic value	$ 3,434
Options, exercisable, aggregate intrinsic value	$ 4,291

Inventory - Summary of Inventories (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Inventory Disclosure [Abstract]
Raw materials	$ 6,449	$ 8,906
Work-in-process	17,591	14,990
Finished goods	6,242	4,814
Total inventory	30,282	28,710
Reserves	(1,599)	(1,177)
Total inventory, net	$ 28,683	$ 27,533

Investments

12 Months Ended

Dec. 31, 2023

Investments, All Other Investments [Abstract]

Investments

Fair value is an exit price, representing the amount that would be received from the sale of an asset or paid to transfer a liability in an orderly transaction between market participants. Fair value is determined based on observable and unobservable inputs. Observable inputs reflect readily obtainable data from independent sources, while unobservable inputs reflect certain market assumptions. As a basis for considering such assumptions, U.S. GAAP establishes a three-tier value hierarchy, which prioritizes the inputs used to develop the assumptions and for measuring fair value as follows: (Level 1) observable inputs such as quoted prices in active markets for identical assets; (Level 2) inputs other than the quoted prices in active markets that are observable either directly or indirectly; and (Level 3) unobservable inputs for which there is little or no market data, which requires the Company to develop its own assumptions. This hierarchy requires the Company to use observable market data, when available, and to minimize the use of unobservable inputs when determining fair value.

The Company’s financial instruments that are measured at fair value on a recurring basis consist of fixed income investments. These valuations are based on observable direct and indirect inputs, primarily quoted prices of similar, but not identical, instruments in active markets or quoted prices for identical or similar instruments in markets that are not active. These fair values are obtained from independent pricing services.

Assets measured at fair value on a recurring basis were as follows (in thousands):


	December 31, 2023
	Quoted Price in Active Markets (Level 1)		Significant Other Observable Inputs (Level 2)		Significant Unobservable Inputs (Level 3)		Total
Assets:
Obligations of U.S. Government and its agencies	$	—	$	277,358	$	—	$	277,358
Certificates of deposit	—		986		—		986
Total assets	$	—	$	278,344	$	—	$	278,344


	December 31, 2022
	Quoted Price in Active Markets (Level 1)		Significant Other Observable Inputs (Level 2)		Significant Unobservable Inputs (Level 3)		Total
Assets:
Obligations of U.S. Government and its agencies	$	—	$	129,371	$	—	$	129,371
Corporate debt securities	—		6,092		—		6,092
Certificates of deposit	—		2,157		—		2,157
Total assets	$	—	$	137,620	$	—	$	137,620

There were no changes in valuation techniques during the years ended December 31, 2023 and 2022. There were no liabilities measured at fair value on a recurring basis as of December 31, 2023 and 2022.

As of December 31, 2023, the Company had 11 securities with a total estimated fair market value of $18,513 in an unrealized loss position. The Company believes the individual unrealized losses represent temporary declines primarily resulting from interest rate changes. The Company does not have intent to sell these investments, and it is more likely than not that the investments will be held until recovery of their amortized cost basis. As such, no allowance was recognized.

The following tables summarize the fair value of the Company’s investments by type (in thousands):


	December 31, 2023
	Amortized Cost		Accrued Interest		Gross Unrealized Gains		Gross Unrealized Losses		Estimated Fair Value
Obligations of U.S. Government and its agencies	$	277,151	$	121	$	150	$	(64)	$	277,358
Certificates of deposit	980		14		—		(8)		986
Total investments	$	278,131	$	135	$	150	$	(72)	$	278,344


	December 31, 2022
	Amortized Cost		Accrued Interest		Gross Unrealized Gains		Gross Unrealized Losses		Estimated Fair Value
Obligations of U.S. Government and its agencies	$	129,940	$	427	$	—	$	(996)	$	129,371
Corporate debt securities	6,093		37		—		(38)		6,092
Certificates of deposit	2,163		23		—		(29)		2,157
Total investments	$	138,196	$	487	$	—	$	(1,063)	$	137,620

The following table summarizes the scheduled maturity for the Company’s investments at December 31, 2023 and 2022 (in thousands):


	December 31,
	2023		2022
Maturing in one year or less	$	278,344	$	119,543
Maturing after one year through two years	—		18,077
Total investments	$	278,344	$	137,620

Investments- Fair Value of the Company's Investments by Type (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Marketable Securities [Line Items]
Amortized Cost	$ 278,131	$ 138,196
Accrued Interest	135	487
Gross Unrealized Gains	150	0
Gross Unrealized Losses	(72)	(1,063)
Estimated Fair Value	$ 278,344	$ 137,620
Debt Securities, Available-for-Sale, Accrued Interest, after Allowance for Credit Loss, Statement of Financial Position [Extensible Enumeration]	Investments, Long-term investments	Investments, Long-term investments
Obligations of U.S. Government and its agencies
Marketable Securities [Line Items]
Amortized Cost	$ 277,151	$ 129,940
Accrued Interest	121	427
Gross Unrealized Gains	150	0
Gross Unrealized Losses	(64)	(996)
Estimated Fair Value	277,358	129,371
Certificates of deposit
Marketable Securities [Line Items]
Amortized Cost	980	2,163
Accrued Interest	14	23
Gross Unrealized Gains	0	0
Gross Unrealized Losses	(8)	(29)
Estimated Fair Value	$ 986	2,157
Corporate debt securities
Marketable Securities [Line Items]
Amortized Cost		6,093
Accrued Interest		37
Gross Unrealized Gains		0
Gross Unrealized Losses		(38)
Estimated Fair Value		$ 6,092

Trade Receivables Textual (Details) - USD ($)	Dec. 31, 2023	Dec. 31, 2022
Trade Accounts Receivable
Accounts, Notes, Loans and Financing Receivable [Line Items]
Accounts receivable, after allowance for credit loss, total	$ 288,000	$ 288,000
ORLADEYO
Accounts, Notes, Loans and Financing Receivable [Line Items]
Allowance		0
ORLADEYO \| Trade Accounts Receivable
Accounts, Notes, Loans and Financing Receivable [Line Items]
Accounts receivable, after allowance for credit loss, total	54,149,000	41,508,000
RAPIVAB
Accounts, Notes, Loans and Financing Receivable [Line Items]
Allowance	0	0
RAPIVAB \| Trade Accounts Receivable
Accounts, Notes, Loans and Financing Receivable [Line Items]
Accounts receivable, after allowance for credit loss, total	$ 505,000	$ 823,000

Workforce Reduction (Details) - USD ($)	3 Months Ended	12 Months Ended
Workforce Reduction (Details) - USD ($)	Mar. 31, 2024	Dec. 31, 2023
Employee Severance
Restructuring Cost and Reserve [Line Items]
Restructuring costs		$ 3,380,000
Restructuring reserve		0
Employee Severance \| Research and Developement
Restructuring Cost and Reserve [Line Items]
Restructuring costs		3,026,000
Employee Severance \| Selling, general and administrative
Restructuring Cost and Reserve [Line Items]
Restructuring costs		$ 354,000
One-time Termination Benefits \| Subsequent Event
Restructuring Cost and Reserve [Line Items]
Restructuring costs	$ 1,264,000
One-time Termination Benefits \| Research and Developement \| Subsequent Event
Restructuring Cost and Reserve [Line Items]
Restructuring costs	1,201,000
One-time Termination Benefits \| Selling, general and administrative \| Subsequent Event
Restructuring Cost and Reserve [Line Items]
Restructuring costs	$ 63,000

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References + Details

Stock-Based Compensation - Share-Based Compensation (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Total stock-based compensation expense	$ 55,615	$ 44,701	$ 34,640
Incentive Plan
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Total stock-based compensation expense	44,581	36,716	27,062
Inducement Plan
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Total stock-based compensation expense	9,958	6,550	6,055
ESPP
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Total stock-based compensation expense	$ 1,076	$ 1,435	$ 1,523

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References

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+ Details

Workforce Reduction	12 Months Ended
Workforce Reduction	Dec. 31, 2023
Restructuring and Related Activities [Abstract]
Workforce Reduction	Workforce Reduction In January 2024, the Company announced a reduction of workforce. The majority of the impacted employees had a termination date in January 2024, with certain employees exiting later in 2024. The Company notified the impacted employees in January 2024. The Company incurred costs related to employee severance, benefits, and related costs which were accounted for as ongoing terminations benefits under ASC Topic 712, Nonretirement Postemployment Benefits. As of December 31, 2023, it was considered probable that payment would be owed and the amount of payment was considered to be reasonably estimable, which resulted in the recognition of $3,380 of costs related to the workforce reduction during the year ended December 31, 2023, of which $3,026 has been recognized in research and development expenses and $354 has been recognized in selling, general and administrative expenses in the Consolidated Statement of Comprehensive Loss. As of December 31, 2023, none of the costs were paid and are included within accrued expenses on the Consolidated Balance Sheet. These costs are expected to be disbursed during the period of January 1, 2024 through December 31, 2024. In addition, the employees impacted by the workforce reduction received an amount equal to the bonus amount the employee would have received through continued employment with the Company, which was considered a one-time termination benefit pursuant to ASC Topic 420, Exit or Disposal Costs. As a result, $1,264 will be recognized during the three months ended March 31, 2024, the period in which the communication occurred, of which $1,201 will be recognized in research and development expenses, and $63 will be recognized in selling, general and administrative expenses in the Consolidated Statement of Comprehensive Loss. This cost is expected to be disbursed during the period of January 1, 2024 through March 31, 2024. The Company does not expect to incur any additional significant costs related to this workforce reduction.

Lease Obligations - Aggregate Lease Expense Under Operating Leases (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Leases [Abstract]
Operating lease expense	$ 2,018	$ 1,578	$ 1,660
Amortization of right-of-use assets	1,212	816	113
Interest on lease liabilities	201	138	22
Total finance lease expense	$ 1,413	$ 954	$ 135

Stock-Based Compensation Textual (Details) $ / shares in Units, $ in Thousands	1 Months Ended		12 Months Ended
	Jan. 31, 2022 shares	Dec. 31, 2014 shares	Dec. 31, 2023 USD ($) plan $ / shares shares	Dec. 31, 2022 USD ($) $ / shares shares	Dec. 31, 2021 USD ($) $ / shares shares
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Number of plans \| plan			3
Total stock-based compensation expense			$ 55,615	$ 44,701	$ 34,640
Exercise of stock options, net (in shares) \| shares			1,276,000
Employee stock purchase plan sales, net (in shares) \| shares			338,000	260,000	321,000
Share-based Payment Arrangement, Option \| Non-employee Directors
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Award vesting period (year)			1 year
Incentive Plan
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Total stock-based compensation expense			$ 44,581	$ 36,716	$ 27,062
Options, grants in period, weighted average grant date fair value (in dollars per share) \| $ / shares			$ 4.75	$ 7.57	$ 7.93
Total unrecognized compensation cost			$ 75,164
Expected Dividend Yield			0.00%	0.00%	0.00%
Options, exercises in period, intrinsic value				$ 21,150	$ 25,484
Options, vested in period, fair value			$ 33,731	$ 28,916	$ 20,510
Weighted average period for recognition			1 year 10 months 24 days
Incentive Plan \| Share-based Payment Arrangement, Option
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Options, grants in period, gross (in shares) \| shares			6,625,000
Options, exercisable, weighted average remaining contractual term (year)			5 years 5 months 8 days
Options, exercises in period, intrinsic value			$ 3,601
Exercise of stock options, net (in shares) \| shares			935,000
Options, aggregate intrinsic value			$ 12,280
Number of stock option awards vested and expected to vest (in shares) \| shares			33,557,000
Weighted average exercise price of stock option awards vested and expected to vest (in dollars per share) \| $ / shares			$ 8.19
Stock option awards vested and expected to vest, weighted average remaining contractual life (year)			6 years 9 months 7 days
Incentive Plan \| Share-based Payment Arrangement, Option \| Maximum
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Options, exercisable, weighted average remaining contractual term (year)			10 years
Incentive Plan \| Share-based Payment Arrangement, Option \| Vest 25% Each Year Until Fully Vested
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Award vesting rights, percentage			25.00%
Award vesting period (year)			4 years
Incentive Plan \| Share-based Payment Arrangement, Option \| Thirty-six Equal Monthly Installments \| Non-employee Directors
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Award vesting period (year)			3 years
Incentive Plan \| Performance Shares \| Vest Upon Successful Completion of Specific Development Milestones
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Award vesting rights, percentage		85.00%
Options, grants in period, gross (in shares) \| shares		1,250,000
Incentive Plan \| Restricted Stock Units (RSUs)
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Restricted stock units, grants in period, weighted average grant date fair value (in dollars per share) \| $ / shares			$ 6.71	$ 11.20	$ 11.36
Total unrecognized compensation cost			$ 40,981
Weighted average period for recognition			2 years
Incentive Plan \| Performance-based Restricted Stock Units \| Vest 25% Each Year Until Fully Vested
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Total stock-based compensation expense			$ 34	$ 158
Award vesting rights, percentage	25.00%
Incentive Plan \| Performance-based Restricted Stock Units \| Vest Upon Successful Completion of Specific Development Milestones
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Issued performance-based RSU award (in shares) \| shares	221			21,000
Incentive Plan \| Performance-based Restricted Stock Units \| Share-Based Payment Arrangement, Tranche One
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Award vesting rights, percentage	50.00%
Incentive Plan \| Performance-based Restricted Stock Units \| Thirty-six Equal Monthly Installments \| Non-employee Directors
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Award vesting period (year)	3 years
Inducement Plan
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Total stock-based compensation expense			$ 9,958	$ 6,550	$ 6,055
Award vesting period (year)			4 years
Options, grants in period, weighted average grant date fair value (in dollars per share) \| $ / shares			$ 5.79	$ 9.54	$ 9.65
Total unrecognized compensation cost			$ 16,216
Expiration period (year)			10 years
Expected Dividend Yield			0.00%	0.00%	0.00%
Options, exercises in period, intrinsic value				$ 3,710	$ 6,700
Options, vested in period, fair value			$ 7,698	$ 4,659	$ 2,885
Weighted average period for recognition			1 year 6 months
Inducement Plan \| Vest 25% Each Year Until Fully Vested
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Award vesting rights, percentage			25.00%
Inducement Plan \| Share-based Payment Arrangement, Option
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Options, grants in period, gross (in shares) \| shares			1,196,000
Options, exercisable, weighted average remaining contractual term (year)			6 years 10 months 20 days
Expected Dividend Yield			0.00%
Options, exercises in period, intrinsic value			$ 1,803
Exercise of stock options, net (in shares) \| shares			344,000
Options, aggregate intrinsic value			$ 3,434
Number of stock option awards vested and expected to vest (in shares) \| shares			5,122,000
Weighted average exercise price of stock option awards vested and expected to vest (in dollars per share) \| $ / shares			$ 8.68
Stock option awards vested and expected to vest, weighted average remaining contractual life (year)			7 years 8 months 19 days
Inducement Plan \| Restricted Stock Units (RSUs)
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Options, grants in period, weighted average grant date fair value (in dollars per share) \| $ / shares			$ 7.81	$ 13.21	$ 0
Restricted stock units, grants in period, weighted average grant date fair value (in dollars per share) \| $ / shares			$ 7.81
Total unrecognized compensation cost			$ 7,044
Weighted average period for recognition			1 year 9 months 18 days
ESPP
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Total stock-based compensation expense			$ 1,076	$ 1,435	$ 1,523
Restricted stock units, grants in period, weighted average grant date fair value (in dollars per share) \| $ / shares			$ 3.82	$ 6.02	$ 2.80
Number of shares authorized (in shares) \| shares			7,975,000
Number of shares available for grant (in shares) \| shares			5,454,000
Percentage of salary to purchase common stock, maximum			15.00%
Percentage of common stock shares, beginning			85.00%
Percentage of common stock shares, ending			85.00%
Maximum number of shares per employee (in shares) \| shares			3,000
Maximum number of shares per employee, amount			$ 25
Weighted average purchase price of shares purchased (in dollars per share) \| $ / shares			$ 7.68	$ 11.03	$ 6.20

Property and Equipment Textual (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Property, Plant and Equipment [Abstract]
Depreciation and amortization	$ 1,655	$ 1,437	$ 777
Loss on impairment	1,548	$ 0	$ 0
Contract termination	$ 440

Audit Information	12 Months Ended
Audit Information	Dec. 31, 2023
Audit Information [Abstract]
Auditor Name	Ernst & Young LLP
Auditor Location	Raleigh, NC
Auditor Firm ID	42

Lease Obligations -Balance Sheets of the Company's Operating Leases (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Assets:
Operating lease assets, net	$ 8,682	$ 4,242
Operating Lease, Liability [Abstract]
Operating lease liabilities	1,058	1,516
Operating lease liabilities	8,390	4,027
Total operating lease liabilities	$ 9,448	$ 5,543
Operating Lease, Right-of-Use Asset, Statement of Financial Position [Extensible Enumeration]	Other assets	Other assets

Income Taxes (Tables)

12 Months Ended

Dec. 31, 2023

Income Tax Disclosure [Abstract]

Schedule Of Loss Before Provision For Income Taxes

The components of loss before provision for income taxes were as follows (in thousands):


	Years Ended December 31,
	2023		2022		2021
Domestic	$	(206,674)	$	(225,127)	$	(159,632)
Foreign	(19,555)		(19,256)		(22,177)
Loss before provision for income taxes	$	(226,229)	$	(244,383)	$	(181,809)

Schedule of Components of Income Tax (Benefit) Expense

The components of the (benefit) expense for income taxes were as follows (in thousands):


	Years Ended December 31,
	2023		2022		2021
Current expense (benefit) provision:
U.S. Federal and state	$	(45)	$	2,430	$	2,179
Foreign	1,037		292		233
Total current expense provision	992		2,722		2,412
Deferred expense (benefit) provision:
U.S. Federal and state	(120)		11		(159)
Foreign	(562)		—		—
Total deferred expense provision	(682)		11		(159)
Total expense provision	$	310	$	2,733	$	2,253

Summary Of The Differences Between The Company's Effective Tax Rate And Statutory Tax Rate

The differences between the Company’s effective tax rate and the statutory tax rate in 2023, 2022, and 2021 were as follows (in thousands):


	Years Ended December 31,
	2023		2022		2021
Income tax benefit at federal statutory rate (21% for 2023, 2022 and 2021)	$	(47,328)	$	(51,321)	$	(38,175)
State and local income taxes net of federal tax benefit	(3,477)		(1,816)		(2,288)
Permanent items	3,015		(1,608)		(1,343)
Expiration of attribute carryforwards	—		—		(1,057)
Research and development tax credits	(3,301)		(9,793)		(5,994)
Foreign rate differential	2,255		1,862		1,940
Other	1,656		(5,485)		1,216
Change in valuation allowance	47,490		70,894		47,954
Income tax expense	$	310	$	2,733	$	2,253

Schedule Of Unrecognized Tax Benefits

A reconciliation of the beginning and ending amount of unrecognized tax benefits is as follows (in thousands):


	2023		2022
Balance at January 1,	$	13,844	$	9,729
Additions to current period tax positions	825		4,115
Reductions to prior period tax provisions	(307)		—
Balance at December 31,	$	14,362	$	13,844

Schedule Of Deferred Tax Assets And Liabilities

Significant components of the Company’s deferred tax assets and liabilities are as follows (in thousands):


	December 31,
	2023		2022
Deferred tax assets:
Net federal and state operating losses	$	108,753	$	101,600
Research and development credits	88,390		86,321
Royalty income	122,391		115,554
Stock-based compensation	26,113		19,374
Capitalized R&D	75,081		62,794
Leasing obligations	3,076		1,842
Other	17,515		4,354
Total deferred tax assets	441,319		391,839
Deferred tax liabilities:
Fixed assets	(678)		(717)
Right of use asset	(2,872)		(1,525)
Total deferred tax liabilities	(3,550)		(2,242)
Valuation allowance	(437,098)		(389,608)
Net deferred tax assets (liabilities)	$	671	$	(11)

Employee 401(k) Plan Textual (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Retirement Benefits [Abstract]
Matching contributions	$ 5,716	$ 3,758	$ 2,834

Royalty Financing Obligations (Details) - USD ($)				12 Months Ended
Royalty Financing Obligations (Details) - USD ($)	Nov. 19, 2021	Dec. 07, 2020	Mar. 09, 2011	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021	Dec. 01, 2020
Royalties Monetizations [Line Items]
(Loss) gain on extinguishment of debt				$ (29,019,000)	$ 0	$ 55,838,000
RPI 2019 Intermediate Finance Trust
Royalties Monetizations [Line Items]
Shares issued, common stock, premium	$ 4,269,000
RPI 2019 Intermediate Finance Trust \| ORLADEYO
Royalties Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of sublicense revenue in other markets		20.00%
RPI 2019 Intermediate Finance Trust \| ORLADEYO \| Annual Net Sales Under $350,000
Royalties Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of annual net sales in key territories		8.75%
RPI 2019 Intermediate Finance Trust \| ORLADEYO \| Annual Net Sales Between $350,000 and $550,000
Royalties Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of annual net sales in key territories		2.75%
RPI 2019 Intermediate Finance Trust \| ORLADEYO \| Annual Net Sales Over $550,000
Royalties Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of annual net sales in key territories		0.00%
RPI 2019 Intermediate Finance Trust \| ORLADEYO \| Annual Net Sales Under $150,000
Royalties Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of annual net sales in other markets		20.00%
RPI 2019 Intermediate Finance Trust \| ORLADEYO \| Annual Net Sales Between $150,000 and $230,000
Royalties Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of annual net sales in other markets		10.00%
RPI 2019 Intermediate Finance Trust \| ORLADEYO \| Annual Net Sales Over $230,000
Royalties Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of annual net sales in other markets		0.00%
RPI 2019 Intermediate Finance Trust \| BCX9930
Royalties Monetizations [Line Items]
Royalty purchase agreement, royalties, maximum tiered profit share percentage on permitted sales in other markets	3.00%
RPI 2019 Intermediate Finance Trust \| BCX9930 \| Annual Net Sales Over $3 Billions
Royalties Monetizations [Line Items]
Royalty purchase agreement, royalties, annual net sales payment threshold	$ 3,000,000,000
RPI 2019 Intermediate Finance Trust \| Future Royalties Payable
Royalties Monetizations [Line Items]
Proceeds from issuance of debt		$ 125,000,000
Debt issuance costs, net, total				$ 8,532,000	$ 8,532,000
RPI 2021 and 2020 Intermediate Finance Trust \| Future Royalties Payable
Royalties Monetizations [Line Items]
Proceeds from issuance of debt	$ 150,000,000
The 2021 RPI Royalty Purchase Agreement \| Annual Net Sales Under $350,000
Royalties Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of annual net sales in key territories	0.75%
The 2021 RPI Royalty Purchase Agreement \| Annual Net Sales Between $350,000 and $550,000
Royalties Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of annual net sales in key territories	1.75%
Royalty purchase agreement, royalties, percentage of annual net sales in other markets	0.00%
The 2021 RPI Royalty Purchase Agreement \| Annual Net Sales Under $150,000
Royalties Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of sublicense revenue in other markets	3.00%
The 2021 RPI Royalty Purchase Agreement \| Annual Net Sales Between $150,000 and $230,000
Royalties Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of sublicense revenue in other markets	2.00%
The 2021 RPI Royalty Purchase Agreement \| Annual Net Sales Over $230,000
Royalties Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of annual net sales in other markets	0.00%
The 2021 RPI Royalty Purchase Agreement \| BCX9930 \| Annual Net Sales Under $1.5 Million
Royalties Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of annual net sales in key territories	3.00%
The 2021 RPI Royalty Purchase Agreement \| BCX9930 \| Annual Net Sales Between $1.5 And $3 Million
Royalties Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of annual net sales in key territories	2.00%
The 2021 RPI Royalty Purchase Agreement \| BCX9930 \| Annual Net Sales Over $3 Billions
Royalties Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of annual net sales in other markets	0.00%
OMERS Capital Markets \| ORLADEYO
Royalties Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of sublicense revenue in other markets	20.00%
Royalty purchase agreement, royalties, percentage of annual net sales in other markets	10.00%
Royalty purchase agreement, royalties, percentage of purchase price	155.00%
Royalty purchase agreement, royalties, purchase price, amount	$ 150,000,000
OMERS Capital Markets \| ORLADEYO \| Annual Net Sales Under $350,000
Royalties Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of annual net sales	7.50%
Royalty purchase agreement, royalties, percentage of annual net sales, based on reduction in sales	10.00%
OMERS Capital Markets \| ORLADEYO \| Annual Net Sales Between $350,000 and $550,000
Royalties Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of annual net sales	6.00%
Royalty purchase agreement, royalties, percentage of annual net sales, based on reduction in sales	3.00%
OMERS Capital Markets \| ORLADEYO \| Annual Net Sales Over $550,000
Royalties Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of annual net sales	0.00%
Royalty purchase agreement, royalties, percentage of annual net sales, based on reduction in sales	0.00%
OMERS Capital Markets \| ORLADEYO \| Annual Net Sales Under $150,000
Royalties Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of sublicense revenue in other markets	20.00%
OMERS Capital Markets \| ORLADEYO \| Annual Net Sales Between $150,000 and $230,000
Royalties Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of sublicense revenue in other markets	10.00%
OMERS Capital Markets \| ORLADEYO \| Annual Net Sales Over $230,000
Royalties Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of sublicense revenue in other markets	0.00%
OMERS Royalty Agreement
Royalties Monetizations [Line Items]
Effective interest rate				10.00%	10.60%
2020 RPI Royalty Agreement
Royalties Monetizations [Line Items]
Effective interest rate				22.30%	22.40%
2021 RPI Royalty Agreement
Royalties Monetizations [Line Items]
Effective interest rate				0.00%	13.10%
JPR Royalty Sub LLC \| PhaRMA Notes
Royalties Monetizations [Line Items]
Private placement of senior secured notes			$ 30,000,000
Debt instrument, interest rate, stated percentage			14.00%
Secured debt, total							$ 30,000,000
Interest payable							$ 20,614,000
(Loss) gain on extinguishment of debt						25,838,000
JPR Royalty Sub LLC \| PhaRMA Notes \| Fair Value, Inputs, Level 2
Royalties Monetizations [Line Items]
Notes payable, fair value disclosure						$ 30,000,000
JPR Royalty Sub LLC \| Royalty Monetization
Royalties Monetizations [Line Items]
Debt instrument, face amount			$ 30,000,000
Prepayment and repayment fees on Athyrium secured term loans			$ 22,691,000

Royalty Financing Obligations (Tables)

12 Months Ended

Dec. 31, 2023

Advance Royalties [Abstract]

Schedule of Royalty Financing Obligations

The following table shows the activity within the Royalty financing obligations account (in thousands) as well as the effective interest rate as of December 31, 2023:


	2020 RPI Royalty Agreement			2021 RPI Royalty Agreement			OMERS Royalty Agreement			Total
Balance as of December 31, 2020	$	124,717		$	—		$	—		$	124,717
2021 Royalty sale: Royalty financing obligations, net of issuance costs	—			150,833			147,309			298,142
Non-cash Interest expense on Royalty financing obligations	33,308			2,897			1,465			37,670
Royalty revenues paid and payable	(10,801)			(353)			—			(11,154)
Balance as of December 31, 2021	$	147,224		$	153,377		$	148,774		$	449,375
Deferred financing costs	—			(34)			—			(34)
Non-cash Interest expense on Royalty financing obligations	39,994			22,239			14,249			76,482
Royalty revenues paid and payable	(22,237)			(1,931)			—			(24,168)
Balance as of December 31, 2022	$	164,981		$	173,651		$	163,023		$	501,655
Non-cash Interest expense on Royalty financing obligations	38,267			14,188			17,901			70,356
Royalty revenues paid and payable	(28,768)			(2,494)			(9,150)			(40,412)
Balance as of December 31, 2023	$	174,480		$	185,345		$	171,774		$	531,599

Effective interest rate	22.3		%	—		%	10.0		%

Investment - Schedule of Maturity of Investments (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Investments, All Other Investments [Abstract]
Maturing in one year or less	$ 278,344	$ 119,543
Maturing after one year through two years	0	18,077
Estimated Fair Value	$ 278,344	$ 137,620

Consolidated Statements of Stockholders' Deficit - USD ($) $ in Thousands	Total	Common Stock	Common Stock Restricted Stock Units (RSUs)	Additional Paid-In Capital	Additional Paid-In Capital Restricted Stock Units (RSUs)	Accumulated Other Comprehensive Income	Accumulated Deficit
Beginning balance at Dec. 31, 2020	$ (19,262)	$ 1,769		$ 1,002,408		$ 3	$ (1,023,442)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Net loss	(184,062)						(184,062)
Other comprehensive income	174					174
Exercise of stock options, net	13,745	33		13,712
Employee stock purchase plan sales, net	1,989	3		1,986
Issuance of common stock, net	45,730	38		45,692
Issuance of shares to directors in lieu of cash retainer	60			60
Stock-based compensation expense	34,640			34,640
Ending balance at Dec. 31, 2021	(106,986)	1,843		1,098,498		177	(1,207,504)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Net loss	(247,116)						(247,116)
Other comprehensive income	(151)					(151)
Exercise of stock options, net	11,900	26		11,874
Employee stock purchase plan sales, net	2,862	3		2,859
Issuance of common stock, net	3	3
Issuance of shares to directors in lieu of cash retainer	190			190
Stock-based compensation expense	44,701			44,701
Vesting of restricted stock units			$ 4		$ (4)
Ending balance at Dec. 31, 2022	(294,597)	1,879		1,158,118		26	(1,454,620)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Net loss	(226,539)						(226,539)
Other comprehensive income	1,311					1,311
Exercise of stock options, net	6,114	13		6,101
Employee stock purchase plan sales, net	2,596	4		2,592
Issuance of shares to directors in lieu of cash retainer	342			342
Stock-based compensation expense	55,615			55,615
Vesting of restricted stock units			$ 13		$ (13)
Shares withheld for taxes for vesting of restricted stock units, 59 shares	(370)	(1)		(369)
Exercise of warrants, 14,997 shares		150		(150)
Ending balance at Dec. 31, 2023	$ (455,528)	$ 2,058		$ 1,222,236		$ 1,337	$ (1,681,159)

Investments - Fair Value, Recurring Basis (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Marketable Securities [Line Items]
Estimated Fair Value	$ 278,344	$ 137,620
Obligations of U.S. Government and its agencies
Marketable Securities [Line Items]
Estimated Fair Value	277,358	129,371
Corporate debt securities
Marketable Securities [Line Items]
Estimated Fair Value		6,092
Certificates of deposit
Marketable Securities [Line Items]
Estimated Fair Value	986	2,157
Fair Value, Inputs, Level 1
Marketable Securities [Line Items]
Estimated Fair Value	0	0
Fair Value, Inputs, Level 1 \| Obligations of U.S. Government and its agencies
Marketable Securities [Line Items]
Estimated Fair Value	0	0
Fair Value, Inputs, Level 1 \| Corporate debt securities
Marketable Securities [Line Items]
Estimated Fair Value		0
Fair Value, Inputs, Level 1 \| Certificates of deposit
Marketable Securities [Line Items]
Estimated Fair Value	0	0
Fair Value, Inputs, Level 2
Marketable Securities [Line Items]
Estimated Fair Value	278,344	137,620
Fair Value, Inputs, Level 2 \| Obligations of U.S. Government and its agencies
Marketable Securities [Line Items]
Estimated Fair Value	277,358	129,371
Fair Value, Inputs, Level 2 \| Corporate debt securities
Marketable Securities [Line Items]
Estimated Fair Value		6,092
Fair Value, Inputs, Level 2 \| Certificates of deposit
Marketable Securities [Line Items]
Estimated Fair Value	986	2,157
Fair Value, Inputs, Level 3
Marketable Securities [Line Items]
Estimated Fair Value	0	0
Fair Value, Inputs, Level 3 \| Obligations of U.S. Government and its agencies
Marketable Securities [Line Items]
Estimated Fair Value	0	0
Fair Value, Inputs, Level 3 \| Corporate debt securities
Marketable Securities [Line Items]
Estimated Fair Value		0
Fair Value, Inputs, Level 3 \| Certificates of deposit
Marketable Securities [Line Items]
Estimated Fair Value	$ 0	$ 0

Pay vs Performance Disclosure - USD ($) $ in Thousands	12 Months Ended
Pay vs Performance Disclosure - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Pay vs Performance Disclosure
Net loss	$ (226,539)	$ (247,116)	$ (184,062)

- Definition

The portion of profit or loss for the period, net of income taxes, which is attributable to the parent.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 235
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.4-08(g)(1)(ii))
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480678/235-10-S99-1

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Topic 323
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 3
-Subparagraph (c)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481687/323-10-50-3

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Topic 825
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 28
-Subparagraph (f)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482907/825-10-50-28

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Topic 220
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 6
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482765/220-10-50-6

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Topic 250
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 3
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147483443/250-10-50-3

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 250
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Subparagraph (b)(2)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147483443/250-10-50-1

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 815
-SubTopic 40
-Name Accounting Standards Codification
-Section 65
-Paragraph 1
-Subparagraph (f)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480175/815-40-65-1

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 250
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 8
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147483443/250-10-50-8

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Topic 250
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 9
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147483443/250-10-50-9

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Topic 250
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 11
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147483443/250-10-50-11

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Topic 250
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 11
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147483443/250-10-50-11

Reference 12: http://www.xbrl.org/2003/role/disclosureRef
-Topic 250
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 4
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147483443/250-10-50-4

Reference 13: http://www.xbrl.org/2003/role/exampleRef
-Topic 946
-SubTopic 830
-Name Accounting Standards Codification
-Section 55
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+ Details

Consolidated Statements of Stockholders' Deficit Parentheticals - shares	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Exercise of stock options, net (in shares)	1,276,000
Employee stock purchase plan sales, net (in shares)	338,000	260,000	321,000
Issuance of common stock, net (in shares)			3,846,000
Warrants exercised (in shares)	14,997,000	253,000
Shares withheld for taxes for vesting of restricted stock units (in shares)	59,000
Common Stock
Exercise of stock options, net (in shares)		2,573,000	3,290,000
Employee stock purchase plan sales, net (in shares)	338,000	260,000	321,000
Issuance of shares to directors in lieu of cash retainer (in shares)	37,000	32,000	9,000
Common Stock \| Restricted Stock Units (RSUs)
Stock option awards vested (in shares)	1,276,000	439,000

Consolidated Balance Sheets (Parentheticals) - $ / shares	Dec. 31, 2023	Dec. 31, 2022
Statement of Financial Position [Abstract]
Preferred stock, par or stated value (in usd per share)	$ 0.01	$ 0.01
Preferred stock, shares authorized (shares)	5,000,000	5,000,000
Preferred stock, shares outstanding (in shares)	0	0
Common stock, par or stated value (in usd per share)	$ 0.01	$ 0.01
Common stock, shares authorized (in shares)	450,000,000	450,000,000
Common stock, shares, issued (in shares)	205,771,000	187,906,000
Common stock, shares, outstanding (in shares)	205,771,000	187,906,000

Income Taxes Textual (Details) - USD ($) $ in Thousands	12 Months Ended
Income Taxes Textual (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Income Tax Disclosure [Line Items]
Change in valuation allowance	$ 47,490	$ 70,894	$ 47,954
Provisions and accruals for interest and penalties	$ 0	$ 0	$ 0
Open tax year	2018 2019 2020 2021
Research Tax Credit Carryforward
Income Tax Disclosure [Line Items]
Tax credit carryforward, amount	$ 102,752
Domestic Tax Authority
Income Tax Disclosure [Line Items]
Operating loss carryforwards, total	432,328
State and Local Jurisdiction
Income Tax Disclosure [Line Items]
Operating loss carryforwards, total	183,478
Foreign Tax Authority
Income Tax Disclosure [Line Items]
Operating loss carryforwards, total	$ 65,416

Significant Accounting Policies and Concentrations of Risk (Policies)

12 Months Ended

Dec. 31, 2023

Accounting Policies [Abstract]

The Company

BioCryst Pharmaceuticals, Inc. (the “Company”) is a global biotechnology company with a deep commitment to improving the lives of people living with complement-mediated and other rare diseases. The Company leverages its expertise in structure-guided drug design with the goal of developing first-in-class or best-in-class oral small-molecule and protein therapeutics to target difficult-to-treat rare diseases. The Company was founded in 1986 and incorporated in Delaware in 1991, and its headquarters is located in Durham, North Carolina. The Company integrates the disciplines of biology, crystallography, medicinal chemistry and computer modeling to discover and develop small molecule and protein therapeutics through the process known as structure-guided drug design.

The Company’s marketed products include oral, once-daily ORLADEYO® for the prevention of hereditary angioedema (“HAE”) attacks and RAPIVAB® (peramivir injection) for the treatment of acute uncomplicated influenza in the United States. ORLADEYO received regulatory approval in the United States in December 2020. ORLADEYO has also received regulatory approvals in multiple global markets. The Company is commercializing ORLADEYO in each of these territories directly or through distributors, except in Japan where Torii Pharmaceutical Co., Ltd. (“Torii”), the Company’s collaborative partner, conducts certain commercialization activities with respect to ORLADEYO for the prevention of HAE attacks in exchange for certain royalty payments to the Company. In addition to its approval in the United States, peramivir injection has received regulatory approvals in Canada, Australia, Japan, Taiwan and Korea.

Based on the Company’s expectations for revenue and operating expenses, the Company believes its financial resources available at December 31, 2023 will be sufficient to fund its operations for at least the next 12 months. The Company has sustained operating losses for the majority of its corporate history and expects that its total 2024 expenses will exceed its total 2024 revenues. The Company expects to continue to incur operating losses and negative cash flows until revenues reach a level sufficient to support ongoing operations. The Company’s liquidity needs will be largely determined by the success of operations in regard to the successful commercialization of its products and the progression of its product candidates in the future. The Company regularly evaluates other opportunities to fund future operations, including: (1) out-licensing rights to certain of its products or product candidates, pursuant to which the Company would receive cash milestone payments; (2) raising additional capital through equity or debt financings or from other sources, including royalty or other monetization transactions; (3) obtaining additional product candidate regulatory approvals, which would generate revenue, milestone payments and cash flow; (4) reducing spending on one or more research and development programs, including by discontinuing development; (5) restructuring operations to change its overhead structure; and/or (6) securing U.S. Government funding of its programs, including obtaining procurement contracts. The Company may, in the future, issue securities, including common stock, preferred stock, depositary shares, purchase contracts, warrants, debt securities and units, through private placement transactions or registered public offerings. The Company’s future liquidity needs, and ability to address those needs, will largely be determined by the success of its products and product candidates; the timing, scope and magnitude of its research and development and commercial expenses; and key developments and regulatory events and its decisions in the future.

Basis of Presentation

The consolidated financial statements include the accounts of the Company and its subsidiaries. All intercompany transactions and balances among the consolidated entities have been eliminated from the consolidated financial statements.

The Company’s consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States (“U.S. GAAP”). Such consolidated financial statements reflect all adjustments that are, in management’s opinion, necessary to present fairly, in all material respects, the Company’s consolidated financial position, results of operations, and cash flows. There were no adjustments other than normal recurring adjustments. Certain prior year amounts have been reclassified to conform to the current year presentation.

The Company has made certain presentation changes relative to its revenue, which management considers fundamental to understanding the Company’s current business and financial performance related to its primary product, ORLADEYO, including expanded international sales of ORLADEYO, relative to the Company’s other sources of revenue. Accordingly, certain disaggregated revenue information has been provided in this Note 1 and “Note 2—Revenue” to these

consolidated financial statements. These presentation changes have been applied to prior year revenue amounts for consistency and comparability.

Use of Estimates

The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets, liabilities, revenues and expenses, and related disclosure of contingent assets and liabilities. Significant estimates in the Company’s consolidated financial statements have been made relative to the calculation of net product sales, the ORLADEYO and Factor D inhibitors royalty financing obligations, inventory reserves, certain accruals, primarily related to the Company’s research and development expenses, the valuation of stock options and the valuation allowance for deferred tax assets resulting from net operating losses. These estimates are based on historical experience and on various other assumptions that are believed to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. Actual results could differ from those estimates.

Revenue Recognition

The Company recorded the following revenues for the years ended December 31, 2023, 2022, and 2021 (in thousands):


	Years Ended December 31,
	2023		2022		2021
Product sales, net	324,696		267,710		136,350
Collaborative and other revenues	6,716		3,117		20,820
Total revenues	$	331,412	$	270,827	$	157,170

Pursuant to Accounting Standards Codification (“ASC”) Topic 606, the Company recognizes revenue to depict the transfer of promised goods or services to customers in an amount that reflects the consideration to which the entity expects to be entitled in exchange for those goods or services. To achieve this core principle, Topic 606 includes provisions within a five step model that includes (i) identifying the contract with a customer, (ii) identifying the performance obligations in the contract, (iii) determining the transaction price, (iv) allocating the transaction price to the performance obligations, and (v) recognizing revenue when, or as, an entity satisfies a performance obligation.

At contract inception, the Company identifies the goods or services promised within each contract, assesses whether each promised good or service is distinct and determines those that are performance obligations. The Company recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when the performance obligation is satisfied.

Product Sales, Net

The Company’s principal sources of product sales are sales of ORLADEYO, which the Company began shipping to patients in December 2020, sales of peramivir to the Company’s licensing partners, and in prior years, sales of RAPIVAB to the U.S. Department of Health and Human Services (“HHS”) under the Company’s historical procurement contract, which was completed in 2022. In the United States, the Company ships ORLADEYO directly to patients through a single specialty pharmacy, which is considered its customer. In the European Union, United Kingdom and elsewhere, the Company sells ORLADEYO to specialty distributors as well as hospitals and pharmacies, which collectively are considered its customers.

The Company recognizes revenue for sales when its customers obtain control of the product, which generally occurs upon delivery. For ORLADEYO, the Company classifies payments to its specialty pharmacy customer for certain services provided by its customer as selling, general and administrative expenses to the extent such services provided are determined to be distinct from the sale of ORLADEYO.

Net revenue from sales of ORLADEYO is recorded at net selling price (transaction price), which includes estimates of variable consideration for which reserves are established for (i) estimated government rebates, such as Medicaid and Medicare Part D reimbursements, and estimated managed care rebates, (ii) estimated chargebacks, (iii) estimated costs of co-payment assistance programs and (iv) product returns. These reserves are based on the amounts earned or to be claimed

on the related sales and are classified as reductions of accounts receivable or as a current liability. Overall, these reserves reflect the Company’s best estimates of the amount of consideration to which it is entitled based on the terms of the applicable contract. The amount of variable consideration included in the transaction price may be constrained and is included in the net sales price only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized will not occur in a future period. Actual amounts of consideration ultimately received may differ from the Company's estimates. If actual results in the future vary from estimates, the Company adjusts these estimates, which would affect net product revenue and earnings in the period such variances become known.

Government and Managed Care Rebates. The Company contracts with government agencies and managed care organizations or, collectively, third-party payors, so that ORLADEYO will be eligible for purchase by, or partial or full reimbursement from, such third-party payors. The Company estimates the rebates it will provide to third-party payors and deducts these estimated amounts from total gross product revenues at the time the revenues are recognized. These reserves are recorded in the same period in which the revenue is recognized, resulting in a reduction of product revenue and the establishment of a current liability. The Company estimates the rebates that it will provide to third-party payors based upon (i) the Company's contracts with these third-party payors, (ii) the government mandated discounts applicable to government-funded programs, (iii) a range of possible outcomes that are probability-weighted for the estimated payor mix, and (iv) product distribution information obtained from the Company's specialty pharmacy.

Chargebacks. Chargebacks are discounts that occur when certain contracted customers, pharmacy benefit managers, insurance companies, and government programs purchase directly from the Company’s specialty pharmacy. These customers purchase the Company’s product under contracts negotiated between them and the Company’s specialty pharmacy. The specialty pharmacy, in turn, charges back to the Company the difference between the price the specialty pharmacy paid and the negotiated price paid by the contracted customers, which may be higher or lower than the specialty pharmacy’s purchase price from the Company. The Company estimates chargebacks and adjusts gross product revenues and accounts receivable based on the estimates at the time revenues are recognized.

Co-payment assistance and patient assistance programs. Patients who have commercial insurance and meet certain eligibility requirements may receive co-payment assistance. Based upon the terms of the program and co-payment assistance utilization reports received from the specialty pharmacy, the Company is able to estimate the co-payment assistance amounts, which are recorded in the same period in which the related revenue is recognized, resulting in a reduction of product revenue. The Company also offers a patient assistance program that provides free drug product, for a limited period of time, to allow a patient’s insurance coverage to be established. Based on patient assistance program utilization reports provided by the specialty pharmacy, the Company records gross revenue of the product provided and a full reduction of the revenue amount for the free drug discount.

Product returns. The Company does not provide contractual return rights to its customers, except in instances where the product is damaged or defective. Non-acceptance by the patient of shipped drug product by the specialty pharmacy is reflected as a reversal of sales in the period in which the sales were originally recorded. Reserves for estimated non-acceptances by patients are recorded as a reduction of revenue in the period that the related revenue is recognized, as well as a reduction to accounts receivable. Estimates of non-acceptance are based on quantitative information provided by the specialty pharmacy.

Collaborative and Other Revenues

The Company has collaboration and license agreements with a number of third parties, as well as research and development agreements with certain government entities. The Company’s primary sources of revenue from these collaborative and other research and development arrangements are license, service and royalty revenues.

Revenue from license fees, royalty payments, milestone payments, and research and development fees are recognized as revenue when the earnings process is complete and the Company has no further continuing performance obligations or the Company has completed the performance obligations under the terms of the agreement.

Arrangements that involve the delivery of more than one performance obligation are initially evaluated as to whether the intellectual property licenses granted by the Company represent distinct performance obligations. If they are determined to be distinct, the value of the intellectual property licenses would be recognized up front while the research and development service fees would be recognized as the performance obligations are satisfied. For performance obligations based on services performed, the Company measures progress using an input method based on the effort it expends or costs it incurs toward the satisfaction of the performance obligation in relation to the total estimated effort or costs. Variable

consideration is assessed at each reporting period as to whether it is not subject to significant future reversal and, therefore, should be included in the transaction price at the inception of the contract. If a contract includes a fixed or minimum amount of research and development support, this also would be included in the transaction price. Changes to collaborations, such as the extensions of the research term or increasing the number of targets or technology covered under an existing agreement, are assessed for whether they represent a modification or should be accounted for as a new contract. For contracts with multiple performance obligations, revenue is allocated to each performance obligation based on its relative standalone selling price. Standalone selling prices are based on observable prices at which the Company separately sells the products or services. If a standalone selling price is not directly observable, then the Company estimates the standalone selling price using either an adjusted market assessment approach or an expected cost plus margin approach, representing the amount that the Company believes the market is willing to pay for the product or service. Analyzing the arrangement to identify performance obligations requires the use of judgment, and each may be an obligation to deliver services, a right or license to use an asset, or another performance obligation.

Milestone payments are recognized as licensing revenue upon the achievement of specified milestones if (i) the milestone is substantive in nature and the achievement of the milestone was not probable at the inception of the agreement; and (ii) the Company has a right to payment. Any milestone payments received prior to satisfying these revenue recognition criteria are recorded as deferred revenue.

Reimbursements received for direct out-of-pocket expenses related to research and development costs are recorded as revenue in the Consolidated Statements of Comprehensive Loss rather than as a reduction in expenses. Under the Company’s historical contracts with the Biomedical Advanced Research and Development Authority within the HHS (“BARDA/HHS”) and the National Institute of Allergy and Infectious Diseases (“NIAID/HHS”), revenue was recognized as reimbursable direct and indirect costs are incurred.

Under certain of the Company’s license agreements, the Company receives royalty payments based upon its licensees’ net sales of covered products. Royalties are recognized at the later of when (i) the subsequent sale or usage occurs, or (ii) the performance obligation to which some or all of the sales-based or usage-based royalty has been satisfied.

Cash and Cash Equivalents

The Company generally considers cash equivalents to be all cash held in commercial checking accounts, money market accounts, or investments in debt instruments and certificates of deposit with maturities of three months or less at the time of purchase. The carrying value of cash and cash equivalents approximates fair value due to the short-term nature of these items.

Restricted Cash

Total restricted cash was $1,804 and $1,472 as of December 31, 2023 and 2022, respectively, and primarily consisted of $1,493 and $1,449 as of December 31, 2023 and 2022, respectively, for a letter of credit the Company is required to maintain associated with its Birmingham lease.

Investments

The Company invests in high credit quality investments in accordance with its investment policy, which is designed to minimize the possibility of loss. The objective of the Company’s investment policy is to ensure the safety and preservation of invested funds, as well as maintaining liquidity sufficient to meet cash flow requirements. The Company places its excess cash with high credit quality financial institutions, commercial companies, and government agencies in order to limit the amount of its credit exposure. In accordance with its policy, the Company is able to invest in marketable debt securities that may consist of U.S. Government and government agency securities, money market and mutual fund investments, certificates of deposits, municipal and corporate notes and bonds, and commercial paper, among others. The Company’s investment policy requires it to purchase high-quality marketable securities with a maximum individual maturity of three years and requires an average portfolio maturity of no more than 12 months. Some of the securities in which the Company invests may have market risk. This means that a change in prevailing interest rates may cause the principal amount of the investment to fluctuate. To minimize this risk, the Company schedules its investments with maturities that coincide with expected cash flow needs, thus avoiding the need to redeem an investment prior to its maturity date. Accordingly, the Company does not believe it has a material exposure to interest rate risk arising from its investments. Generally, the Company’s investments are not collateralized. The Company has not realized any significant losses from its investments.

The Company classifies all of its investments as available-for-sale. Available-for-sale investments are reported at fair value at each balance sheet date, and include any unrealized holding gains and losses in accumulated other comprehensive income, unless an unrealized loss is considered to be other than temporary, in which case the unrealized loss is charged to operations. The Company reviews its investments for other than temporary declines in fair value below cost basis at the end of each reporting period and whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. Factors considered include whether a decline in fair value below the amortized cost basis is due to credit-related factors or non-credit-related factors, the financial condition and near-term prospects of the Company, and the Company's intent and ability to hold the investment to allow for an anticipated recovery in fair value. A credit-related impairment is recognized as an allowance on the balance sheet with a corresponding adjustment to earnings. Any impairment that is not credit-related is recognized in other comprehensive income, net of applicable taxes unless deemed other than temporary. Realized gains and losses are reflected in interest and other income in the Consolidated Statements of Comprehensive Loss and are determined using the specific identification method with transactions recorded on a settlement date basis. Investments with original maturities at date of purchase beyond three months and which mature at or less than 12 months from the balance sheet date are classified as current. Investments with a maturity beyond 12 months from the balance sheet date are classified as long-term.

Fair Value Measurements

Assets and liabilities recorded at fair value on a recurring basis on the Consolidated Balance Sheets are categorized based upon the level of judgment associated with the inputs used to measure their fair values. Fair value is defined as the exchange price that would be received for an asset or paid to transfer a liability (an exit price) in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. Valuation techniques used to measure fair value must maximize the use of observable inputs and minimize the use of unobservable inputs.

Items measured at fair value on a recurring basis include investments (Note 3). The carrying amounts reflected in the Consolidated Balance Sheets for cash and cash equivalents, trade receivables, prepaid expenses and other current assets, accounts payable and accrued expenses approximate their fair values due to the short-term nature of these assets and liabilities.

Trade Receivables

The majority of the Company’s trade receivables arise from product sales and primarily represent amounts due from its specialty pharmacy customer in the United States and other third-party distributors, hospitals and pharmacies in the European Union, United Kingdom and elsewhere and have standard payment terms that generally require payment within 30 to 90 days.

Receivables from collaborations are recorded for amounts due to the Company related to reimbursable research and development costs from the HHS (Note 15), and royalty receivables from the Company’s partners, including Shionogi, Green Cross, and Torii (Note 15).

Monthly invoices were submitted to the HHS related to reimbursable research and development costs. The Company was also entitled to monthly reimbursement of indirect costs based on rates stipulated in the underlying contract. The Company’s calculations of its indirect cost rates are subject to audit by the U.S. Government.

The Company does not adjust its receivables for the effects of a significant financing component at contract inception if it expects to collect the receivables in one year or less from the time of sale.

The Company provides reserves against trade receivables for estimated losses that may result from a customer's inability to pay. Receivables are evaluated to determine if any reserve or allowance should be recorded based on consideration of the current economic environment, expectations of future economic conditions, specific circumstances and the Company’s own historical collection experience. Amounts determined to be uncollectible are charged or written-off against the reserve.

Inventory

The Company’s inventories primarily relate to ORLADEYO. Additionally, the Company’s inventories include RAPIVAB and peramivir.

The Company values its inventories at the lower of cost or estimated net realizable value. The Company determines the cost of its inventories, which includes amounts related to materials, labor, manufacturing overhead, and shipping and handling costs on a first-in, first-out (FIFO) basis. Raw materials and work-in-process include all inventory costs prior to packaging and labelling, including raw material, active product ingredient, and the drug product. Finished goods include packaged and labelled products.

The Company’s inventories are subject to expiration dating. The Company regularly evaluates the carrying value of its inventories and provides valuation reserves for any estimated obsolete, short-dated or unmarketable inventories. In addition, the Company may experience spoilage of its raw materials and supplies. The Company’s determination that a valuation reserve might be required, in addition to the quantification of such reserve, requires it to utilize significant judgment.

The Company expenses costs related to the production of inventories as research and development expenses in the period incurred until such time it is believed that future economic benefit is expected to be recognized, which generally is reliant upon receipt of regulatory approval. Upon regulatory approval, the Company capitalizes subsequent costs related to the production of inventories.

Property and Equipment

Property and equipment are recorded at cost. Depreciation is computed using the straight-line method over the estimated useful lives of the assets. Computer equipment is depreciated over a life of three years. Laboratory equipment, office equipment, and software are depreciated over a life of five years. Furniture and fixtures are depreciated over a life of seven years. Leasehold improvements are amortized over their estimated useful lives or the expected lease term, whichever is less.

The Company periodically reviews its property and equipment for impairment when events or changes in circumstances indicate that the carrying amount of such assets may not be recoverable. Determination of recoverability is based on an estimate of undiscounted future cash flows resulting from the use of the asset and its eventual disposition. In the event that such cash flows are not expected to be sufficient to recover the carrying amount of the assets, the assets are written down to their estimated fair values. Property and equipment to be disposed of are reported at the lower of carrying amount or fair value less cost to sell.

Accrued Expenses

The Company enters into contractual agreements with third-party vendors who provide research and development, manufacturing, distribution, and other services in the ordinary course of business. Some of these contracts are subject to milestone-based invoicing, and services are completed over an extended period of time. The Company records liabilities under these contractual commitments when it determines an obligation has been incurred, regardless of the timing of the invoice. This process involves reviewing open contracts and purchase orders, communicating with applicable Company personnel to identify services that have been performed on its behalf and estimating the level of service performed and the associated cost incurred for the service when the Company has not yet been invoiced or otherwise notified of actual cost. The majority of service providers invoice the Company monthly in arrears for services performed. The Company makes estimates of accrued expenses as of each balance sheet date in its financial statements based on the facts and circumstances which can include assumptions such as expected patient enrollment, site activation and estimated project duration. The Company periodically confirms the accuracy of its estimates with the service providers and makes adjustments if necessary. Examples of estimated accrued expenses include (i) fees paid to clinical research organizations (“CROs”) in connection with preclinical and toxicology studies and clinical trials; (ii) fees paid to investigative sites in connection with clinical trials; (iii) fees paid to contract manufacturers in connection with the production of the Company’s raw materials, drug substance, drug products, and product candidates; and (iv) professional fees.

The Company bases its expenses related to clinical trials on its estimates of the services received and efforts expended pursuant to contracts with multiple research institutions and CROs that conduct and manage clinical trials on the Company’s behalf. The financial terms of these agreements are subject to negotiation, vary from contract to contract and

may result in uneven payment flows. Payments under some of these contracts depend on factors such as the successful enrollment of patients and the completion of clinical trial milestones. In accruing service fees, the Company estimates the time period over which services will be performed and the level of effort expended in each period. If the actual timing of the performance of services or the level of effort varies from the estimate, the Company will adjust the accrual accordingly. If the Company underestimates or overestimates the level of these costs, actual expenses could differ from such estimates. As of December 31, 2023 and December 31, 2022, the carrying value of accrued expenses approximates their fair value due to their short-term settlement.

Cost of Product Sales

Cost of product sales includes the cost of producing and distributing inventories that are related to product revenue during the respective period, including freight. In addition, shipping and handling costs for product shipments are recorded as incurred. Finally, cost of product sales may also include costs related to excess or obsolete inventory adjustment charges.

Research and Development Expenses

The Company’s research and development costs are expensed when incurred. Research and development expenses include all direct and indirect development costs related to the development of the Company’s portfolio of product candidates. Advance payments for goods or services that will be used or rendered for future research and development activities are deferred and capitalized. Such amounts are recognized as expense when the related goods are delivered or the related services are performed. Research and development expenses include, among other items, personnel costs, including salaries and benefits, manufacturing costs, clinical, regulatory, and toxicology services performed by CROs, materials and supplies, and overhead allocations consisting of various administrative and facilities related costs, as well as termination fees and other commitments associated with discontinued programs. Most of the Company’s manufacturing and clinical and preclinical studies are performed by third-party CROs. Costs for studies performed by CROs are accrued by the Company over the service periods specified in the contracts, and estimates are adjusted based upon the Company’s ongoing review of the level of services actually performed.

Additionally, the Company has license agreements with third parties, such as Albert Einstein College of Medicine of Yeshiva University, Industrial Research, Ltd., and the University of Alabama at Birmingham (“UAB”), which require fees related to sublicense agreements. The Company expenses sublicense payments as incurred.

The Company groups its research and development expenses into two major categories: direct expenses and indirect expenses. Direct expenses consist of compensation for research and development personnel and costs of outside parties to conduct laboratory studies, develop manufacturing processes and manufacture the product candidate, conduct and manage clinical trials, as well as other costs related to the Company’s clinical and preclinical studies. Additionally, direct expenses consist of those costs necessary to discontinue and close out a development program, including termination fees and other commitments. These costs are accumulated and tracked by active program. Indirect expenses consist of lab supplies and services, facility expenses, depreciation of development equipment and other overhead of the Company’s research and development efforts. These costs apply to work on non-active product candidates and the Company’s discovery research efforts.

Selling, General and Administrative Expenses

Selling, general and administrative expenses are primarily comprised of compensation and benefits associated with sales and marketing, finance, human resources, legal, information technology and other administrative personnel. Additionally, selling, general and administrative expenses are comprised of market research, marketing, advertising and legal expenses, including patent costs, licenses and other general and administrative costs.

Advertising costs related to ORLADEYO of $14,404, $14,891 and $5,705 were expensed as incurred for the years ended December 31, 2023, 2022 and 2021 respectively.

All patent related costs are expensed to selling, general and administrative expenses when incurred as recoverability of such expenditures is uncertain.

Leases

The Company leases certain assets under operating and finance leases, which consist of real estate leases, laboratory equipment leases and office equipment leases as of December 31, 2023. The Company accounts for lease obligations in accordance with ASU 2016-02: Leases (Topic 842), which requires a lessee to recognize a right-of-use asset and a lease liability on its balance sheet for most leases.

Certain of the Company’s operating leases provide for renewal options, which can vary by lease. The right-of-use asset and lease liabilities on the Company’s Consolidated Balance Sheets represent payments over the lease term, which includes renewal options for certain real estate leases that the Company is likely to exercise. As part of the Company’s assessment of the lease term, the Company elected the hindsight practical expedient, which allows companies to use current knowledge and expectations when determining the likelihood to extend lease options. Certain operating leases include rent escalation provisions, which the Company recognizes as expense on a straight-line basis. Lease expense for leases with an initial term of twelve months or less was not material.

The discount rate used in the calculation of the Company’s right-of-use asset and lease liability was determined based on the stated rate within each contract when available, or the Company’s collateralized borrowing rate from lending institutions.

The Company has not made any residual value guarantees related to its leases; therefore, the Company has no corresponding liability recorded on its Consolidated Balance Sheets.

Share-Based Compensation

Stock-Based Compensation

All share-based payments, including grants of stock option awards and restricted stock unit awards, are recognized in the Company’s Consolidated Statements of Comprehensive Loss based on their fair values. Stock-based compensation cost is estimated at the grant date based on the fair value of the award and is recognized as expense on a straight-line basis over the requisite service period of the award. Determining the appropriate fair value model and the related assumptions for the model requires judgment, including estimating the life of an award, the stock price volatility, and the expected term. The Company utilizes the Black-Scholes option-pricing model to value its stock option awards and recognize compensation expense on a straight-line basis over the vesting periods. The Company reduces stock-based compensation expense for estimated forfeitures. The estimation of share-based payment awards that will ultimately vest requires judgment, and to the extent actual results or updated estimates differ from the Company’s current estimates, such amounts will be recorded as a cumulative adjustment in the period estimates are revised. In addition, the Company has outstanding performance-based stock options and restricted stock units for which no compensation expense is recognized until “performance” is deemed to have occurred. Significant management judgment is also required in determining estimates of future stock price volatility to be used in the valuation of the options. Actual results, and future changes in estimates, may differ substantially from the Company’s current estimates.

Interest Expense and Deferred Financing Costs

Interest expense primarily relates to the PhaRMA Notes (Note 8), royalty financing obligations (Note 8), the secured term loan borrowings under the Athyrium Credit Agreement (Note 9), and during 2023, the term loan borrowings under the Pharmakon Loan Agreement (Note 9).

Costs directly associated with the borrowings have been capitalized and are netted against the corresponding debt liabilities on the Consolidated Balance Sheets. These costs are being amortized to interest expense over the terms of the corresponding borrowings using the effective interest rate method. When utilizing the effective interest method, in periods in which payment-in-kind (“PIK”) interest was designated and added to the outstanding principal balance of the borrowing, the amortization of the deferred debt fees and issuance costs was accretive.

Interest Expense and Royalty Financing Obligations

The royalty financing obligations are eligible to be repaid based on royalties from net sales of ORLADEYO and BCX10013. Interest expense is accrued using the effective interest rate method over the estimated period each of the related liabilities will be paid. This requires the Company to estimate the total amount of future royalty payments to be generated from product sales over the life of the agreement. The Company imputes interest on the carrying value of each of the royalty financing obligations and records interest expense using an imputed effective interest rate. The Company

reassesses the expected royalty payments each reporting period and accounts for any changes through an adjustment to the effective interest rate on a prospective basis. The assumptions used in determining the expected repayment term of the debt and amortization period of the issuance costs require that the Company make estimates that could impact the carrying value of each of the liabilities, as well as the periods over which associated issuance costs will be amortized. A significant increase or decrease in forecasted net sales could materially impact each of the liability balances, interest expense and the time periods for repayment.

Income Tax

Income Taxes

The liability method is used in the Company’s accounting for income taxes. Under this method, deferred tax assets and liabilities are determined based on differences between financial reporting and tax bases of assets and liabilities and are measured using the enacted tax rates and laws that are expected to be in effect when the differences are expected to reverse.

The Company accounts for uncertain tax positions in accordance with U.S. GAAP. Significant management judgment is required in determining the Company’s provision for income taxes, deferred tax assets and liabilities and any valuation allowance recorded against net deferred tax assets. The Company has recorded a valuation allowance against substantially all potential tax assets, due to uncertainties in its ability to utilize deferred tax assets, primarily consisting of certain net operating losses carried forward, before they expire. The valuation allowance is based on estimates of taxable income in each of the jurisdictions in which the Company operates and the period over which its deferred tax assets will be recoverable.

Beginning in fiscal year 2021, the Company began accruing for U.S. state taxes and foreign income taxes as a result of increased nexus in both U.S. state and foreign jurisdictions where historically the Company had no presence.

In addition, starting in 2022, amendments to Section 174 of the Internal Revenue Code of 1986, as amended (“IRC”), will no longer permit an immediate deduction for research and development expenditures in the tax year that such costs are incurred. Instead, these IRC Section 174 development costs must now be capitalized and amortized over either a five- or 15-year period, depending on the location of the activities performed. The new amortization period begins with the midpoint of any taxable year that IRC Section 174 costs are first incurred, regardless of whether the expenditures were made prior to or after July 1, and runs until the midpoint of year five for activities conducted in the United States or year fifteen in the case of development conducted on foreign soil.

Foreign Currency

The functional currencies of the Company’s foreign subsidiaries primarily are the local currencies of the country in which the subsidiary operates. The Company’s asset and liability accounts are translated at the current exchange rate as of the balance sheet date. Revenue and expense accounts are translated at the average exchange rate over the period. Adjustments resulting from the translation of the financial statements of the Company’s foreign subsidiaries into U.S. dollars are accumulated as a separate component of stockholders’ equity within accumulated other comprehensive income. Gains or losses resulting from transactions denominated in foreign currencies are included in foreign currency losses, net, within the Consolidated Statement of Comprehensive Loss.

Net Loss Per Share

Basic net loss per share is based upon the weighted average number of common shares outstanding during the period. Diluted net loss per share is computed by dividing net loss by the weighted average number of common shares outstanding during the period, after giving consideration to the dilutive effect of potentially dilutive common shares. The Company has generated a net loss in all periods presented, so the diluted net loss per share is equivalent to basic net loss per share for all periods presented herein because common equivalent shares from unexercised stock options, warrants and common shares expected to be issued under the Company’s equity compensation plans would be anti-dilutive. The Company excluded the following potential common shares, presented based on amounts outstanding as of December 31, 2023 and 2022, from the computation of diluted net loss per share attributable to common stockholders because including them would have had an anti-dilutive effect:


	December 31,
	2023	2022
Outstanding stock options	41,032	36,520
Unvested restricted stock unit awards	6,507	4,747
Warrants to purchase common stock	—	15,023
Total	47,539	56,290

Accumulated Other Comprehensive Income

Accumulated other comprehensive income is comprised of cumulative foreign currency translation adjustments and unrealized gains and losses on available-for-sale investments and is disclosed as a separate component of stockholders’ equity. Realized gain and loss amounts on available-for-sale investments are reclassified from accumulated other comprehensive loss and recorded as interest and other income on the Consolidated Statements of Comprehensive Loss. There were no realized gains or losses reclassified out of accumulated other comprehensive income for the years ended December 31, 2023 and 2022. For the year ended December 31, 2021, realized gains of $1 were reclassified out of accumulated other comprehensive income.

Significant Customers and Other Risks

Significant Customers

The Company’s primary sources of revenue and cash flow are the sales of ORLADEYO in the United States and other global markets and, during the years ended December 31, 2022 and 2021, sales of RAPIVAB (peramivir injection) under the Company’s historical procurement contract with the Assistant Secretary for Preparedness and Response within HHS. Additionally, the Company previously received reimbursement of galidesivir (formerly BCX4430) development expenses earned under cost-plus-fixed-fee contracts with BARDA/HHS and NIAID/HHS during the years ended December 31, 2022 and 2021.

ORLADEYO is distributed through an arrangement with a single specialty pharmacy in the United States, which represents the substantial majority of the ORLADEYO net product sales. The specialty pharmacy subsequently sells ORLADEYO to its customers (pharmacy benefit managers, insurance companies, government programs and group purchasing organizations) and dispenses product to patients. The specialty pharmacy’s inability or unwillingness to continue these distribution activities could adversely impact the Company’s business, results of operations and financial condition.

The Company is distributing ORLADEYO in other global markets directly or through distributors, except in Japan where Torii, the Company’s collaborative partner, has the exclusive right to commercialize ORLADEYO.

The Company relied on BARDA/HHS and NIAID/HHS to reimburse predominantly all of the development costs for its galidesivir program and stockpiling sales of RAPIVAB to HHS. Accordingly, reimbursement of these expenses represented a significant portion of the Company’s collaborative and other research and development revenues. All government funding for galidesivir expired in 2022. Additionally, HHS was the primary customer for RAPIVAB, and it exercised the remaining options for the purchase of RAPIVAB under the procurement contract with the Company during 2022.

Further, the Company’s drug development activities are performed by a limited group of third-party vendors. If any of these vendors were unable to perform its services, this could significantly impact the Company’s ability to complete its drug development activities.

Risks from Third-Party Manufacturing and Distribution Concentration

The Company relies on a single source manufacturer for active pharmaceutical ingredient and finished drug product manufacturing of product candidates in development and on a single specialty pharmacy for distribution of approved drug product in the United States. Delays or disruption in the manufacture or distribution of any product could adversely impact the future procurement stockpiling of the Company’s commercial product, commercial revenue and product candidates.

Credit Risk

Cash equivalents and investments are financial instruments that potentially subject the Company to concentration of risk to the extent recorded on the Consolidated Balance Sheets. The Company deposits excess cash with major financial institutions in the United States. Balances may exceed the amount of insurance provided on such deposits. The Company believes it has established guidelines for investment of its excess cash relative to diversification and maturities that maintain safety and liquidity. To minimize the exposure due to adverse shifts in interest rates, the Company maintains a portfolio of investments with an average maturity of approximately 12 months or less.

The Company’s receivables from sales of ORLADEYO are primarily due from one customer, resulting in a concentration of credit risk. Sales of ORLADEYO from the Company to the specialty pharmacy only occur once an order of product has been received by the specialty pharmacy from one of its customers, which include pharmacy benefit managers, insurance companies, government programs and group purchasing organizations.

The majority of the Company’s receivables from collaborations are due from the U.S. Government, for which there is no assumed credit risk.

Recently Adopted Accounting Pronouncements and New Accounting Pronouncements Not Yet Adopted

Recently Adopted Accounting Pronouncements

In December 2020, the FASB issued ASU No. 2019-12 (ASC Topic 740), Simplifying the Accounting for Income Taxes. ASU 2019-12 simplifies accounting for income taxes by removing certain exceptions to the general principles and clarifying existing guidance. This standard is effective for fiscal years, and interim periods within those years, beginning after December 15, 2020. The Company adopted ASU No. 2019-12 as of January 1, 2021. The adoption of this standard did not have a material impact to the Company’s financial position, results of operations or cash flows.

New Accounting Pronouncements Not Yet Adopted

The Company has reviewed new accounting pronouncements that were issued as of December 31, 2023 and does not believe that these pronouncements are either applicable to the Company, or that they will have a material impact on its financial position or results of operations.

Significant Accounting Policies and Concentrations of Risk

12 Months Ended

Dec. 31, 2023

Accounting Policies [Abstract]

Significant Accounting Policies and Concentrations of Risk

The Company

Basis of Presentation

consolidated financial statements. These presentation changes have been applied to prior year revenue amounts for consistency and comparability.

Use of Estimates

Revenue Recognition

The Company recorded the following revenues for the years ended December 31, 2023, 2022, and 2021 (in thousands):


	Years Ended December 31,
	2023		2022		2021
Product sales, net	324,696		267,710		136,350
Collaborative and other revenues	6,716		3,117		20,820
Total revenues	$	331,412	$	270,827	$	157,170

Product Sales, Net

Collaborative and Other Revenues

Cash and Cash Equivalents

Restricted Cash

Investments

Fair Value Measurements

Trade Receivables

Inventory

The Company’s inventories primarily relate to ORLADEYO. Additionally, the Company’s inventories include RAPIVAB and peramivir.

Property and Equipment

Accrued Expenses

Cost of Product Sales

Research and Development Expenses

Selling, General and Administrative Expenses

Advertising costs related to ORLADEYO of $14,404, $14,891 and $5,705 were expensed as incurred for the years ended December 31, 2023, 2022 and 2021 respectively.

All patent related costs are expensed to selling, general and administrative expenses when incurred as recoverability of such expenditures is uncertain.

Leases

The Company has not made any residual value guarantees related to its leases; therefore, the Company has no corresponding liability recorded on its Consolidated Balance Sheets.

Stock-Based Compensation

Interest Expense and Deferred Financing Costs

Interest Expense and Royalty Financing Obligations

Income Taxes

Foreign Currency

Net Loss Per Share


	December 31,
	2023	2022
Outstanding stock options	41,032	36,520
Unvested restricted stock unit awards	6,507	4,747
Warrants to purchase common stock	—	15,023
Total	47,539	56,290

Accumulated Other Comprehensive Income

Significant Customers and Other Risks

Significant Customers

Risks from Third-Party Manufacturing and Distribution Concentration

Credit Risk

The majority of the Company’s receivables from collaborations are due from the U.S. Government, for which there is no assumed credit risk.

Recently Adopted Accounting Pronouncements

New Accounting Pronouncements Not Yet Adopted

Lease Obligations - Balance Sheets of the Company's Finance Leases (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Finance Lease Assets [Abstract]
Finance Lease, Right-of-Use Asset, after Accumulated Amortization	$ 4,322	$ 2,564
Finance Lease, Liability [Abstract]
Finance lease liabilities	1,590	853
Finance lease liabilities	2,845	1,777
Finance lease liabilities	$ 4,435	$ 2,630
Finance Lease, Right-of-Use Asset, Statement of Financial Position [Extensible Enumeration]	Other assets	Other assets

Accrued Expenses

12 Months Ended

Dec. 31, 2023

Accounts Payable and Accrued Liabilities [Abstract]

Accrued Expenses

Accrued expenses consisted of the following (in thousands):


	December 31,
	2023		2022
Compensation and benefits	$	36,518	$	22,125
Revenue-related reserves for discounts and allowances	26,509		14,332
Royalties payable	18,524		7,700
Development costs	13,677		30,360
Inventory	190		4,193
Other	7,464		8,855
Total accrued expenses	$	102,882	$	87,565

Significant Accounting Policies and Concentrations of Risk (Tables)

12 Months Ended

Dec. 31, 2023

Accounting Policies [Abstract]

Summary of Revenues

The Company recorded the following revenues for the years ended December 31, 2023, 2022, and 2021 (in thousands):


	Years Ended December 31,
	2023		2022		2021
Product sales, net	324,696		267,710		136,350
Collaborative and other revenues	6,716		3,117		20,820
Total revenues	$	331,412	$	270,827	$	157,170

The Company recorded the following revenues for the years ended December 31 (in thousands):


	Years Ended December 31,
	2023		2022		2021
ORLADEYO:
U.S.	$	288,361	$	226,358	$	117,836
Outside of U.S.	37,629		25,275		4,719
Total ORLADEYO	325,990		251,633		122,555
Other revenues	5,422		19,194		34,615
Total revenues	$	331,412	$	270,827	$	157,170

Schedule of Antidilutive Securities Excluded from Computation of Earnings Per Share

The Company excluded the following potential common shares, presented based on amounts outstanding as of December 31, 2023 and 2022, from the computation of diluted net loss per share attributable to common stockholders because including them would have had an anti-dilutive effect:


	December 31,
	2023	2022
Outstanding stock options	41,032	36,520
Unvested restricted stock unit awards	6,507	4,747
Warrants to purchase common stock	—	15,023
Total	47,539	56,290

Income Taxes - Schedule of Components of Income Tax (Benefit) Expense (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Current expense (benefit) provision:
U.S. Federal and state	$ (45)	$ 2,430	$ 2,179
Foreign	1,037	292	233
Total current expense provision	992	2,722	2,412
Deferred expense (benefit) provision:
U.S. Federal and state	(120)	11	(159)
Foreign	(562)	0	0
Total deferred expense provision	(682)	11	(159)
Income tax expense	$ 310	$ 2,733	$ 2,253

- Definition

Amount of current income tax expense (benefit) and deferred income tax expense (benefit) pertaining to continuing operations.

+ References + Details

Accrued Expenses (Tables)

12 Months Ended

Dec. 31, 2023

Accounts Payable and Accrued Liabilities [Abstract]

Schedule of Accrued Liabilities

Accrued expenses consisted of the following (in thousands):


	December 31,
	2023		2022
Compensation and benefits	$	36,518	$	22,125
Revenue-related reserves for discounts and allowances	26,509		14,332
Royalties payable	18,524		7,700
Development costs	13,677		30,360
Inventory	190		4,193
Other	7,464		8,855
Total accrued expenses	$	102,882	$	87,565

Income Taxes - Unrecognized Tax Benefits (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022
Income Tax Disclosure [Roll Forward]
Beginning balance	$ 13,844	$ 9,729
Additions to current period tax positions	825	4,115
Reductions to prior period tax provisions	(307)	0
Ending balance	$ 14,362	$ 13,844

Lease Obligations

12 Months Ended

Dec. 31, 2023

Leases [Abstract]

Lease, Obligations

Lease Obligations

The Company leases certain assets under operating and finance leases, which consist of real estate leases, laboratory equipment leases and office equipment leases as of December 31, 2023. Renewal options for the Company’s leases range from 1 to 3 years in length and begin from 2024 through 2030.

Lease expense under operating and finance leases was as follows (in thousands):


	Years Ended December 31,
	2023		2022		2021
Operating lease expense	$	2,018	$	1,578	$	1,660
Finance lease expense:
Amortization of right-of-use assets	$	1,212	$	816	113
Interest on lease liabilities	$	201	$	138	22
Total finance lease expense	$	1,413	$	954	$	135

Other supplemental information related to leases was as follows:


	As of December 31,
	2023		2022
Weighted average remaining lease term:
Operating leases	9.7 years		10.6 years
Finance leases	3.0 years		3.0 years
Weighted average discount rate:
Operating leases	10.88	%	12.54	%
Finance leases	7.46	%	5.00	%

The following table summarizes the presentation in the Consolidated Balance Sheets of the Company’s operating leases (in thousands):


		As of December 31,
	Balance Sheet Location	2023		2022
Operating lease assets:
Operating lease assets, net	Other Assets	$	8,682	$	4,242
Operating lease liabilities:
Current operating lease liabilities	Operating lease liabilities – current liabilities	$	1,058	$	1,516
Non-current operating lease liabilities	Operating lease liabilities – long-term liabilities	8,390		4,027
Total operating lease liabilities		$	9,448	$	5,543

The following table summarizes the presentation in the Consolidated Balance Sheets of the Company’s finance leases (in thousands):


		As of December 31,
	Balance Sheet Location	2023		2022
Finance lease assets:
Finance lease assets, net	Other Assets	$	4,322	$	2,564
Finance lease liabilities:
Current finance lease liabilities	Finance lease liabilities – current liabilities	$	1,590	$	853
Non-current finance lease liabilities	Finance lease liabilities – long-term liabilities	2,845		1,777
Total finance lease liabilities		$	4,435	$	2,630

Operating lease assets are recorded net of accumulated amortization of $4,794 and $3,268 as of December 31, 2023 and 2022, respectively. Finance lease assets are recorded net of accumulated amortization of $2,293 and $1,081 as of December 31, 2023 and 2022, respectively.

Maturities of lease liabilities as of December 31, 2023 are as follows (in thousands):


	Operating Leases		Finance Leases
2024	$	2,014	$	1,857
2025	1,701		1,732
2026	1,509		899
2027	1,463		437
2028	1,312		27
Thereafter	7,952		—
Total lease payments	15,951		4,952
Less imputed interest	(6,503)		(517)
Total	$	9,448	$	4,435

Supplemental cash flow information related to leases was as follows (in thousands):


	Years Ended December 31,
	2023		2022		2021
Cash paid for amounts included in the measurement of lease liabilities:
Operating cash flows for finance leases	$	201	$	898	$	125
Operating cash flows for operating leases	$	1,920	$	1,555	$	1,490
Financing cash flows for finance leases	$	1,165	$	—	$	—

Operating lease assets obtained in exchange for operating lease liabilities:	$	4,695	$	755	$	1,536
Finance lease assets obtained in exchange for finance lease liabilities:	$	2,971	$	1,302	$	2,190
Non-cash increase to operating lease assets due to remeasurement of operating lease liabilities:	$	924	$	—	$	—

Lessee, Finance Leases

Lease Obligations

The Company leases certain assets under operating and finance leases, which consist of real estate leases, laboratory equipment leases and office equipment leases as of December 31, 2023. Renewal options for the Company’s leases range from 1 to 3 years in length and begin from 2024 through 2030.

Lease expense under operating and finance leases was as follows (in thousands):


	Years Ended December 31,
	2023		2022		2021
Operating lease expense	$	2,018	$	1,578	$	1,660
Finance lease expense:
Amortization of right-of-use assets	$	1,212	$	816	113
Interest on lease liabilities	$	201	$	138	22
Total finance lease expense	$	1,413	$	954	$	135

Other supplemental information related to leases was as follows:


	As of December 31,
	2023		2022
Weighted average remaining lease term:
Operating leases	9.7 years		10.6 years
Finance leases	3.0 years		3.0 years
Weighted average discount rate:
Operating leases	10.88	%	12.54	%
Finance leases	7.46	%	5.00	%

The following table summarizes the presentation in the Consolidated Balance Sheets of the Company’s operating leases (in thousands):


		As of December 31,
	Balance Sheet Location	2023		2022
Operating lease assets:
Operating lease assets, net	Other Assets	$	8,682	$	4,242
Operating lease liabilities:
Current operating lease liabilities	Operating lease liabilities – current liabilities	$	1,058	$	1,516
Non-current operating lease liabilities	Operating lease liabilities – long-term liabilities	8,390		4,027
Total operating lease liabilities		$	9,448	$	5,543

The following table summarizes the presentation in the Consolidated Balance Sheets of the Company’s finance leases (in thousands):


		As of December 31,
	Balance Sheet Location	2023		2022
Finance lease assets:
Finance lease assets, net	Other Assets	$	4,322	$	2,564
Finance lease liabilities:
Current finance lease liabilities	Finance lease liabilities – current liabilities	$	1,590	$	853
Non-current finance lease liabilities	Finance lease liabilities – long-term liabilities	2,845		1,777
Total finance lease liabilities		$	4,435	$	2,630

Maturities of lease liabilities as of December 31, 2023 are as follows (in thousands):


	Operating Leases		Finance Leases
2024	$	2,014	$	1,857
2025	1,701		1,732
2026	1,509		899
2027	1,463		437
2028	1,312		27
Thereafter	7,952		—
Total lease payments	15,951		4,952
Less imputed interest	(6,503)		(517)
Total	$	9,448	$	4,435

Supplemental cash flow information related to leases was as follows (in thousands):


	Years Ended December 31,
	2023		2022		2021
Cash paid for amounts included in the measurement of lease liabilities:
Operating cash flows for finance leases	$	201	$	898	$	125
Operating cash flows for operating leases	$	1,920	$	1,555	$	1,490
Financing cash flows for finance leases	$	1,165	$	—	$	—

Operating lease assets obtained in exchange for operating lease liabilities:	$	4,695	$	755	$	1,536
Finance lease assets obtained in exchange for finance lease liabilities:	$	2,971	$	1,302	$	2,190
Non-cash increase to operating lease assets due to remeasurement of operating lease liabilities:	$	924	$	—	$	—

Insider Trading Arrangements

3 Months Ended

12 Months Ended

Dec. 31, 2023

Trading Arrangements, by Individual

Rule 10b5-1 Arrangement Adopted

false

Non-Rule 10b5-1 Arrangement Adopted

false

Rule 10b5-1 Arrangement Terminated

true

Non-Rule 10b5-1 Arrangement Terminated

false

Officer Trading Arrangement [Member]

Trading Arrangements, by Individual

Material Terms of Trading Arrangement

During the three months ended December 31, 2023, the directors and officers of the Company adopted or terminated the “Rule 10b5-1 trading arrangements” or “non-Rule 10b5-1 trading arrangements” (as each term is defined in Item 408(a) of Regulation S-K) set forth in the table below.

Name (Title)

Action

Termination Date

Type of Trading Arrangement

Duration of Trading Arrangement

Aggregate Number of Securities

Jon P. Stonehouse, President and Chief Executive Officer

Termination

November 7, 2023

Rule 10b5-1 trading arrangement

(1)

Anthony J. Doyle, Chief Financial Officer and Treasurer

Termination

November 7, 2023

Rule 10b5-1 trading arrangement

(1)

Helen M. Thackray, M.D., Chief Research & Development Officer

Termination

November 8, 2023

Rule 10b5-1 trading arrangement

(2)

Alane P. Barnes, Chief Legal Officer and Secretary

Termination

November 7, 2023

Rule 10b5-1 trading arrangement

(1)

Charles K. Gayer, Chief Commercial Officer

Termination

November 7, 2023

Rule 10b5-1 trading arrangement

(2)

(1) This trading plan was originally adopted on March 11, 2022, to cover tax withholding obligations, commissions and any fees related to the vesting of restricted stock units, with a scheduled expiration date of December 31, 2025. The aggregate number of securities that would have been sold under this plan is indeterminable because the number of shares that would have been sold to satisfy applicable tax withholding obligations upon vesting is unknown, as the number would vary based on the extent to which vesting conditions were satisfied and the market price of the Company’s common stock at the time of settlement.

(2) This trading plan was originally adopted on March 14, 2022, to cover tax withholding obligations, commissions and any fees related to the vesting of restricted stock units, with a scheduled expiration date of December 31, 2025. The aggregate number of securities that would have been sold under this plan is indeterminable because the number of shares that would have been sold to satisfy applicable tax withholding obligations upon vesting is unknown, as the number would vary based on the extent to which vesting conditions were satisfied and the market price of the Company’s common stock at the time of settlement.

Officer Trading Arrangement [Member] | Jon P. Stonehouse [Member]

Trading Arrangements, by Individual

Name

Jon P. Stonehouse

Title

President and Chief Executive Officer

Rule 10b5-1 Arrangement Terminated

true

Termination Date

November 7, 2023

Officer Trading Arrangement [Member] | Anthony J. Doyle [Member]

Trading Arrangements, by Individual

Name

Anthony J. Doyle

Title

Chief Financial Officer and Treasurer

Rule 10b5-1 Arrangement Terminated

true

Termination Date

November 7, 2023

Officer Trading Arrangement [Member] | Helen M. Thackray [Member]

Trading Arrangements, by Individual

Name

Helen M. Thackray, M.D.

Title

Chief Research & Development Officer

Rule 10b5-1 Arrangement Terminated

true

Termination Date

November 8, 2023

Officer Trading Arrangement [Member] | Alane P. Barnes [Member]

Trading Arrangements, by Individual

Name

Alane P. Barnes

Title

Chief Legal Officer and Secretary

Rule 10b5-1 Arrangement Terminated

true

Termination Date

November 7, 2023

Officer Trading Arrangement [Member] | Charles K. Gayer [Member]

Trading Arrangements, by Individual

Name

Charles K. Gayer

Title

Chief Commercial Officer

Rule 10b5-1 Arrangement Terminated

true

Termination Date

November 7, 2023

Stockholders' Equity Textual (Details) - USD ($) $ / shares in Units, $ in Thousands			12 Months Ended
	Oct. 23, 2023	Nov. 19, 2021	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Subsidiary, Sale of Stock [Line Items]
Issuance of common stock, net (in shares)					3,846,000
Sale of common stock, net			$ 0	$ 0	$ 50,000
Common Stock
Subsidiary, Sale of Stock [Line Items]
Stock issued during period (in shares)	14,997,000
Warrant
Subsidiary, Sale of Stock [Line Items]
Outstanding warrants (in shares)	0
Common Stock Purchase Agreement with RPI
Subsidiary, Sale of Stock [Line Items]
Issuance of common stock, net (in shares)		3,846,000
Shares issued, price per share (in dollars per share)		$ 13.00
Sale of common stock, net		$ 50,000
Shares issued, price per share, premium (in dollars per share)		$ 1.11
Shares issued, price per share, closing price (in dollars per share)		$ 11.89
Shares issued, common stock, premium		$ 4,269

Collaborative and Other Relationships	12 Months Ended
Collaborative and Other Relationships	Dec. 31, 2023
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Collaborative and Other Relationships	Collaborative and Other Relationships Government Collaborations National Institute of Allergy and Infectious Diseases (“NIAID/HHS”) In September 2013, NIAID/HHS contracted with the Company for the development of galidesivir as a treatment for Marburg virus disease and subsequently, Yellow Fever and Ebola virus disease. On September 15, 2021, the Company entered into an amendment to pay for certain additional costs, including additional manufacturing development costs and overhead, and to change the total value of the contract, as amended, to $47,315 from $45,931. All options under the contract have been awarded. In August 2020, NIAID/HHS awarded the Company a new contract, with potential aggregate funding of up to $43,908 if all contract options were exercised, to manufacture and evaluate the safety, efficacy and tolerability of galidesivir. NIAID/HHS made an initial award of $6,326 to the Company under this contract. Revenue related to this contract was recognized during the years ended December 31, 2022 and 2021. Biomedical Advanced Research and Development Authority (“BARDA/HHS”) In March 2015, BARDA/HHS awarded the Company a contract for the continued development of galidesivir as a potential treatment for diseases caused by RNA pathogens, including filoviruses. This BARDA/HHS contract included a base contract of $16,265 to support galidesivir drug manufacturing, as well as $22,855 in additional development options that could be exercised by the government, bringing the potential value of the contract to $39,120. As of December 31, 2022, a total of $20,574 was awarded under exercised options within this contract. The most recent development option was completed as of December 31, 2022. The contracts with NIAID/HHS and BARDA/HHS were cost-plus-fixed-fee contracts. That is, the Company was entitled to receive reimbursement for all costs incurred in accordance with the contract provisions that were related to the development of galidesivir plus a fixed fee, or profit. BARDA/HHS and NIAID/HHS made periodic assessments of progress, and the continuation of the contracts was based on the Company’s performance, the timeliness and quality of deliverables, and other factors. The government had rights under certain contract clauses to terminate these contracts. These contracts were terminable by the government at any time for breach or without cause. As of December 31, 2022, all of the Company’s government funding for galidesivir had expired. U.S. Department of Health and Human Services (“HHS”) In September 2018, HHS awarded the Company a $34,660 contract for the procurement of up to 50,000 doses of RAPIVAB (peramivir injection) over a five-year period. The Company initially delivered 20,000 doses of RAPIVAB under this contract in 2019 for a total price of approximately $13,864. The Company further delivered 20,000 and 9,980 doses of RAPIVAB in 2022 and 2021, respectively, and recorded revenue of $13,864 and $6,918 for the years ending December 31, 2022 and 2021, respectively. As of December 31, 2022, the Company had delivered a total of 49,980 RAPIVAB doses of the 50,000 RAPIVAB doses available under the contract, effectively completing the contract with HHS. ORLADEYO Torii Pharmaceutical Co., Ltd. (“Torii”) On November 5, 2019, the Company entered into a Commercialization and License Agreement with Torii (the “Original Torii Agreement”), granting Torii the exclusive right to commercialize ORLADEYO for the prevention of hereditary angioedema (“HAE”) attacks in Japan. Under the Original Torii Agreement, the Company received an upfront, non-refundable payment of $22,000. The Company received an additional milestone payment of $15,000 in the second quarter of 2021 upon receipt from the Japanese NHI of a reimbursement price approval for ORLADEYO. In addition, the Company was entitled to receive tiered royalty payments, ranging from 20% to 40% of annual net sales of ORLADEYO in Japan during each calendar year. Torii’s royalty payment obligations were subject to customary reductions in certain circumstances, but could not be reduced by more than 50% of the amount that otherwise would have been payable to the Company in the applicable calendar quarter. The Company identified performance obligations under the Original Torii Agreement related to (i) the license to develop and commercialize ORLADEYO, (ii) regulatory approval support, and (iii) reimbursement pricing approval support. These were each determined to be distinct from the other performance obligations. The Company allocated the $22,000 upfront consideration to the identified performance obligations using estimation approaches to determine the standalone selling prices under ASC Topic 606. Specifically, in determining the value related to the license, a valuation approach utilizing risk adjusted discounted cash flow projections was used, and an expected cost plus margin approach was utilized for the other performance obligations. On November 30, 2023, the Company entered into an Amended and Restated Commercialization and License Agreement with Torii (as amended, the “Torii Agreement”). Under the Torii Agreement, the Company is entitled to receive tiered royalty payments, ranging from 20% to 80% of annual net sales of ORLADEYO in Japan during each calendar year. The Company is now responsible for all commercial promotion activities to support ORLADEYO sales in Japan, and Torii is responsible for HAE disease awareness activities in Japan. The Company will receive a 20% royalty on annual Japanese sales below a prespecified threshold and an 80% royalty on annual Japanese sales above the prespecified threshold. Torii’s updated royalty payment obligations commenced upon November 30, 2023 and expire upon the later of (i) the tenth anniversary of the date of first commercial sale of ORLADEYO in Japan, (ii) the expiration of the Company’s patents covering ORLADEYO, and (iii) the expiration of regulatory exclusivity for ORLADEYO in Japan. The Company determined that the Torii Agreement represented a contract modification to be accounted for as if it were part of the Original Torii Agreement under ASC Topic 606. As the performance obligations under the Original Torii agreement had been fully satisfied, the Company was not required to adjust revenue previously recognized. Peramivir Injection (RAPIVAB, RAPIACTA, PERAMIFLU) Shionogi & Co., Ltd. (“Shionogi”) In February 2007, the Company entered into an exclusive license agreement with Shionogi to develop and commercialize peramivir in Japan for the treatment of seasonal and potentially life-threatening human influenza. Under the terms of the agreement, Shionogi obtained rights to injectable formulations of peramivir in Japan. In October 2008, the Company and Shionogi amended the license agreement to expand the territory covered by the agreement to include Taiwan. Shionogi has commercially launched peramivir under the commercial name RAPIACTA in Japan and Taiwan. The Company developed peramivir under a license from UAB and will owe sublicense payments to UAB on any future milestone payments and/or royalties received by the Company from Shionogi. Green Cross Corporation (“Green Cross”) In June 2006, the Company entered into an agreement with Green Cross to develop and commercialize peramivir in Korea. Under the terms of the agreement, Green Cross is responsible for all development, regulatory, and commercialization costs in Korea and the Company is entitled to share in profits resulting from the sale of peramivir in Korea, including the sale of peramivir to the Korean government for stockpiling purposes. Furthermore, Green Cross will pay the Company a premium over its cost to supply peramivir for development and any future marketing of peramivir products in Korea. Other Collaborations Clearside Biomedical, Inc. (“Clearside”) On November 3, 2023, the Company announced that it entered into a license agreement (the “Clearside Agreement”) with Clearside, enabling the Company to develop its investigational plasma kallikrein inhibitor, avoralstat, with Clearside’s SCS Microinjector® to deliver avoralstat to the back of the eye through the suprachoroidal space to treat patients with diabetic macular edema. Under the Clearside Agreement, Clearside received a $5,000 upfront license fee from the Company, which has been recognized in research and development expenses during the year ended December 31, 2023. Clearside is eligible to receive up to an additional $30,000 in clinical and regulatory milestone payments, and up to a total of $47,500 in three post-approval sales-based milestone payments as annual global net sales progress to $2,000,000. The Company will pay Clearside tiered mid-single digit royalties on annual global net product sales, at three tiers, including a top tier of >$1,500,000.

Income Taxes - Loss Before Provision for Income Taxes (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Income Tax Disclosure [Abstract]
Domestic	$ (206,674)	$ (225,127)	$ (159,632)
Foreign	(19,555)	(19,256)	(22,177)
Loss before income taxes	$ (226,229)	$ (244,383)	$ (181,809)

- Definition

Amount of income (loss) from continuing operations, including income (loss) from equity method investments, before deduction of income tax expense (benefit), and income (loss) attributable to noncontrolling interest.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 944
-SubTopic 220
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.7-04(11))
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147483586/944-220-S99-1

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Topic 280
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 22
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482810/280-10-50-22

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Topic 280
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 30
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482810/280-10-50-30

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Topic 280
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 32
-Subparagraph (f)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482810/280-10-50-32

Reference 5: http://www.xbrl.org/2003/role/exampleRef
-Topic 280
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 31
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482810/280-10-50-31

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 280
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 32
-Subparagraph (c)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482810/280-10-50-32

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 942
-SubTopic 235
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.9-05(b)(2))
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479557/942-235-S99-1

Reference 8: http://fasb.org/us-gaap/role/ref/legacyRef
-Name Accounting Standards Codification
-Section 25
-Paragraph 1
-SubTopic 20
-Topic 940
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481913/940-20-25-1

Reference 9: http://fasb.org/us-gaap/role/ref/legacyRef
-Topic 220
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 2
-Subparagraph (SX 210.5-03(10))
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147483621/220-10-S99-2

Reference 10: http://fasb.org/us-gaap/role/ref/legacyRef
-Topic 942
-SubTopic 220
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.9-04(15))
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147483589/942-220-S99-1

+ Details

Royalty Financing Obligations

12 Months Ended

Dec. 31, 2023

Advance Royalties [Abstract]

Royalty Financing Obligations

ORLADEYO and Factor D Inhibitors

On December 7, 2020, the Company and RPI 2019 Intermediate Finance Trust (“RPI”) entered into a Purchase and Sale Agreement (the “2020 RPI Royalty Purchase Agreement”), pursuant to which the Company sold to RPI the right to receive certain royalty payments from the Company for a purchase price of $125,000 in cash (the “2020 RPI Royalty Sale”). Under the 2020 RPI Royalty Purchase Agreement, RPI is entitled to receive tiered, sales-based royalties on net product sales of ORLADEYO in the United States and certain key European markets (collectively, the “Key Territories”), and other markets where the Company sells ORLADEYO directly or through distributors (collectively, the “Direct Sales”) in an amount equal to: (i) 8.75% of aggregate annual net sales of ORLADEYO for annual net sales up to $350,000 and (ii) 2.75% of annual net sales for annual net sales between $350,000 and $550,000. No royalty payments are payable on annual Direct Sales over $550,000.

Under the 2020 RPI Royalty Purchase Agreement, RPI is also entitled to receive a tiered revenue share on ORLADEYO sublicense revenue or net sales by licensees outside of the Key Territories (the “Other Markets”) equal to: (i) 20% of the proceeds received by the Company for upfront license fees and development milestones for ORLADEYO in the Other Markets; (ii) 20% of proceeds received on annual net sales of up to $150,000 in the Other Markets; and (iii) 10% of proceeds received by the Company on annual net sales between $150,000 and $230,000 in the Other Markets. No royalty payments are payable on annual net sales above $230,000 in the Other Markets.

On November 19, 2021, the Company and RPI entered into (i) a Purchase and Sale Agreement (the “2021 RPI Royalty Purchase Agreement” and together with the 2020 RPI Royalty Purchase Agreement, the “RPI Royalty Purchase Agreements”), pursuant to which the Company sold to RPI the right to receive certain royalty payments from the Company for a purchase price of $150,000 in cash, and (ii) a Purchase and Sale Agreement with OCM IP Healthcare Holdings Limited, an affiliate of OMERS Capital Markets (“OMERS”) (the “OMERS Royalty Purchase Agreement” and collectively with the RPI Royalty Purchase Agreements, the “Royalty Purchase Agreements”), pursuant to which the Company sold to OMERS the right to receive certain royalty payments from the Company for a purchase price of an additional $150,000 in cash.

Under the 2021 RPI Royalty Purchase Agreement, RPI is entitled to receive tiered, sales-based royalties on Direct Sales in an amount equal to: (i) 0.75% of aggregate annual net sales of ORLADEYO for annual net sales up to $350,000 and (ii) 1.75% of annual net sales of ORLADEYO for annual net sales between $350,000 and $550,000. No royalty payments are payable on Direct Sales over $550,000. RPI is also entitled to receive a tiered revenue share on ORLADEYO sublicense revenue or net sales by licensees in the Other Markets in an amount equal to (i) 3.0% of proceeds received by the Company on annual net sales of up to $150,000 in the Other Markets and (ii) 2.0% of proceeds received by the Company on annual net sales between $150,000 and $230,000 in the Other Markets. No royalty payments are payable on annual net sales above $230,000 in the Other Markets.

Under the 2021 RPI Royalty Purchase Agreement, RPI is also entitled to receive tiered, sales-based royalties on net product sales of BCX10013 in an amount equal to: (i) 3.0% of worldwide aggregate annual net sales up to $1,500,000 and (ii) 2.0% of worldwide aggregate annual net sales between $1,500,000 and $3,000,000. No royalty payments are payable

on annual net sales above $3,000,000. RPI is also entitled to receive tiered profit share amounts of up to 3.0% from certain other permitted sales in certain other markets.

The royalties payable under the 2021 RPI Royalty Purchase Agreement are in addition to the royalties payable to RPI under the 2020 RPI Royalty Purchase Agreement.

Under the OMERS Royalty Purchase Agreement, commencing with the calendar quarter beginning October 1, 2023, OMERS is entitled to receive tiered, sales-based royalties on Direct Sales in an amount equal to: (i) 7.5% of aggregate annual net sales of ORLADEYO for annual net sales up to $350,000 and (ii) 6.0% of annual net sales of ORLADEYO for annual net sales between $350,000 and $550,000 (with no royalty payments payable on annual Direct Sales over $550,000). For each calendar quarter beginning on or after January 1, 2024, OMERS is entitled to receive tiered, sales-based royalties on Direct Sales in an amount equal to: (i) 10.0% of aggregate annual net sales of ORLADEYO for annual net sales up to $350,000 and (ii) 3.0% of annual net sales of ORLADEYO for annual net sales between $350,000 and $550,000 (with no royalty payments payable on annual Direct Sales over $550,000).

Under the OMERS Royalty Purchase Agreement, OMERS is also entitled to receive a tiered revenue share on ORLADEYO sublicense revenue or net sales by licensees in the Other Markets in an amount equal to: (i) 20.0% of the proceeds received by the Company for upfront license fees and development milestones for ORLADEYO in the Other Markets, (ii) 20.0% of proceeds received by the Company on annual net sales of up to $150,000 in the Other Markets, and (iii) 10.0% of proceeds received by the Company on annual net sales between $150,000 and $230,000 in the Other Markets. No royalty payments are payable on annual net sales above $230,000 in the Other Markets. OMERS is also entitled to receive profit share amounts of up to 10% from certain other permitted sales in certain other markets.

Under the 2020 RPI Royalty Purchase Agreement, the Company is required to make royalty payments of amounts owed to RPI each calendar quarter following the first commercial sale of ORLADEYO in any country. Under the 2021 RPI Royalty Purchase Agreement, the Company is required to make payments to RPI in respect of net sales or sublicense revenue in each calendar quarter from and after October 1, 2021. Under the OMERS Royalty Purchase Agreement, the Company is required to make payments to OMERS in respect of net sales or sublicense revenue in each calendar quarter from and after October 1, 2023. OMERS will no longer be entitled to receive any payments on the date in which aggregate payments actually received by OMERS equals 155.0% of the $150,000 purchase price.

The transactions contemplated by each of the Royalty Purchase Agreements are referred to herein as the “Royalty Sales.”

Under the Royalty Purchase Agreements, the Company has agreed to specified affirmative and negative covenants, including covenants regarding periodic reporting of information by the Company to RPI and OMERS, third-party audits of royalties paid under the Royalty Purchase Agreements, and restrictions on the ability of the Company or any of its subsidiaries to incur indebtedness other than certain royalty sales and as was permitted to be incurred under the terms of the Athyrium Credit Agreement (as defined in Note 9 herein) through its payoff and termination on April 17, 2023 or, subsequent to that date and the Pharmakon Loan Agreement (as defined in Note 9 herein), as applicable. See “Note 9—Debt” for further details on the Athyrium Credit Agreement and the Pharmakon Loan Agreement. The restrictions under the Royalty Purchase Agreements on the ability of the Company or any of its subsidiaries to incur indebtedness are eliminated after the achievement of certain specified milestones in the Royalty Purchase Agreements.

The cash consideration obtained pursuant to the Royalty Purchase Agreements is recorded in “Royalty financing obligations” on the Company’s Consolidated Balance Sheets. The fair value for the royalty financing obligations at the time of the transactions was based on the Company’s estimates of future royalties expected to be paid to the counterparty over the life of the arrangement. The Company subsequently records the obligations at their carrying value using the effective interest method. In order to amortize the royalty financing obligations, the Company utilizes the prospective method to estimate the future royalties to be paid by the Company to the counterparty over the life of the arrangement. Under the prospective method, a new effective interest rate is determined based on the revised estimate of remaining cash flows. The new rate is the discount rate that equates the present value of the revised estimate of remaining cash flows with the carrying amount of the debt, and it will be used to recognize interest expense for the remaining periods. The Company periodically assesses the amount and timing of expected royalty payments using a combination of internal projections and forecasts from external sources. The estimates of future net product sales (and resulting royalty payments) are based on key assumptions including population, penetration, probability of success, and sales price, among others. To the extent such payments are greater or less than the Company’s initial estimates or the timing of such payments is materially different than its original estimates, the Company will prospectively adjust the amortization of the royalty financing obligations and

the effective interest rate. On a quarterly basis, the Company assesses the projected royalty payments relative to the projected interest accretion for the next twelve months to determine if the royalty liability balance is reduced relative to the current outstanding liability, which would signify a repayment of the liability. In such case of excess payments relative to interest accretion for the next twelve months, the excess payments are considered to be a short-term liability and classified within current liabilities on the Company’s Consolidated Balance Sheets.

During the year ended December 31, 2023, the Company adjusted its forecasts related to its BCX10013 program and updated its ORLADEYO forecast based on actual results for the year ended December 31, 2023. The primary factors that impacted forecasts on the BCX10013 development program were development delays, reduced probability of success, reduced pricing assumptions and reduced market share assumptions. These adjustments impacted the amount and timing of expected royalties to be made under the Royalty Purchase Agreements. As a result, the effective interest rate related to the 2020 RPI Royalty Purchase Agreement decreased from 22.4% to 22.3%, the effective interest rate related to the 2021 RPI Royalty Purchase Agreement decreased from 13.1% to 0.0%, and the effective interest rate related to the OMERS Royalty Purchase Agreement decreased from 10.6% to 10.0%.

The following table shows the activity within the Royalty financing obligations account (in thousands) as well as the effective interest rate as of December 31, 2023:


	2020 RPI Royalty Agreement			2021 RPI Royalty Agreement			OMERS Royalty Agreement			Total
Balance as of December 31, 2020	$	124,717		$	—		$	—		$	124,717
2021 Royalty sale: Royalty financing obligations, net of issuance costs	—			150,833			147,309			298,142
Non-cash Interest expense on Royalty financing obligations	33,308			2,897			1,465			37,670
Royalty revenues paid and payable	(10,801)			(353)			—			(11,154)
Balance as of December 31, 2021	$	147,224		$	153,377		$	148,774		$	449,375
Deferred financing costs	—			(34)			—			(34)
Non-cash Interest expense on Royalty financing obligations	39,994			22,239			14,249			76,482
Royalty revenues paid and payable	(22,237)			(1,931)			—			(24,168)
Balance as of December 31, 2022	$	164,981		$	173,651		$	163,023		$	501,655
Non-cash Interest expense on Royalty financing obligations	38,267			14,188			17,901			70,356
Royalty revenues paid and payable	(28,768)			(2,494)			(9,150)			(40,412)
Balance as of December 31, 2023	$	174,480		$	185,345		$	171,774		$	531,599

Effective interest rate	22.3		%	—		%	10.0		%

Deferred issuance costs pursuant to the Royalty financing obligations, which consist primarily of advisory and legal fees, totaled $8,532 as of December 31, 2023 and 2022. The Royalty financing obligations liabilities and the associated deferred issuance costs are amortized using the effective interest method over the term of the arrangement.

Concurrent with entering into the 2021 RPI Royalty Purchase Agreement, the Company and RPI entered into a Common Stock Purchase Agreement (the “Common Stock Purchase Agreement”), pursuant to which the Company sold common stock to RPI for a premium of $4,269. This premium has been deferred and is being amortized through interest expense using the effective interest method over the term of the applicable arrangement. See “Note 11—Stockholders’ Equity” for further details on the common stock sale premium.

RAPIACTA

Overview

On March 9, 2011, the Company completed a $30,000 financing transaction to monetize certain future royalty and milestone payments under the Company’s agreement (the “Shionogi Agreement”) with Shionogi & Co., Ltd. (“Shionogi”), pursuant to which Shionogi licensed from the Company the rights to market RAPIACTA in Japan and Taiwan. The Company received net proceeds of $22,691 from the transaction.

As part of the transaction, the Company entered into a purchase and sale agreement dated as of March 9, 2011 with JPR Royalty Sub LLC, a wholly-owned subsidiary of the Company (“Royalty Sub”), whereby the Company transferred to Royalty Sub, among other things, its rights to receive certain royalty and milestone payments from Shionogi arising under the Shionogi Agreement. Royalty payments are paid by Shionogi in Japanese Yen, and any milestone payments will be paid in U.S. dollars. The Company’s collaboration with Shionogi was not impacted as a result of this transaction.

Non-Recourse Notes Payable

On March 9, 2011, Royalty Sub completed a private placement to institutional investors of $30,000 in aggregate principal amount of its PhaRMA Senior Secured 14% Notes due on December 1, 2020 (the “PhaRMA Notes”). The PhaRMA Notes were issued by Royalty Sub under an Indenture, dated as of March 9, 2011 (the “Indenture”), by and between Royalty Sub and U.S. Bank National Association, as Trustee. Principal and interest on the PhaRMA Notes are payable from, and are secured by, the rights to royalty and milestone payments under the Shionogi Agreement transferred by the Company to Royalty Sub. The PhaRMA Notes bear interest at 14% per annum, payable annually in arrears on September 1st of each year. The Company remains entitled to receive any royalties and milestone payments related to sales of peramivir by Shionogi following repayment of the PhaRMA Notes. Royalty Sub’s obligations to pay principal and interest on the PhaRMA Notes are obligations solely of Royalty Sub and are without recourse to any other person, including the Company, except to the extent of the Company’s pledge of its equity interests in Royalty Sub in support of the PhaRMA Notes.

In September 2014, Royalty Sub was unable to pay the accrued interest obligation due September 3, 2013. Under the terms of the Indenture, Royalty Sub’s inability to pay the full amount of interest payable in September 2013 by the next succeeding payment date for the PhaRMA Notes, which was September 1, 2014, constituted an event of default. Due to the non-recourse nature of the PhaRMA Notes, in the event of any potential foreclosure, the primary impact to the Company would be the loss of future royalty payments, if any, from Shionogi and legal costs associated with retiring the PhaRMA Notes. The PhaRMA Notes had a final legal maturity date of December 1, 2020, at which time the outstanding principal amount of the PhaRMA Notes of $30,000, together with accrued and unpaid interest of $20,614, was due in full.

Non-Recourse Notes Payable – Debt Extinguishment

During 2021, the royalty-bearing patents associated with RAPIACTA in Japan expired. Accordingly, the Company evaluated the current circumstances of the PhaRMA Notes, including (i) their non-recourse nature relative to the Company, (ii) the current state of default since September 1, 2014 and the legal maturity on December 1, 2020 and (iii) the loss of patent protection relative to RAPIACTA in Japan, upon which any significant repayment of the PhaRMA Notes is predicated. As a result, the Company determined that it was no longer the financial obligor, and as a result, the principal balance of $30,000 and associated accrued interest payable balance of $25,838 were written off, resulting in a gain on extinguishment recorded in other income (expense) for the year ended December 31, 2021.

Significant Accounting Policies and Concentrations of Risk Textual (Details) - USD ($)	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021	Apr. 17, 2023
Significant Accounting Policies and Concentrations of Risk [Line Items]
Restricted cash	$ 1,804,000	$ 1,472,000
Long-term investment maturity, minimum (in months)	12 months
Advertising expense	$ 14,404,000	$ 14,891,000	$ 5,705,000
Antidilutive securities excluded from computation of earnings per share, amount (in shares)	47,539,000	56,290,000
Reclassification from accumulated other comprehensive income, current period, before tax	$ 0	$ 0	$ 1,000
Computer Equipment
Significant Accounting Policies and Concentrations of Risk [Line Items]
Property, plant and equipment, useful life (in years)	3 years
Laboratory Equipment, Office Equipment and Software
Significant Accounting Policies and Concentrations of Risk [Line Items]
Property, plant and equipment, useful life (in years)	5 years
Furniture and fixtures
Significant Accounting Policies and Concentrations of Risk [Line Items]
Property, plant and equipment, useful life (in years)	7 years
Royalty Receivable
Significant Accounting Policies and Concentrations of Risk [Line Items]
Restricted cash	$ 1,804,000	1,472,000
Collateral for Credit
Significant Accounting Policies and Concentrations of Risk [Line Items]
Restricted cash	$ 1,493,000	$ 1,449,000
Maximum
Significant Accounting Policies and Concentrations of Risk [Line Items]
Maturity period of high quality marketable securities (in years)	3 years
Average maturity period of high quality marketable securities (in months)	12 months
Maturity period of short term investment (in months)	12 months
Pharmakon Advisors, LP \| Term Loan Tranche A \| Line of Credit
Significant Accounting Policies and Concentrations of Risk [Line Items]
Interest expense, debt, total	$ 27,326,000
Debt instrument, face amount				$ 300,000,000

Consolidated Statements of Cash Flows - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Cash flows from operating activities:
Net loss	$ (226,539)	$ (247,116)	$ (184,062)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation and amortization	1,655	1,437	777
Inventory obsolescence	422	932	0
Stock-based compensation expense	55,615	44,701	34,640
Non-cash interest expense on royalty financing obligations and secured term loan and amortization of debt issuance costs	85,803	98,918	54,204
Amortization of premium/discount on investments	(10,263)	(1,777)	(2)
Loss (gain) on extinguishment of debt	29,019	0	(55,838)
Loss on impairment	1,548	0	0
Changes in operating assets and liabilities:
Receivables	(6,095)	(21,470)	(20,817)
Inventory	(1,450)	(12,423)	(8,767)
Prepaid expenses and other assets	(6,820)	(2,583)	(7,155)
Accounts payable and accrued expenses	(16,806)	(22,360)	39,412
Interest payable	0	0	4,168
Deferred revenue	(1,230)	(109)	1,283
Net cash used in operating activities	(95,141)	(161,850)	(142,157)
Cash flows from investing activities:
Acquisitions of property and equipment	(2,168)	(1,351)	(2,385)
Purchases of investments	(514,407)	(244,283)	(10,012)
Sales and maturities of investments	385,077	117,396	28,201
Net cash (used in) provided by investing activities	(131,498)	(128,238)	15,804
Cash flows from financing activities:
Sale of common stock, net	0	0	50,000
Net proceeds from common stock issued under stock-based compensation plans	8,340	14,765	15,734
Net proceeds from common stock issued to directors in lieu of cash retainer	342	190	60
Withholding taxes paid on stock-based awards	(2,172)	0	0
Net proceeds from secured term loans	300,000	73,072	0
Repayment of Athyrium secured term loans principal	(240,452)	0	0
Prepayment and repayment fees on Athyrium secured term loans	(21,261)	0	0
Payment of debt issuance costs on Pharmakon Tranche A term loan	(11,147)	0	0
Principal payments on finance lease liabilities	(1,165)	0	0
Net proceeds from royalty financing liabilities	0	0	293,874
Net cash provided by financing activities	32,485	88,027	359,668
Effects of exchange rates on cash, cash equivalents and restricted cash	362	566	71
(Decrease) increase in cash, cash equivalents and restricted cash	(193,792)	(201,495)	233,386
Cash, cash equivalents and restricted cash at beginning of year	306,239	507,734	274,348
Cash, cash equivalents and restricted cash at end of year	112,447	306,239	507,734
Supplemental cash flow disclosure:
Cash paid for interest	22,139	0	900
Cash paid for taxes	1,434	3,542	118
Taxes withheld on stock-based awards included in accrued expenses	$ 4,199	$ 1,990	$ 984

Consolidated Statements of Comprehensive Loss - USD ($) shares in Thousands, $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Revenues [Abstract]
Revenues	$ 331,412	$ 270,827	$ 157,170
Expenses:
Cost of product sales	4,481	6,408	7,229
Research and development	216,566	253,297	208,808
Selling, general and administrative	213,894	159,371	118,818
Royalty	180	186	35
Total operating expenses	435,121	419,262	334,890
Loss from operations	(103,709)	(148,435)	(177,720)
Interest and other income	15,777	5,127	62
Interest expense	(108,239)	(99,092)	(59,294)
(Loss) gain on extinguishment of debt	(29,019)	0	55,838
Foreign currency losses, net	(1,039)	(1,983)	(695)
Loss before income taxes	(226,229)	(244,383)	(181,809)
Income tax expense	310	2,733	2,253
Net loss	(226,539)	(247,116)	(184,062)
Foreign currency translation adjustment	180	890	189
Unrealized gain (loss) on available for sale investments	1,131	(1,041)	(15)
Net comprehensive loss	$ (225,228)	$ (247,267)	$ (183,888)
Earnings per share, basic (in usd per share)	$ (1.18)	$ (1.33)	$ (1.03)
Earnings per share, diluted (in usd per share)	$ (1.18)	$ (1.33)	$ (1.03)
Weighted average shares outstanding, basic (in shares)	192,198	185,908	179,117
Weighted average shares outstanding, diluted (in shares)	192,198	185,908	179,117

Property and Equipment - Property and Equipment (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Property, Plant and Equipment [Line Items]
Total property and equipment	$ 19,120	$ 18,167
Less accumulated depreciation and amortization	(11,210)	(9,550)
Property and equipment, net	7,910	8,617
Furniture and fixtures
Property, Plant and Equipment [Line Items]
Total property and equipment	1,384	1,308
Office equipment
Property, Plant and Equipment [Line Items]
Total property and equipment	719	633
Software
Property, Plant and Equipment [Line Items]
Total property and equipment	1,252	1,501
Laboratory equipment
Property, Plant and Equipment [Line Items]
Total property and equipment	5,559	4,588
Leasehold improvements
Property, Plant and Equipment [Line Items]
Total property and equipment	$ 10,206	$ 10,137

Consolidated Balance Sheets - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
ASSETS
Cash and cash equivalents	$ 110,643	$ 304,767
Restricted cash	1,804	1,472
Investments	278,344	119,543
Trade receivables	56,950	50,599
Inventory, net	28,683	27,533
Prepaid expenses and other current assets	19,542	12,586
Total current assets	495,966	516,500
Property and equipment, net	7,910	8,617
Long-term investments	0	18,077
Other assets	13,084	6,806
Total assets	516,960	550,000
LIABILITIES AND STOCKHOLDERS’ DEFICIT
Accounts payable	20,893	14,356
Accrued expenses	102,882	87,565
Deferred revenue	0	1,224
Operating lease liabilities	1,058	1,516
Finance lease liabilities	1,590	853
Royalty financing obligations	23,565	0
Total current liabilities	149,988	105,514
Operating lease liabilities	8,390	4,027
Finance lease liabilities	2,845	1,777
Royalty financing obligations	508,034	501,655
Secured term loan	303,231	231,624
Stockholders’ deficit:
Preferred stock, $0.01 par value; shares authorized — 5,000; no shares outstanding at December 31, 2023 and 2022	0	0
Common stock, $0.01 par value; shares authorized — 450,000; shares issued and outstanding – 205,771 and 187,906 at December 31, 2023 and 2022, respectively	2,058	1,879
Additional paid-in capital	1,222,236	1,158,118
Accumulated other comprehensive income	1,337	26
Accumulated deficit	(1,681,159)	(1,454,620)
Total stockholders’ deficit	(455,528)	(294,597)
Total liabilities and stockholders’ deficit	$ 516,960	$ 550,000

Inventory

12 Months Ended

Dec. 31, 2023

Inventory Disclosure [Abstract]

Inventory

At December 31, 2023 and 2022, the Company’s inventory primarily related to ORLADEYO. Additionally, inventory included RAPIVAB and peramivir, which is manufactured for the Company’s partners.

The Company’s inventories consisted of the following (in thousands):


	December 31,
	2023		2022
Raw materials	$	6,449	$	8,906
Work-in-process	17,591		14,990
Finished goods	6,242		4,814
Total inventory	$	30,282	$	28,710
Reserves	(1,599)		(1,177)
Total inventory, net	$	28,683	$	27,533

During the years ended December 31, 2023 and 2022, the Company evaluated its inventory levels and associated expiration dating relative to the latest sales forecasts for ORLADEYO, RAPIVAB and peramivir and recorded estimated inventory valuation reserves for those inventories at risk of obsolescence.

Income Taxes

12 Months Ended

Dec. 31, 2023

Income Tax Disclosure [Abstract]

Income Taxes

The components of loss before provision for income taxes were as follows (in thousands):


	Years Ended December 31,
	2023		2022		2021
Domestic	$	(206,674)	$	(225,127)	$	(159,632)
Foreign	(19,555)		(19,256)		(22,177)
Loss before provision for income taxes	$	(226,229)	$	(244,383)	$	(181,809)

The components of the (benefit) expense for income taxes were as follows (in thousands):


	Years Ended December 31,
	2023		2022		2021
Current expense (benefit) provision:
U.S. Federal and state	$	(45)	$	2,430	$	2,179
Foreign	1,037		292		233
Total current expense provision	992		2,722		2,412
Deferred expense (benefit) provision:
U.S. Federal and state	(120)		11		(159)
Foreign	(562)		—		—
Total deferred expense provision	(682)		11		(159)
Total expense provision	$	310	$	2,733	$	2,253

The differences between the Company’s effective tax rate and the statutory tax rate in 2023, 2022, and 2021 were as follows (in thousands):


	Years Ended December 31,
	2023		2022		2021
Income tax benefit at federal statutory rate (21% for 2023, 2022 and 2021)	$	(47,328)	$	(51,321)	$	(38,175)
State and local income taxes net of federal tax benefit	(3,477)		(1,816)		(2,288)
Permanent items	3,015		(1,608)		(1,343)
Expiration of attribute carryforwards	—		—		(1,057)
Research and development tax credits	(3,301)		(9,793)		(5,994)
Foreign rate differential	2,255		1,862		1,940
Other	1,656		(5,485)		1,216
Change in valuation allowance	47,490		70,894		47,954
Income tax expense	$	310	$	2,733	$	2,253

The Company recognizes the impact of a tax position in its financial statements if it is more likely than not that the position will be sustained on audit based on the technical merits of the position. The Company has concluded that it has an uncertain tax position pertaining to its research and development and orphan drug credit carryforwards. The Company has established these credits based on information and calculations it believes are appropriate and the best estimate of the underlying credit. Any changes to the Company’s unrecognized tax benefits are offset by an adjustment to the valuation

allowance and there would be no impact on the Company’s financial statements. The Company does not expect its unrecognized tax benefits to change significantly over the next 12 months.

A reconciliation of the beginning and ending amount of unrecognized tax benefits is as follows (in thousands):


	2023		2022
Balance at January 1,	$	13,844	$	9,729
Additions to current period tax positions	825		4,115
Reductions to prior period tax provisions	(307)		—
Balance at December 31,	$	14,362	$	13,844

The Company’s ability to utilize the net operating loss and tax credit carryforwards in the future may be subject to substantial restrictions in the event of past or future ownership changes as defined in Section 382 of the IRC and similar state tax law.

Significant components of the Company’s deferred tax assets and liabilities are as follows (in thousands):


	December 31,
	2023		2022
Deferred tax assets:
Net federal and state operating losses	$	108,753	$	101,600
Research and development credits	88,390		86,321
Royalty income	122,391		115,554
Stock-based compensation	26,113		19,374
Capitalized R&D	75,081		62,794
Leasing obligations	3,076		1,842
Other	17,515		4,354
Total deferred tax assets	441,319		391,839
Deferred tax liabilities:
Fixed assets	(678)		(717)
Right of use asset	(2,872)		(1,525)
Total deferred tax liabilities	(3,550)		(2,242)
Valuation allowance	(437,098)		(389,608)
Net deferred tax assets (liabilities)	$	671	$	(11)

The majority of the Company’s deferred tax assets relate to net operating loss and research and development carryforwards that can only be realized if the Company is profitable in future periods. It is uncertain whether the Company will realize any tax benefit related to these carryforwards. Accordingly, the Company has provided a valuation allowance against substantially all the net deferred tax assets due to uncertainties as to their ultimate realization. The valuation allowance will remain at the full amount of the deferred tax assets until it is more likely than not that the related tax benefits will be realized. The Company’s valuation allowance increased by $47,490, $70,894, and $47,954 in 2023, 2022, and 2021, respectively.

As of December 31, 2023, the Company had U.S. federal operating loss carryforwards of $432,328, state operating loss carryforwards of $183,478, foreign net operating losses of $65,416, and U.S. research and development and orphan drug credit carryforwards of $102,752, which will expire at various dates from 2024 through 2043. Federal losses, state losses, and research and development credit carryforwards began expiring in 2021. The foreign net operating losses have an indefinite carryforward period.

Tax years 2020-2023 remain open to examination by the major taxing jurisdictions to which the Company is subject. Additionally, years prior to 2020 are also open to examination to the extent of loss and credit carryforwards from those

years. The Company recognizes interest and penalties accrued related to unrecognized tax benefits as components of its income tax provision. However, there were no provisions or accruals for interest and penalties in 2023, 2022, and 2021.

As of December 31, 2023, the Company has minimal accumulated undistributed earnings generated by its foreign subsidiaries which have already been subject to local and U.S. tax (as part of the global intangible low-taxed income provisions). The Company intends to indefinitely reinvest these earnings, as well as future earnings from its foreign subsidiaries to fund its international operations. In addition, the Company expects future U.S. cash generation will be sufficient to meet future U.S. cash needs.

- Definition

The entire disclosure for income taxes. Disclosures may include net deferred tax liability or asset recognized in an enterprise's statement of financial position, net change during the year in the total valuation allowance, approximate tax effect of each type of temporary difference and carryforward that gives rise to a significant portion of deferred tax liabilities and deferred tax assets, utilization of a tax carryback, and tax uncertainties information.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 13
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480990/946-20-50-13

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Topic 235
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.4-08(h)(2))
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480678/235-10-S99-1

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-Name Accounting Standards Codification
-Publisher FASB
-URI https://asc.fasb.org//740/tableOfContent

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 14
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482685/740-10-50-14

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 21
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482685/740-10-50-21

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 270
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482526/740-270-50-1

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 17
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482685/740-10-50-17

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SAB TOPIC 6.I.5.Q1)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479360/740-10-S99-1

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 2
-Subparagraph (SAB Topic 11.C)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479360/740-10-S99-2

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Topic 740
-SubTopic 30
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482603/740-30-50-2

+ Details

Lease Obligations - Supplemental Cash Flows (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Leases [Abstract]
Operating cash flows for finance leases	$ 201	$ 898	$ 125
Operating cash flows for operating leases	1,920	1,555	1,490
Financing cash flows for finance leases	1,165	0	0
Operating lease assets obtained in exchange for operating lease liabilities:	4,695	755	1,536
Finance lease assets obtained in exchange for finance lease liabilities:	2,971	1,302	2,190
Non-cash increase to operating lease assets due to remeasurement of operating lease liabilities:	$ 924	$ 0	$ 0

Cover Page - USD ($)	12 Months Ended
Cover Page - USD ($)	Dec. 31, 2023	Feb. 23, 2024	Jun. 30, 2023
Cover [Abstract]
Document Type	10-K
Document Annual Report	true
Document Period End Date	Dec. 31, 2023
Current Fiscal Year End Date	--12-31
Document Transition Report	false
Entity File Number	000-23186
Entity Registrant Name	BIOCRYST PHARMACEUTICALS, INC.
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	62-1413174
Entity Address, Address Line One	4505 Emperor Blvd., Suite 200
Entity Address, City or Town	Durham
Entity Address, State or Province	NC
Entity Address, Postal Zip Code	27703
City Area Code	919
Local Phone Number	859-1302
Title of 12(b) Security	Common Stock, $0.01 par value
Trading Symbol	BCRX
Security Exchange Name	NASDAQ
Entity Well-known Seasoned Issuer	Yes
Entity Voluntary Filers	No
Entity Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Filer Category	Large Accelerated Filer
Entity Small Business	false
Entity Emerging Growth Company	false
ICFR Auditor Attestation Flag	true
Document Financial Statement Error Correction [Flag]	false
Entity Shell Company	false
Entity Public Float			$ 1,322,900,797
Entity Common Stock, Shares Outstanding		206,149,929
Documents Incorporated by Reference	DOCUMENTS INCORPORATED BY REFERENCE Portions of the registrant’s definitive proxy statement to be filed in connection with the solicitation of proxies for its 2024 annual meeting of stockholders are incorporated by reference into Items 10, 11, 12, 13, and 14 under Part III hereof.
Entity Central Index Key	0000882796
Document Fiscal Year Focus	2023
Document Fiscal Period Focus	FY
Amendment Flag	false

Lease Obligations - Other Supplemental Information Related to Leases (Details)	Dec. 31, 2023	Dec. 31, 2022
Lessee, Operating Lease, Liability, to be Paid, Fiscal Year Maturity [Abstract]
Weighted average remaining lease term (in years)	9 years 8 months 12 days	10 years 7 months 6 days
Weighted average discount rate:	10.88%	12.54%
Finance Lease, Liability, to be Paid, Fiscal Year Maturity [Abstract]
Weighted average remaining lease term (in years)	3 years	3 years
Weighted average discount rate:	7.46%	5.00%

Stockholders' Equity - Shares Reserved For Future Issuance (Details) - shares shares in Thousands	Dec. 31, 2023	Dec. 31, 2022
Subsidiary, Sale of Stock [Line Items]
Shares reserved for future issuance (in shares)	58,020	67,235
Incentive Plan
Subsidiary, Sale of Stock [Line Items]
Shares reserved for future issuance (in shares)	3,376	4,206
Inducement Plan
Subsidiary, Sale of Stock [Line Items]
Shares reserved for future issuance (in shares)	1,651	947
ESPP
Subsidiary, Sale of Stock [Line Items]
Shares reserved for future issuance (in shares)	5,454	5,792
Share-based Payment Arrangement, Option
Subsidiary, Sale of Stock [Line Items]
Shares reserved for future issuance (in shares)	41,032	36,520
Restricted Stock Units (RSUs)
Subsidiary, Sale of Stock [Line Items]
Shares reserved for future issuance (in shares)	6,507	4,747
Warrant
Subsidiary, Sale of Stock [Line Items]
Shares reserved for future issuance (in shares)	0	15,023

Significant Accounting Policies and Concentrations of Risk - Net Loss Per Share (Details) - shares	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022
Significant Accounting Policies and Concentrations of Risk [Line Items]
Antidilutive securities excluded from computation of earnings per share, amount (in shares)	47,539,000	56,290,000
Share-based Payment Arrangement, Option
Significant Accounting Policies and Concentrations of Risk [Line Items]
Antidilutive securities excluded from computation of earnings per share, amount (in shares)	41,032,000	36,520,000
Restricted Stock Units (RSUs)
Significant Accounting Policies and Concentrations of Risk [Line Items]
Antidilutive securities excluded from computation of earnings per share, amount (in shares)	6,507,000	4,747,000
Warrant
Significant Accounting Policies and Concentrations of Risk [Line Items]
Antidilutive securities excluded from computation of earnings per share, amount (in shares)	0	15,023,000

Income Taxes - Differences Between the Company's Effective Tax Rate and the Statutory Tax Rate Narrative (Details)	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Income Tax Disclosure [Abstract]
Income tax benefit, federal statutory rate	21.00%	21.00%	21.00%

Revenue (Tables)

12 Months Ended

Dec. 31, 2023

Revenue Recognition [Abstract]

Summary of Revenues

The Company recorded the following revenues for the years ended December 31, 2023, 2022, and 2021 (in thousands):


	Years Ended December 31,
	2023		2022		2021
Product sales, net	324,696		267,710		136,350
Collaborative and other revenues	6,716		3,117		20,820
Total revenues	$	331,412	$	270,827	$	157,170

The Company recorded the following revenues for the years ended December 31 (in thousands):


	Years Ended December 31,
	2023		2022		2021
ORLADEYO:
U.S.	$	288,361	$	226,358	$	117,836
Outside of U.S.	37,629		25,275		4,719
Total ORLADEYO	325,990		251,633		122,555
Other revenues	5,422		19,194		34,615
Total revenues	$	331,412	$	270,827	$	157,170

Stock-Based Compensation (Tables)

12 Months Ended

Dec. 31, 2023

Share-Based Payment Arrangement [Abstract]

Disclosure of Share-Based Compensation Arrangements by Share-Based Payment Award

The Company recorded the following stock-based compensation expense (in thousands):


	Years Ended December 31,
	2023		2022		2021
Incentive Plan	$	44,581	$	36,716	$	27,062
Inducement Plan	9,958		6,550		6,055
ESPP	1,076		1,435		1,523
Stock-based compensation expense	$	55,615	$	44,701	$	34,640

The following table summarizes stock option activity under the Incentive Plan:


	Shares (in thousands)	Weighted Average Exercise Price per Share		Weighted Average Remaining Contractual Term (in years)	Aggregate Intrinsic Value (in thousands)
Outstanding at December 31, 2022	31,179	$	8.56
Granted	6,625	6.67
Exercised	(935)	5.21			$	3,601
Cancelled or Forfeited	(1,364)	10.00
Outstanding at December 31, 2023	35,505	$	8.24	6.85	$	12,280
Exercisable at December 31, 2023	21,008	$	7.87	5.44	$	12,280
Vested and expected to vest at December 31, 2023	33,557	$	8.19	6.77	$	12,280

The following table summarizes stock option activity under the Inducement Plan:


	Shares (in thousands)	Weighted Average Exercise Price per Share		Weighted Average Remaining Contractual Term (in years)	Aggregate Intrinsic Value (in thousands)
Outstanding at December 31, 2022	5,341	$	8.80
Granted	1,196	8.15
Exercised	(344)	3.70			$	1,803
Cancelled or Forfeited	(666)	10.47
Outstanding at December 31, 2023	5,527	$	8.77	7.76	$	4,463
Exercisable at December 31, 2023	2,448	$	7.17	6.89	$	3,434
Vested and expected to vest at December 31, 2023	5,122	$	8.68	7.72	$	4,291

Stock-Based Compensation Allocation

Total stock-based compensation was allocated as follows:


	Years Ended December 31,
	2023		2022		2021
Research and development	$	29,377	$	24,936	$	20,179
Selling, general and administrative	26,238		19,765		14,461
Total stock-based compensation expense	$	55,615	$	44,701	$	34,640

Weighted Average Assumptions for Stock Option Awards Granted to Employees and Directors Under the Plans

Stock Incentive Plan

The following table summarizes the key assumptions used by the Company to value the stock option awards granted under the Incentive Plan during the years ended December 31, 2023, 2022, and 2021:


	Years Ended December 31,
	2023			2022			2021
Expected Life in Years	5.7			5.5			5.5
Expected Volatility	82.5		%	84.2		%	84.2		%
Expected Dividend Yield	0.0		%	0.0		%	0.0		%
Risk-Free Interest Rate	3.9		%	3.6		%	1.1		%
Weighted average grant date fair value per share	$	4.75		$	7.57		$	7.93

Inducement Equity Incentive Plan

The following table summarizes the key assumptions used by the Company to value the stock option awards granted under the Inducement Plan during the years ended December 31, 2023, 2022, and 2021:


	Years Ended December 31,
	2023			2022			2021
Expected Life in Years	5.6			5.5			5.5
Expected Volatility	83.5		%	84.2		%	84.2		%
Expected Dividend Yield	0.0		%	0.0		%	0.0		%
Risk-Free Interest Rate	4.0		%	3.2		%	0.9		%
Weighted average grant date fair value per share	$	5.79		$	9.54		$	9.65

Share-Based Payment Arrangement, Restricted Stock and Restricted Stock Unit, Activity

The following table summarizes restricted stock unit activity under the Incentive Plan:


	Shares (in thousands)	Weighted Average Grant Date Fair Value
Unvested at December 31, 2022	4,159	$	10.69
Granted	3,106	6.71
Vested	(1,137)	11.04
Forfeited	(536)	7.91
Unvested at December 31, 2023	5,592	$	8.67

The following table summarizes restricted stock unit activity under the Inducement Plan:


	Shares (in thousands)	Weighted Average Grant Date Fair Value
Unvested at December 31, 2022	588	$	13.14
Granted	581	7.81
Vested	(139)	13.13
Forfeited	(115)	11.98
Unvested at December 31, 2023	915	$	9.90

Revenue - Narrative (Details) - USD ($) $ in Thousands	12 Months Ended
Revenue - Narrative (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Disaggregation of Revenue [Line Items]
Revenues	$ 331,412	$ 270,827	$ 157,170
Royalty \| Torii Pharmaceutical Co
Disaggregation of Revenue [Line Items]
Revenues	$ 2,178	$ 1,944	690
Milestone
Disaggregation of Revenue [Line Items]
Revenues			$ 15,000

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References + Details

- Definition

Amount, including tax collected from customer, of revenue from satisfaction of performance obligation by transferring promised good or service to customer. Tax collected from customer is tax assessed by governmental authority that is both imposed on and concurrent with specific revenue-producing transaction, including, but not limited to, sales, use, value-added and excise.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 924
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SAB Topic 11.L)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479941/924-10-S99-1

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 5
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-5

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Topic 280
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 30
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482810/280-10-50-30

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Topic 280
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 42
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482810/280-10-50-42

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Topic 280
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 22
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482810/280-10-50-22

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 280
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 32
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482810/280-10-50-32

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 280
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 40
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482810/280-10-50-40

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 280
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 22
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482810/280-10-50-22

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Topic 280
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 32
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482810/280-10-50-32

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Topic 280
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 41
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482810/280-10-50-41

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 4
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-4

+ Details

Property and Equipment (Tables)

12 Months Ended

Dec. 31, 2023

Property, Plant and Equipment [Abstract]

Summary of Property and Equipment

Property and equipment consisted of the following (in thousands):


	December 31,
	2023		2022
Furniture and fixtures	$	1,384	$	1,308
Office equipment	719		633
Software	1,252		1,501
Laboratory equipment	5,559		4,588
Leasehold improvements	10,206		10,137
Total property and equipment	$	19,120	$	18,167
Less accumulated depreciation and amortization	(11,210)		(9,550)
Property and equipment, net	$	7,910	$	8,617

Investments - Narrative (Details)	Dec. 31, 2023 USD ($) securities
Investments, All Other Investments [Abstract]
Securities \| securities	11
Estimated fair market value \| $	$ 18,513,000

Debt Textual (Details)			12 Months Ended
Debt Textual (Details)	Apr. 17, 2023 USD ($)	Dec. 07, 2020 USD ($)	Dec. 31, 2023 USD ($)	Dec. 31, 2022 USD ($)	Dec. 31, 2021 USD ($)	Jul. 29, 2022 USD ($)
Debt Instrument [Line Items]
Net proceeds from secured term loans			$ 300,000,000	$ 73,072,000	$ 0
Long-term debt, total					298,142,000
Gain (loss) on extinguishment of debt			29,019,000	0	(55,838,000)
Pharmakon Advisors, LP
Debt Instrument [Line Items]
Capitalized interest rate	0.50
Pharmakon Advisors, LP \| Secured Overnight Financing Rate (SOFR) Overnight Index Swap Rate
Debt Instrument [Line Items]
Variable rate floor	0.0175
Debt instrument, basis spread on variable rate	7.00%
Pharmakon Advisors, LP \| Secured Overnight Financing Rate (SOFR) Overnight Index Swap Rate \| PIK Interest Payment is Made
Debt Instrument [Line Items]
Debt instrument, basis spread on variable rate	7.25%
Pharmakon Advisors, LP \| Line of Credit
Debt Instrument [Line Items]
Periodic payment			$ 25,000,000
Debt instrument, prepayment fee, percentage of principal voluntary payments			3.00%
Pharmakon Advisors, LP \| Line of Credit \| Between the Second and Third Anniversaries
Debt Instrument [Line Items]
Debt instrument, prepayment fee, percentage of principal voluntary payments			3.00%
Pharmakon Advisors, LP \| Line of Credit \| After Third Anniversary
Debt Instrument [Line Items]
Debt instrument, prepayment fee, percentage of principal voluntary payments			2.00%
Pharmakon Advisors, LP \| Line of Credit \| After Fourth Anniversary
Debt Instrument [Line Items]
Debt instrument, prepayment fee, percentage of principal voluntary payments			1.00%
Athyrium \| Senior Credit Facility
Debt Instrument [Line Items]
Repayments of lines of credit	$ 241,787,000
MidCap Financial Services, LLC \| Senior Credit Facility
Debt Instrument [Line Items]
Repayments of lines of credit		$ 43,298,000
Loan Agreement \| Pharmakon Advisors, LP
Debt Instrument [Line Items]
Debt instrument, face amount	450,000,000
Net proceeds from secured term loans	25,805,000
Term Loan Tranche A \| Pharmakon Advisors, LP \| Line of Credit
Debt Instrument [Line Items]
Debt instrument, face amount	$ 300,000,000
Net proceeds from secured term loans			$ 300,000,000
Effective interest rate			13.30%
Interest expense, debt, total			$ 27,326,000
Periodic payment, interest			13,663,000
Fair value disclosure			313,663,000
Debt instrument, unamortized discount (premium) and debt issuance costs, net, total			11,147,000
Amortization of debt issuance costs			715,000
Debt instrument, term (month)	18 months
Term Loan Tranche A \| Pharmakon Advisors, LP \| Line of Credit \| PIK Interest Payment is Made
Debt Instrument [Line Items]
Percent of interest payment	0.50
Term Loan Tranche B \| Pharmakon Advisors, LP \| Line of Credit
Debt Instrument [Line Items]
Debt instrument, face amount	$ 50,000,000
Term Loan Tranche C \| Pharmakon Advisors, LP \| Line of Credit
Debt Instrument [Line Items]
Debt instrument, face amount	50,000,000
Term Loan Tranche D \| Pharmakon Advisors, LP \| Line of Credit
Debt Instrument [Line Items]
Debt instrument, face amount	50,000,000
Credit Agreement \| Athyrium
Debt Instrument [Line Items]
Debt instrument, face amount		$ 200,000,000
Debt instrument, LIBOR floor		1.75%
Debt instrument, LIBOR cap		3.50%
Debt instrument, exit fee, percentage of principal		2.00%
Debt instrument, commitment fee percentage		1.00%
Paid-in-kind interest			8,476,000	23,387,000
Long-term debt, total	240,452,000
Interest expense, long-term debt	$ 1,335,000
Debt instrument, term (month)	17 days
Debt instrument, fee amount	$ 17,261,000
Debt issuance costs, net, total	11,758,000
Gain (loss) on extinguishment of debt	$ 29,019,000
Credit Agreement \| Athyrium \| London Interbank Offered Rate (LIBOR)
Debt Instrument [Line Items]
Debt instrument, basis spread on variable rate		8.25%
Credit Agreement \| Athyrium \| London Interbank Offered Rate (LIBOR) \| PIK Interest Payment is Made
Debt Instrument [Line Items]
Debt instrument, basis spread on variable rate		10.25%
Credit Agreement \| Athyrium \| Term A Loan
Debt Instrument [Line Items]
Net proceeds from secured term loans		$ 125,000,000
Amortization of debt issuance costs			$ 1,069,000	$ 916,000	$ 531,000
Credit Agreement \| Athyrium \| Term B Loan
Debt Instrument [Line Items]
Debt instrument, face amount						$ 25,000,000
Credit Agreement \| Athyrium \| Term C Loan
Debt Instrument [Line Items]
Debt instrument, face amount						50,000,000
Credit Agreement \| Athyrium \| Term B And C Loan
Debt Instrument [Line Items]
Debt instrument, face amount						75,000,000
Deferred debt fees and issuance costs						$ 3,428,000
Credit Agreement \| Athyrium \| Term Loans
Debt Instrument [Line Items]
Effective interest rate			13.71%	12.87%

Debt	12 Months Ended
Debt	Dec. 31, 2023
Debt Disclosure [Abstract]
Debt	Debt Pharmakon Loan Agreement On April 17, 2023, the Company entered into a $450,000 Loan Agreement (the “Pharmakon Loan Agreement”) with BioPharma Credit Investments V (Master) LP and BPCR Limited Partnership, as lenders, and BioPharma Credit PLC, as collateral agent for the lenders. Certain of the Company’s wholly-owned subsidiaries are guarantors to the Pharmakon Loan Agreement. The Pharmakon Loan Agreement provides for an initial term loan in the principal amount of $300,000 (the “Tranche A Loan”) funded on April 17, 2023 (the “Tranche A Closing Date”). The Company used a portion of the proceeds from the Tranche A Loan to repay the $241,787 of outstanding indebtedness (principal and interest due as of April 17, 2023) under the then-existing Athyrium Credit Agreement (defined below) and to pay associated transaction costs and fees, and used the remaining net proceeds of $25,805 for other general corporate purposes. The Pharmakon Loan Agreement also provides for three additional term loan tranches, at the Company’s option, in principal amounts of $50,000 each (each a “Subsequent Tranche Loan” and, collectively with the Tranche A Loan, the “Pharmakon Term Loans” and each, a “Pharmakon Term Loan”), which may be requested on or prior to September 30, 2024. The maturity date of the Pharmakon Loan Agreement is April 17, 2028 (the “Maturity Date”), the fifth anniversary of the Tranche A Closing Date. The Pharmakon Loan Agreement provides for quarterly interest-only payments until the Maturity Date, with the unpaid principal amount of the outstanding Pharmakon Term Loans due and payable on the Maturity Date. During the first 18 months following the Tranche A Closing Date, the Company has the option to make a portion of the applicable interest payment on the Tranche A Loan in-kind (a “Pharmakon PIK Interest Payment”) by capitalizing as principal up to 50% of the amount of interest accrued on the Tranche A Loan during the applicable interest period. The Pharmakon Term Loans will bear interest at a rate equal to the three-month Secured Overnight Financing Rate (“SOFR”) rate, which shall be no less than 1.75%, plus 7.00%, per annum or, for each interest period in which a Pharmakon PIK Interest Payment is made, with respect to the Tranche A Loan, SOFR plus 7.25%, per annum. The Tranche A Loan accrued interest at an effective interest rate of 13.30% for the year ended December 31, 2023. The Company is required to make a mandatory prepayment of the Pharmakon Term Loans (i) upon the occurrence of a change of control and (ii) prior to any repayment of any convertible debt that the Company may issue in the future, subject to certain exceptions. The Company may make voluntary prepayments in whole or in part, in minimum $25,000 increments. Prepayments are subject to a prepayment premium equal to, (i) with respect to any prepayment made prior to the second anniversary of the applicable Pharmakon Term Loan borrowing date, the sum of (1) 3.00% of the principal amount of the Pharmakon Term Loan being prepaid plus (2) the aggregate amount of all interest that would have accrued on the principal amount of the Pharmakon Term Loan being prepaid from the date of prepayment through and including the second anniversary of the date of the borrowing of such Pharmakon Term Loan; (ii) with respect to any prepayment made on or after the second anniversary and prior to the third anniversary of the applicable Pharmakon Term Loan borrowing date, 3.00% of the principal amount of the Pharmakon Term Loan being prepaid; (iii) with respect to any prepayment made on or after the third anniversary and prior to the fourth anniversary of the applicable Pharmakon Term Loan, 2.00% of the principal amount of the Pharmakon Term Loan being prepaid; and (iv) with respect to any prepayment made on or after the fourth anniversary of the applicable Pharmakon Term Loan borrowing date and before the Maturity Date, 1.00% of the principal amount of the Pharmakon Term Loan being prepaid. In addition, upon the drawing of any Subsequent Tranche Loan, certain funding fees are required to be paid. The Pharmakon Loan Agreement also contains representations and warranties and affirmative and negative covenants customary for financings of this type, as well as customary events of default. Certain of the customary negative covenants limit the ability of the Company and certain of its subsidiaries to, among other things, dispose of assets, engage in mergers, acquisitions, and similar transactions, incur additional indebtedness, grant liens, make investments, pay dividends or make distributions or certain other restricted payments in respect of equity, prepay other indebtedness, enter into restrictive agreements, undertake fundamental changes or amend certain material contracts, among other customary covenants, in each case subject to certain exceptions. A failure to comply with the covenants in the Pharmakon Loan Agreement, or an occurrence of any other event of default, could permit the lenders under the Pharmakon Loan Agreement to declare the borrowings thereunder, together with accrued interest and fees, and any applicable prepayment premium, to be immediately due and payable. The Company’s obligations under the Pharmakon Loan Agreement are secured by a security interest in, subject to certain exceptions, substantially all of the Company’s assets. As of December 31, 2023, the Company had total borrowings of $300,000 under the Pharmakon Loan Agreement. Interest expense on the Tranche A Loan for the year ended December 31, 2023 totaled $27,326. As allowable under the Pharmakon Loan Agreement, the Company has designated and accounted for 50% of the quarterly interest payments for the year ended December 31, 2023 as a Pharmakon PIK Interest Payment and the amount of $13,663 has been added to the outstanding principal balance of the borrowing. The remaining 50% of the quarterly interest payments of $13,663 have been paid at the end of each quarterly period. As of December 31, 2023, borrowings, including the Pharmakon PIK Interest Payments, totaled $313,663. The fair value of the debt approximates its carrying value based on prevailing interest rates as of the balance sheet date and is considered as Level 2 in the fair value hierarchy. Incurred debt fees and issuance costs associated with the Tranche A Loan under the Pharmakon Loan Agreement totaled $11,147 and have been deferred and are being amortized as interest expense on an effective interest rate method over the remaining term of the Tranche A Loan. Deferred financing amortization of $715 was recognized for the year ended December 31, 2023. Athyrium Credit Agreement On December 7, 2020, the Company entered into a $200,000 Credit Agreement (the “Athyrium Credit Agreement”) with Athyrium Opportunities III Co-Invest 1 LP (“Athyrium”), as lender and as administrative agent for the lenders. Certain of the Company’s direct and indirect subsidiaries were guarantors to the Athyrium Credit Agreement. The Athyrium Credit Agreement provided for an initial term loan in the principal amount of $125,000 (the “Term A Loan”), which was received by the Company on December 7, 2020 and is recorded in “Secured term loan” on the Company’s balance sheet. The Company used a portion of the proceeds from the Term A Loan to repay $43,298 of outstanding indebtedness, including accrued interest, under its prior credit facility with MidCap Financial Trust. The Athyrium Credit Agreement also provided for two additional term loans, at the Company’s option, in the respective principal amounts of $25,000 (the “Term B Loan”) and $50,000 (the “Term C Loan” and, collectively with the Term A Loan and the Term B Loan, the “Athyrium Term Loans”). Having achieved all required revenue-based milestones, the Company exercised its option to draw upon the additional funding available under the Athyrium Credit Agreement, borrowing the principal amounts of $25,000 under the Term B Loan and $50,000 under the Term C Loan. Both the Term B Loan and the Term C Loan were funded on July 29, 2022 in the aggregate principal amount of $75,000. The Company incurred deferred debt fees and issuance costs associated with the Term B and Term C Loans of $3,428. The Term B Loan and the Term C Loan were subject to all the provisions under the Athyrium Credit Agreement. On November 19, 2021, the Company entered into an amendment to the Athyrium Credit Agreement to, among other things, (i) permit the Company to enter into the 2021 RPI Royalty Purchase Agreement, the OMERS Royalty Purchase Agreement, and the other definitive documentation related thereto and to perform its obligations thereunder; and (ii) require the Company to pay to Athyrium, for the account of the lenders, a make-whole premium plus certain fees set forth in the Athyrium Credit Agreement in the event that the Company prepaid or repaid, or was required to prepay or repay, voluntarily or pursuant to mandatory prepayment obligations under the Athyrium Credit Agreement (e.g., with the proceeds of certain asset sales, certain ORLADEYO out-licensing or royalty financing transactions (excluding the Royalty Sales), extraordinary receipts, debt issuances, or upon a change of control of the Company and specified other events, subject to certain exceptions), all of the then-outstanding Athyrium Term Loans, in each case, subject to certain exceptions set forth in the Athyrium Credit Agreement. The Athyrium Credit Agreement provided for quarterly interest-only payments until the maturity date, with the unpaid principal amount of the outstanding Athyrium Term Loans due and payable on the maturity date. For each of the first eight full fiscal quarters following December 7, 2020, the Company had the option to make the applicable interest payment-in-kind (an “Athyrium PIK Interest Payment”) by capitalizing the entire amount of interest accrued during the applicable interest period with the unpaid original principal amount outstanding on the last day of such period. The Athyrium Term Loans accrued interest at a rate equal to the three-month LIBOR rate, which was no less than 1.75% and no more than 3.50% (“LIBOR”), plus 8.25%, or for each interest period in which an Athyrium PIK Interest Payment was made, LIBOR plus 10.25%. The quarter ended December 31, 2022 was the last period eligible for the Athyrium PIK Interest Payment designation. The Athyrium Term Loans accrued interest at an effective interest rate of 13.71% during the period in which the debt was outstanding for the year ended 2023 compared to 12.87% for fiscal year 2022. Quarterly interest payments under the Athyrium Credit Agreement for the year ended December 31, 2023 totaled $8,476. Quarterly interest payments under the Athyrium Credit Agreement for the year ended December 31, 2022 totaled $23,387 and were designated and accounted for as Athyrium PIK Interest Payments and added to the outstanding principal balance of the borrowing. From the Athyrium Term Loans’ inception through December 31, 2022, the quarterly interest payments were designated and accounted for as Athyrium PIK Interest Payments and added to the outstanding principal balance of the borrowing. The quarter ended December 31, 2022 was the last period eligible for the Athyrium PIK Interest Payment designation. Deferred financing amortization of $1,069 and $916, and $531 was recognized for the years ended December 31, 2023, 2022, and 2021 respectively. On April 17, 2023, the outstanding principal of the Athyrium Term Loans, including the Athyrium PIK Interest Payments of $240,452 along with interest accrued of $1,335 for the first 17 days of the quarterly interest period ended June 30, 2023, was repaid with the funding received through the Pharmakon Loan Agreement. In accordance with the Athyrium Credit Agreement, upon the prepayment or repayment of all or any of the Athyrium Term Loans, the Company was obligated to pay an exit fee in an amount equal to 2.00% of the principal amount of the Athyrium Term Loans prepaid or repaid. In addition, each Athyrium Term Loan was subject to a 1.00% commitment fee at its respective borrowing date. As a result, the Company incurred prepayment and final payment fees of $17,261 upon repayment of the Athyrium Term Loans. Additionally, unamortized deferred financing costs of $11,758 associated with the Athyrium Term Loans were written-off at the time of repayment. Collectively, the prepayment and final payment fees and unamortized deferred financing costs totaled $29,019 and are reflected as a one-time loss on extinguishment of debt on the Consolidated Statements of Comprehensive Loss for the year ended December 31, 2023.

- Definition

The entire disclosure for information about short-term and long-term debt arrangements, which includes amounts of borrowings under each line of credit, note payable, commercial paper issue, bonds indenture, debenture issue, own-share lending arrangements and any other contractual agreement to repay funds, and about the underlying arrangements, rationale for a classification as long-term, including repayment terms, interest rates, collateral provided, restrictions on use of assets and activities, whether or not in compliance with debt covenants, and other matters important to users of the financial statements, such as the effects of refinancing and noncompliance with debt covenants.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1B
-Subparagraph (h)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481139/470-20-50-1B

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Topic 235
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.4-08(c))
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480678/235-10-S99-1

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-Name Accounting Standards Codification
-Publisher FASB
-URI https://asc.fasb.org//470/tableOfContent

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
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-Name Accounting Standards Codification
-Section 50
-Paragraph 1B
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-URI https://asc.fasb.org//1943274/2147481139/470-20-50-1B

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
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-Name Accounting Standards Codification
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-Paragraph 1B
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-URI https://asc.fasb.org//1943274/2147481139/470-20-50-1B

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
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Reference 7: http://www.xbrl.org/2003/role/disclosureRef
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Reference 8: http://www.xbrl.org/2003/role/disclosureRef
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Reference 9: http://www.xbrl.org/2003/role/disclosureRef
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Reference 10: http://www.xbrl.org/2003/role/disclosureRef
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Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
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-Subparagraph (a)
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Reference 12: http://www.xbrl.org/2003/role/disclosureRef
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Reference 13: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
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-URI https://asc.fasb.org//1943274/2147481139/470-20-50-1I

+ Details

Investments (Tables)

12 Months Ended

Dec. 31, 2023

Investments, All Other Investments [Abstract]

Schedule of Fair Value, Assets and Liabilities Measured on Recurring Basis

Assets measured at fair value on a recurring basis were as follows (in thousands):


	December 31, 2023
	Quoted Price in Active Markets (Level 1)		Significant Other Observable Inputs (Level 2)		Significant Unobservable Inputs (Level 3)		Total
Assets:
Obligations of U.S. Government and its agencies	$	—	$	277,358	$	—	$	277,358
Certificates of deposit	—		986		—		986
Total assets	$	—	$	278,344	$	—	$	278,344


	December 31, 2022
	Quoted Price in Active Markets (Level 1)		Significant Other Observable Inputs (Level 2)		Significant Unobservable Inputs (Level 3)		Total
Assets:
Obligations of U.S. Government and its agencies	$	—	$	129,371	$	—	$	129,371
Corporate debt securities	—		6,092		—		6,092
Certificates of deposit	—		2,157		—		2,157
Total assets	$	—	$	137,620	$	—	$	137,620

Fair Value of the Company's Investments by Type


	December 31, 2023
	Amortized Cost		Accrued Interest		Gross Unrealized Gains		Gross Unrealized Losses		Estimated Fair Value
Obligations of U.S. Government and its agencies	$	277,151	$	121	$	150	$	(64)	$	277,358
Certificates of deposit	980		14		—		(8)		986
Total investments	$	278,131	$	135	$	150	$	(72)	$	278,344


	December 31, 2022
	Amortized Cost		Accrued Interest		Gross Unrealized Gains		Gross Unrealized Losses		Estimated Fair Value
Obligations of U.S. Government and its agencies	$	129,940	$	427	$	—	$	(996)	$	129,371
Corporate debt securities	6,093		37		—		(38)		6,092
Certificates of deposit	2,163		23		—		(29)		2,157
Total investments	$	138,196	$	487	$	—	$	(1,063)	$	137,620

Schedule of the Maturity of Investments

The following table summarizes the scheduled maturity for the Company’s investments at December 31, 2023 and 2022 (in thousands):


	December 31,
	2023		2022
Maturing in one year or less	$	278,344	$	119,543
Maturing after one year through two years	—		18,077
Total investments	$	278,344	$	137,620

- Definition

Tabular disclosure of investment in debt security measured at fair value with change in fair value recognized in other comprehensive income (available-for-sale).

+ References

Reference 1: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 9
-Subparagraph (a)
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Reference 2: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
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Reference 3: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 4: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 5: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 6: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 8: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 9: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 10: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 12: http://www.xbrl.org/2009/role/commonPracticeRef
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+ Details

- Definition

Tabular disclosure of information about investment in debt security measured at amortized cost (held-to-maturity).

+ References

Reference 1: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 5
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481800/320-10-50-5

Reference 2: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 3: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 4: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 5: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 17: http://www.xbrl.org/2009/role/commonPracticeRef
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-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481800/320-10-50-5B

+ Details

Accrued Expenses - Accrued Expenses (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Accounts Payable and Accrued Liabilities [Abstract]
Compensation and benefits	$ 36,518	$ 22,125
Revenue-related reserves for discounts and allowances	26,509	14,332
Royalties payable	18,524	7,700
Development costs	13,677	30,360
Inventory	190	4,193
Other	7,464	8,855
Total accrued expenses	$ 102,882	$ 87,565

Income Taxes - Deferred Tax Assets and Liabilities (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Deferred tax assets:
Net federal and state operating losses	$ 108,753	$ 101,600
Research and development credits	88,390	86,321
Royalty income	122,391	115,554
Stock-based compensation	26,113	19,374
Capitalized R&D	75,081	62,794
Leasing obligations	3,076	1,842
Other	17,515	4,354
Total deferred tax assets	441,319	391,839
Deferred tax liabilities:
Fixed assets	(678)	(717)
Right of use asset	(2,872)	(1,525)
Total deferred tax liabilities	(3,550)	(2,242)
Valuation allowance	(437,098)	(389,608)
Net deferred tax assets (liabilities)	$ 671
Net deferred tax assets (liabilities)		$ (11)

Property and Equipment

12 Months Ended

Dec. 31, 2023

Property, Plant and Equipment [Abstract]

Property and Equipment

Property and equipment consisted of the following (in thousands):


	December 31,
	2023		2022
Furniture and fixtures	$	1,384	$	1,308
Office equipment	719		633
Software	1,252		1,501
Laboratory equipment	5,559		4,588
Leasehold improvements	10,206		10,137
Total property and equipment	$	19,120	$	18,167
Less accumulated depreciation and amortization	(11,210)		(9,550)
Property and equipment, net	$	7,910	$	8,617

Depreciation expense for the years ended December 31, 2023, 2022, and 2021 was $1,655, $1,437, and $777, respectively. The Company recorded an impairment loss of $1,548 and contract termination fees of $440 related to the discontinuation of the Birmingham research facilities expansion, which has been recognized in research and development expenses during the year ended December 31, 2023. The Company did not record any impairment losses during the years ended December 31, 2022 and 2021.

Stock-Based Compensation - Weighted Average Assumptions for Stock Option Awards Granted to Employees and Directors Under the Plans (Details) - $ / shares	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Incentive Plan
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Expected Life in Years	5 years 8 months 12 days	5 years 6 months	5 years 6 months
Expected Volatility	82.50%	84.20%	84.20%
Expected Dividend Yield	0.00%	0.00%	0.00%
Risk-Free Interest Rate	3.90%	3.60%	1.10%
Options, grants in period, weighted average grant date fair value (in dollars per share)	$ 4.75	$ 7.57	$ 7.93
Inducement Plan
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Expected Life in Years	5 years 7 months 6 days	5 years 6 months	5 years 6 months
Expected Volatility	83.50%	84.20%	84.20%
Expected Dividend Yield	0.00%	0.00%	0.00%
Risk-Free Interest Rate	4.00%	3.20%	0.90%
Options, grants in period, weighted average grant date fair value (in dollars per share)	$ 5.79	$ 9.54	$ 9.65

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References

+ Details

Subsequent Events	12 Months Ended
Subsequent Events	Dec. 31, 2023
Subsequent Events [Abstract]
Subsequent Events	Subsequent Events The Company considers events or transactions that occur after the balance sheet date but prior to the issuance of the consolidated financial statements to provide additional evidence for certain estimates or to identify matters that require additional disclosure. The Company has concluded that no subsequent events have occurred that require disclosure, except as described in “Note 16— Workforce Reduction”.

Royalty Financing Obligations - Schedule of Royalty Financing Obligations (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Royalties Monetizations [Line Items]
Balance	$ 501,655	$ 449,375	$ 124,717
Long-term debt, total			298,142
Non-cash Interest expense on Royalty financing obligations	70,356	76,482	37,670
Royalty revenues paid and payable	(40,412)	(24,168)	(11,154)
Deferred financing costs		(34)
Balance	531,599	501,655	449,375
2020 RPI Royalty Agreement
Royalties Monetizations [Line Items]
Balance	164,981	147,224	124,717
Non-cash Interest expense on Royalty financing obligations	38,267	39,994	33,308
Royalty revenues paid and payable	(28,768)	(22,237)	(10,801)
Balance	$ 174,480	$ 164,981	147,224
Effective interest rate	22.30%	22.40%
2021 RPI Royalty Agreement
Royalties Monetizations [Line Items]
Balance	$ 173,651	$ 153,377
Long-term debt, total			150,833
Non-cash Interest expense on Royalty financing obligations	14,188	22,239	2,897
Royalty revenues paid and payable	(2,494)	(1,931)	(353)
Deferred financing costs		(34)
Balance	$ 185,345	$ 173,651	153,377
Effective interest rate	0.00%	13.10%
OMERS Royalty Agreement
Royalties Monetizations [Line Items]
Balance	$ 163,023	$ 148,774
Long-term debt, total			147,309
Non-cash Interest expense on Royalty financing obligations	17,901	14,249	1,465
Royalty revenues paid and payable	(9,150)
Balance	$ 171,774	$ 163,023	$ 148,774
Effective interest rate	10.00%	10.60%

Lease Obligations (Tables)

12 Months Ended

Dec. 31, 2023

Leases [Abstract]

Lease, Cost

Lease expense under operating and finance leases was as follows (in thousands):


	Years Ended December 31,
	2023		2022		2021
Operating lease expense	$	2,018	$	1,578	$	1,660
Finance lease expense:
Amortization of right-of-use assets	$	1,212	$	816	113
Interest on lease liabilities	$	201	$	138	22
Total finance lease expense	$	1,413	$	954	$	135

Supplemental cash flow information related to leases was as follows (in thousands):


	Years Ended December 31,
	2023		2022		2021
Cash paid for amounts included in the measurement of lease liabilities:
Operating cash flows for finance leases	$	201	$	898	$	125
Operating cash flows for operating leases	$	1,920	$	1,555	$	1,490
Financing cash flows for finance leases	$	1,165	$	—	$	—

Operating lease assets obtained in exchange for operating lease liabilities:	$	4,695	$	755	$	1,536
Finance lease assets obtained in exchange for finance lease liabilities:	$	2,971	$	1,302	$	2,190
Non-cash increase to operating lease assets due to remeasurement of operating lease liabilities:	$	924	$	—	$	—

Other Supplemental Information Related to Leases

Other supplemental information related to leases was as follows:


	As of December 31,
	2023		2022
Weighted average remaining lease term:
Operating leases	9.7 years		10.6 years
Finance leases	3.0 years		3.0 years
Weighted average discount rate:
Operating leases	10.88	%	12.54	%
Finance leases	7.46	%	5.00	%

Lessee, Operating Lease, Assets and Liabilities

The following table summarizes the presentation in the Consolidated Balance Sheets of the Company’s operating leases (in thousands):


		As of December 31,
	Balance Sheet Location	2023		2022
Operating lease assets:
Operating lease assets, net	Other Assets	$	8,682	$	4,242
Operating lease liabilities:
Current operating lease liabilities	Operating lease liabilities – current liabilities	$	1,058	$	1,516
Non-current operating lease liabilities	Operating lease liabilities – long-term liabilities	8,390		4,027
Total operating lease liabilities		$	9,448	$	5,543

The following table summarizes the presentation in the Consolidated Balance Sheets of the Company’s finance leases (in thousands):


		As of December 31,
	Balance Sheet Location	2023		2022
Finance lease assets:
Finance lease assets, net	Other Assets	$	4,322	$	2,564
Finance lease liabilities:
Current finance lease liabilities	Finance lease liabilities – current liabilities	$	1,590	$	853
Non-current finance lease liabilities	Finance lease liabilities – long-term liabilities	2,845		1,777
Total finance lease liabilities		$	4,435	$	2,630

Maturities of Operating Lease Liabilities

Maturities of lease liabilities as of December 31, 2023 are as follows (in thousands):


	Operating Leases		Finance Leases
2024	$	2,014	$	1,857
2025	1,701		1,732
2026	1,509		899
2027	1,463		437
2028	1,312		27
Thereafter	7,952		—
Total lease payments	15,951		4,952
Less imputed interest	(6,503)		(517)
Total	$	9,448	$	4,435

Revenue - Summary of Revenues (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Disaggregation of Revenue [Line Items]
Revenues	$ 331,412	$ 270,827	$ 157,170
Revenues
Disaggregation of Revenue [Line Items]
Revenues	324,696	267,710	136,350
Revenues \| Geographic Distribution, Domestic
Disaggregation of Revenue [Line Items]
Revenues	288,361	226,358	117,836
Revenues \| Geographic Distribution, Foreign
Disaggregation of Revenue [Line Items]
Revenues	37,629	25,275	4,719
ORLADEYO
Disaggregation of Revenue [Line Items]
Revenues	325,990	251,633	122,555
Other revenues
Disaggregation of Revenue [Line Items]
Revenues	$ 5,422	$ 19,194	$ 34,615

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References + Details

- Definition

+ References

+ Details

Stockholders' Equity (Tables)

12 Months Ended

Dec. 31, 2023

Equity [Abstract]

Schedule of Stock by Class

The Company had reserved shares of common stock for issuance as follows (in thousands):


	December 31,
	2023	2022
Shares reserved for exercises of outstanding stock options	41,032	36,520
Shares reserved for vesting of restricted stock units	6,507	4,747
Shares reserved for exercises of warrants	—	15,023
Shares reserved for future issuance under the Stock Incentive Plan	3,376	4,206
Shares reserved for future issuance under the Inducement Equity Incentive Plan	1,651	947
Shares reserved for future issuance under the Employee Stock Purchase Plan	5,454	5,792
Total shares reserved for future issuance	58,020	67,235

- Definition

Tabular disclosure of an entity's stock, including par or stated value per share, number and dollar amount of share subscriptions, shares authorized, shares issued, shares outstanding, number and dollar amount of shares held in an employee trust, dividend per share, total dividends, share conversion features, par value plus additional paid in capital, the value of treasury stock and other information necessary to a fair presentation, and EPS information. Stock by class includes common, convertible, and preferred stocks which are not redeemable or redeemable solely at the option of the issuer. Includes preferred stock with redemption features that are solely within the control of the issuer and mandatorily redeemable stock if redemption is required to occur only upon liquidation or termination of the reporting entity. If more than one issue is outstanding, state the title of each issue and the corresponding dollar amount; dollar amount of any shares subscribed but unissued and the deduction of subscriptions receivable there from; number of shares authorized, issued, and outstanding.

+ References

Reference 1: http://fasb.org/us-gaap/role/ref/legacyRef
-Name Accounting Standards Codification
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Reference 2: http://fasb.org/us-gaap/role/ref/legacyRef
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Reference 4: http://fasb.org/us-gaap/role/ref/legacyRef
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Reference 5: http://fasb.org/us-gaap/role/ref/legacyRef
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Reference 9: http://fasb.org/us-gaap/role/ref/legacyRef
-Name Accounting Standards Codification
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Reference 11: http://fasb.org/us-gaap/role/ref/legacyRef
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Reference 13: http://fasb.org/us-gaap/role/ref/legacyRef
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+ Details

Stock-Based Compensation - Restricted Stock Unit Activity (Details) - Restricted Stock Units (RSUs) - $ / shares shares in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Incentive Plan
Share-Based Compensation Arrangement by Share-Based Payment Award, Equity Instruments Other than Options, Nonvested, Number of Shares [Roll Forward]
Unvested RSU beginning balance (in shares)	4,159
Issued RSU (in shares)	3,106
Vested RSU (in shares)	(1,137)
Forfeited RSU (in shares)	(536)
Unvested RSU ending balance (in shares)	5,592	4,159
Share-Based Compensation Arrangement by Share-Based Payment Award, Equity Instruments Other than Options, Nonvested, Weighted Average Grant Date Fair Value [Abstract]
Nonvested RSU weighted average grant date fair value beginning balance (in dollars per share)	$ 10.69
Restricted stock units, grants in period, weighted average grant date fair value (in dollars per share)	6.71	$ 11.20	$ 11.36
Restricted stock units, vested in period, weighted average grant date fair value (in dollars per share)	11.04
Restricted stock units, forfeited in period, weighted average grant date fair value (in dollars per share)	7.91
Nonvested RSU weighted average grant date fair value ending balance (in dollars per share)	$ 8.67	$ 10.69
Inducement Plan
Share-Based Compensation Arrangement by Share-Based Payment Award, Equity Instruments Other than Options, Nonvested, Number of Shares [Roll Forward]
Unvested RSU beginning balance (in shares)	588
Issued RSU (in shares)	581
Vested RSU (in shares)	(139)
Forfeited RSU (in shares)	(115)
Unvested RSU ending balance (in shares)	915	588
Share-Based Compensation Arrangement by Share-Based Payment Award, Equity Instruments Other than Options, Nonvested, Weighted Average Grant Date Fair Value [Abstract]
Nonvested RSU weighted average grant date fair value beginning balance (in dollars per share)	$ 13.14
Restricted stock units, grants in period, weighted average grant date fair value (in dollars per share)	7.81
Restricted stock units, vested in period, weighted average grant date fair value (in dollars per share)	13.13
Restricted stock units, forfeited in period, weighted average grant date fair value (in dollars per share)	11.98
Nonvested RSU weighted average grant date fair value ending balance (in dollars per share)	$ 9.90	$ 13.14

Collaborative and Other Relationships Textual (Details)					1 Months Ended		12 Months Ended
Collaborative and Other Relationships Textual (Details)	Nov. 03, 2023 USD ($) payment	Sep. 15, 2021 USD ($)	Nov. 05, 2019 USD ($)	Sep. 30, 2013 USD ($)	Aug. 31, 2020 USD ($)	Sep. 30, 2018 USD ($) dose	Dec. 31, 2022 USD ($) dose	Dec. 31, 2021 USD ($) dose	Dec. 31, 2019 USD ($) dose	Nov. 30, 2023	Mar. 31, 2015 USD ($)
U.S. Department of Health and Human Services, net
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Collaborative agreement contract value						$ 34,660,000
Contract term (year)						5 years
U.S. Department of Health and Human Services, net \| RAPIVAB
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Maximum number of products, doses \| dose						50,000
Number of product delivered, doses \| dose							49,980		20,000
Proceeds from collaborators							$ 13,864,000	$ 6,918,000	$ 13,864,000
Number of additional products, doses \| dose							20,000	9,980
Torii Pharmaceutical Co
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Contract with customer, liability			$ 22,000,000
Potential milestone payments receivable if regulatory approval before december 31, 2021			$ 15,000,000
Maximum customary reduction on royalty rate			50.00%
Royalty payments receivable, expiration term from first commercial (year)			10 years
Torii Pharmaceutical Co \| Minimum
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Royalty rate if maintains sakigake designation			20.00%							20.00%
Torii Pharmaceutical Co \| Maximum
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Royalty rate if maintains sakigake designation			40.00%							80.00%
Clearside Biomedical, Inc.
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
License agreement, upfront payment	$ 5,000,000
Number of milestone payments \| payment	3
Royalty payment, number of tiers \| payment	3
Clearside Biomedical, Inc. \| Annual Net Sales Of $2 Million
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Annual global net sales	$ 2,000,000,000
Clearside Biomedical, Inc. \| Annual Net Sales Greater Than $1.5 Million
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Annual global net sales	1,500,000,000
Clearside Biomedical, Inc. \| Maximum
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Potential milestone payments payable	30,000,000
Potential milestone payments payable if regulatory approval before milestone	$ 47,500,000
Base Contract
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Government contract receivable											$ 16,265,000
Additional Development Options
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Government contract receivable											22,855,000
ASPRBARDA Contract
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Government contract receivable											$ 39,120,000
Proceeds from awards for research and development contracts							$ 20,574,000
National Institute of Allergy and Infectious Diseases
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Receivable from awards for research and development contracts		$ 47,315,000		$ 45,931,000
Potential aggregate maximum amount of funding					$ 43,908,000
Collaborative agreement contract value					$ 6,326,000

Lease Obligations - Maturities of Operating Lease Liabilities (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Lessee, Operating Lease, Liability, to be Paid, Fiscal Year Maturity [Abstract]
2024	$ 2,014
2025	1,701
2026	1,509
2027	1,463
2028	1,312
Thereafter	7,952
Total lease payments	15,951
Less imputed interest	(6,503)
Total	9,448	$ 5,543
Finance Lease, Liability, to be Paid, Fiscal Year Maturity [Abstract]
2024	1,857
2025	1,732
2026	899
2027	437
2028	27
Thereafter	0
Total lease payments	4,952
Less imputed interest	(517)
Total	$ 4,435	$ 2,630

Trade Receivables

12 Months Ended

Dec. 31, 2023

Receivables [Abstract]

Trade Receivables

Product Sales

Receivables from product sales are recorded for amounts due to the Company related to sales of ORLADEYO and RAPIVAB. At December 31, 2023 and 2022, receivables, net of reserves, related to sales of ORLADEYO were $54,149 and $41,508, respectively. A returns reserve related to sales of ORLADEYO of $288 was recorded as of December 31, 2023. No reserve or allowance amounts were recorded as of December 31, 2022 related to sales of ORLADEYO. At December 31, 2023 and 2022, receivables related to sales of RAPIVAB were $505 and $823, respectively. No reserve or allowance amounts were recorded as of December 31, 2023 and 2022, related to sales of RAPIVAB.

Collaborations

Receivables from collaborations were as follows (in thousands):


	December 31, 2023
	Billed		Unbilled		Total
Royalty receivables from partners	$	—	$	2,296	$	2,296
Total receivables from collaborators	$	—	$	2,296	$	2,296


	December 31, 2022
	Billed		Unbilled		Total
U.S. Department of Health and Human Services, net	$	7,218	$	284	$	7,502
Royalty receivables from partners	741		—		741
Other collaborations	—		25		25
Total receivables from collaborators	$	7,959	$	309	$	8,268

As of December 31, 2023 and 2022, the reserve related to royalties from collaborations was not material.

Stock-Based Compensation

12 Months Ended

Dec. 31, 2023

Share-Based Payment Arrangement [Abstract]

Stock-Based Compensation

Note 12— Stock-Based Compensation

As of December 31, 2023, the Company had three stock-based employee compensation plans: the Amended and Restated Stock Incentive Plan (“Incentive Plan”), the Amended and Restated Inducement Equity Incentive Plan (“Inducement Plan”) and the Amended and Restated Employee Stock Purchase Plan (“ESPP”). The Incentive Plan was most recently amended and restated on April 24, 2023 and approved by the Company’s stockholders on June 13, 2023. The Inducement Plan was most recently amended and restated by the Company’s Board of Directors on October 26, 2023. The ESPP was most recently amended and restated by the Company’s Board of Directors on July 7, 2023.

The Company recorded the following stock-based compensation expense (in thousands):


	Years Ended December 31,
	2023		2022		2021
Incentive Plan	$	44,581	$	36,716	$	27,062
Inducement Plan	9,958		6,550		6,055
ESPP	1,076		1,435		1,523
Stock-based compensation expense	$	55,615	$	44,701	$	34,640

Total stock-based compensation was allocated as follows:


	Years Ended December 31,
	2023		2022		2021
Research and development	$	29,377	$	24,936	$	20,179
Selling, general and administrative	26,238		19,765		14,461
Total stock-based compensation expense	$	55,615	$	44,701	$	34,640

Stock Incentive Plan

The Company grants stock option awards, restricted stock and restricted stock units to its employees, directors, and consultants under the Incentive Plan. Under the Incentive Plan, stock option awards are granted with an exercise price equal to the market price of the Company’s common stock at the date of grant. Stock option awards and restricted stock units granted to employees generally vest 25% each year until fully vested after four years.

In December 2014, the Company issued 1,250 performance-based stock options. These awards vest upon successful completion of specific development milestones. As of December 31, 2023, 85% of these grants have vested.

In January 2022, the Company issued 221 performance-based restricted stock unit awards. 21 of the awards met the performance objectives in 2022 and became eligible for vesting at 50% on the first anniversary of the grant date and 25% on each of the second and third anniversaries of the grant date, until fully vested after three years. The Company recognized $34 and $158 of stock compensation expense related to these awards during the years ended December 31, 2023 and December 31, 2022, respectively.

Stock option awards and restricted stock unit awards granted to non-employee directors of the Company generally vest over one year. Stock option awards granted to new non-employee directors when they first join the Company’s Board of Directors generally vest, subject to the terms of the Incentive Plan, in 36 equal monthly installments over a three-year period measured from the grant date. All stock option awards have contractual terms of 10 years. Restricted stock unit awards granted to new non-employee directors when they first join the Company’s Board of Directors generally vest, subject to the terms of the Incentive Plan, in three equal annual installments beginning on the first anniversary of the grant date. The vesting and exercise provisions of all awards granted under the Incentive Plan are subject to acceleration in the event of certain stockholder-approved transactions, or upon the occurrence of a change in control as defined in the Incentive Plan.

The following table summarizes stock option activity under the Incentive Plan:


	Shares (in thousands)	Weighted Average Exercise Price per Share		Weighted Average Remaining Contractual Term (in years)	Aggregate Intrinsic Value (in thousands)
Outstanding at December 31, 2022	31,179	$	8.56
Granted	6,625	6.67
Exercised	(935)	5.21			$	3,601
Cancelled or Forfeited	(1,364)	10.00
Outstanding at December 31, 2023	35,505	$	8.24	6.85	$	12,280
Exercisable at December 31, 2023	21,008	$	7.87	5.44	$	12,280
Vested and expected to vest at December 31, 2023	33,557	$	8.19	6.77	$	12,280

The total intrinsic value of stock option awards exercised under the Incentive Plan was $21,150 and $25,484 during the years ended December 31, 2022 and 2021, respectively. The aggregate intrinsic value represents the total proceeds (calculated as the difference between the exercise price of the underlying stock options and the estimated fair value of the Company’s common stock on the date of exercise for those stock options that had exercise prices lower than the fair value of the Company’s common stock on the exercise date) received by all individuals who exercised stock option awards during the period.

The following table summarizes restricted stock unit activity under the Incentive Plan:


	Shares (in thousands)	Weighted Average Grant Date Fair Value
Unvested at December 31, 2022	4,159	$	10.69
Granted	3,106	6.71
Vested	(1,137)	11.04
Forfeited	(536)	7.91
Unvested at December 31, 2023	5,592	$	8.67

For restricted stock unit awards granted under the Incentive Plan, the fair value of the awards is determined based on the market value of the Company’s shares on the grant date. The weighted average grant date fair value of these awards granted during 2023, 2022, and 2021 was $6.71, $11.20, and 11.36, respectively. The fair value of the restricted stock unit awards is amortized to expense over the vesting periods using a straight-line expense attribution method.

As of December 31, 2023, total unrecognized compensation cost related to unvested restricted stock unit awards granted under the Incentive Plan was $40,981, which is expected to be recognized over a weighted average period of 2.0 years.

Inducement Equity Incentive Plan

The Company has the ability to grant stock option and restricted stock unit awards to newly-hired employees as inducements material to each employee entering employment with the Company. Awards granted to newly hired employees generally vest 25% each year until fully vested after four years and are subject to the terms and conditions of the Inducement Plan. Each stock option has a term of 10 years. The vesting and exercise provisions of all awards granted under the Inducement Plan are subject to acceleration in the event of certain stockholder-approved transactions, or upon the occurrence of a change in control as defined in the Inducement Plan.

The following table summarizes stock option activity under the Inducement Plan:


	Shares (in thousands)	Weighted Average Exercise Price per Share		Weighted Average Remaining Contractual Term (in years)	Aggregate Intrinsic Value (in thousands)
Outstanding at December 31, 2022	5,341	$	8.80
Granted	1,196	8.15
Exercised	(344)	3.70			$	1,803
Cancelled or Forfeited	(666)	10.47
Outstanding at December 31, 2023	5,527	$	8.77	7.76	$	4,463
Exercisable at December 31, 2023	2,448	$	7.17	6.89	$	3,434
Vested and expected to vest at December 31, 2023	5,122	$	8.68	7.72	$	4,291

The total intrinsic value of stock option awards exercised under the Inducement Plan was $3,710 and $6,700 during the years ended December 31, 2022 and 2021, respectively. The aggregate intrinsic value represents the total proceeds (calculated as the difference between the exercise price of the underlying stock options and the estimated fair value of the Company’s common stock on the date of exercise for those stock options that had exercise prices lower than the fair value of the Company’s common stock on the exercise date) received by all individuals who exercised stock option awards during the period.

The following table summarizes restricted stock unit activity under the Inducement Plan:


	Shares (in thousands)	Weighted Average Grant Date Fair Value
Unvested at December 31, 2022	588	$	13.14
Granted	581	7.81
Vested	(139)	13.13
Forfeited	(115)	11.98
Unvested at December 31, 2023	915	$	9.90

For restricted stock unit awards granted under the Inducement Plan, the fair value of the awards is determined based on the market value of the Company’s shares on the grant date. The weighted average grant date fair value of these awards granted during 2023 and 2022 was $7.81 and 13.21, respectively. The fair value of the restricted stock unit awards is amortized to expense over the vesting periods using a straight-line expense attribution method. No restricted stock unit awards were granted under the Inducement Plan during 2021.

As of December 31, 2023, total unrecognized compensation cost related to unvested restricted stock unit awards granted under the Inducement Plan was $7,044, which is expected to be recognized over a weighted average period of 1.8 years.

Weighted Average Assumptions for Stock Option Awards Granted to Employees and Directors under the Incentive and Inducement Plans

For stock option awards granted under the Incentive Plan and the Inducement Plan, the fair value is estimated on the date of grant using a Black-Scholes option pricing model and the assumptions noted below. The fair value of the stock option awards is amortized to expense over the vesting periods using a straight-line expense attribution method.

Historically, the expected life was based on the average of the assumption that all outstanding stock option awards will be exercised at full vesting and the assumption that all outstanding stock option awards will be exercised at the midpoint of the current date (if already vested) or at full vesting (if not yet vested) and the full contractual term. Effective July 1, 2023, the expected life is based on the historical settlement of options by taking into account exercises and post-

vesting terminations and weighing them based on the number of options settled. This change in approach did not have a significant impact on the value of the stock option awards granted. The expected volatility represents the historical volatility on the Company’s publicly-traded common stock. The Company has assumed no expected dividend yield, as dividends have never been paid to stock or option holders and will not be paid for the foreseeable future. The weighted average risk-free interest rate is the implied yield currently available on zero-coupon government issues with a remaining term equal to the expected term.

Stock Incentive Plan

The following table summarizes the key assumptions used by the Company to value the stock option awards granted under the Incentive Plan during the years ended December 31, 2023, 2022, and 2021:


	Years Ended December 31,
	2023			2022			2021
Expected Life in Years	5.7			5.5			5.5
Expected Volatility	82.5		%	84.2		%	84.2		%
Expected Dividend Yield	0.0		%	0.0		%	0.0		%
Risk-Free Interest Rate	3.9		%	3.6		%	1.1		%
Weighted average grant date fair value per share	$	4.75		$	7.57		$	7.93

The total fair value of the stock option awards vested under the Incentive Plan was $33,731, $28,916, and $20,510 during the years ended December 31, 2023, 2022, and 2021, respectively. As of December 31, 2023, total unrecognized compensation cost related to unvested stock option awards granted under the Incentive Plan was $75,164, which is expected to be recognized over a weighted average period of 1.9 years.

Inducement Equity Incentive Plan

The following table summarizes the key assumptions used by the Company to value the stock option awards granted under the Inducement Plan during the years ended December 31, 2023, 2022, and 2021:


	Years Ended December 31,
	2023			2022			2021
Expected Life in Years	5.6			5.5			5.5
Expected Volatility	83.5		%	84.2		%	84.2		%
Expected Dividend Yield	0.0		%	0.0		%	0.0		%
Risk-Free Interest Rate	4.0		%	3.2		%	0.9		%
Weighted average grant date fair value per share	$	5.79		$	9.54		$	9.65

The total fair value of the stock option awards vested under the Inducement Plan was $7,698, $4,659, and $2,885 during the years ended December 31, 2023, 2022, and 2021, respectively. As of December 31, 2023, total unrecognized compensation cost related to unvested stock option awards granted under the Inducement Plan was $16,216, which is expected to be recognized over a weighted average period of 1.5 years.

Employee Stock Purchase Plan

The Company has reserved a total of 7,975 shares of common stock to be purchased under the ESPP, of which 5,454 shares remain available for purchase at December 31, 2023. Eligible employees may authorize up to 15% of their salary to purchase common stock at the lower of 85% of the beginning or 85% of the ending price during six-month purchase intervals. No more than three thousand shares may be purchased by any one employee at each purchase date, and no employee may purchase stock having a fair market value at the commencement date of $25 or more in any one calendar year.

During the years ended December 31, 2023, 2022, and 2021, the Company issued 338, 260, and 321 shares of common stock under the ESPP, respectively, at a weighted average price per share of $7.68, $11.03, and $6.20,

respectively. Compensation expense for shares purchased under the ESPP related to the purchase discount and the “look-back” option were determined using a Black-Scholes option pricing model. The weighted average grant date fair values of shares granted under the ESPP during the years ended December 31, 2023, 2022, and 2021, were $3.82, $6.02, and $2.80, respectively.

Employee 401(k) Plan	12 Months Ended
Employee 401(k) Plan	Dec. 31, 2023
Retirement Benefits [Abstract]
Employee 401(k) Plan	Employee 401(k) Plan In January 1991, the Company adopted an employee retirement plan (“401(k) Plan”) under Section 401(k) of the IRC covering all employees. Employee contributions may be made to the 401(k) Plan up to limits established by the Internal Revenue Service. Company matching contributions may be made at the discretion of the Board of Directors. The Company made matching contributions of $5,716, $3,758, and $2,834 in 2023, 2022, and 2021, respectively.

- Definition

The entire disclosure for retirement benefits.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 715
-SubTopic 70
-Name Accounting Standards Codification
-Section 50
-Paragraph 1
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480794/715-70-50-1

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-Topic 715
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+ Details

Stock-Based Compensation - Stock-based Compensation Allocation (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Total stock-based compensation expense	$ 55,615	$ 44,701	$ 34,640
Research and development
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Total stock-based compensation expense	29,377	24,936	20,179
Selling, general and administrative
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Total stock-based compensation expense	$ 26,238	$ 19,765	$ 14,461

- Definition

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

+ References

+ Details

Income Taxes - Differences Between the Company's Effective Tax Rate and the Statutory Tax Rate (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Income Tax Disclosure [Abstract]
Income tax benefit at federal statutory rate (21% for 2023, 2022 and 2021)	$ (47,328)	$ (51,321)	$ (38,175)
State and local income taxes net of federal tax benefit	(3,477)	(1,816)	(2,288)
Permanent items	3,015	(1,608)	(1,343)
Expiration of attribute carryforwards	0	0	(1,057)
Research and development tax credits	(3,301)	(9,793)	(5,994)
Foreign rate differential	2,255	1,862	1,940
Other	1,656	(5,485)	1,216
Change in valuation allowance	47,490	70,894	47,954
Income tax expense	$ 310	$ 2,733	$ 2,253

Significant Accounting Policies and Concentrations of Risk - Summary of Revenues (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Significant Accounting Policies and Concentrations of Risk [Line Items]
Revenues	$ 331,412	$ 270,827	$ 157,170
Revenues
Significant Accounting Policies and Concentrations of Risk [Line Items]
Revenues	324,696	267,710	136,350
Collaborative and other revenues
Significant Accounting Policies and Concentrations of Risk [Line Items]
Revenues	$ 6,716	$ 3,117	$ 20,820

- Definition

+ References

+ Details

Revenue

12 Months Ended

Dec. 31, 2023

Revenue Recognition [Abstract]

Revenue

The Company recorded the following revenues for the years ended December 31 (in thousands):


	Years Ended December 31,
	2023		2022		2021
ORLADEYO:
U.S.	$	288,361	$	226,358	$	117,836
Outside of U.S.	37,629		25,275		4,719
Total ORLADEYO	325,990		251,633		122,555
Other revenues	5,422		19,194		34,615
Total revenues	$	331,412	$	270,827	$	157,170

ORLADEYO revenues represent total revenues from product sales, collaborative revenues and royalties. Royalty revenue from sales of ORLADEYO in Japan by the Company’s collaborative partner, Torii, were $2,178, $1,944, and $690 for the years ended December 31, 2023, 2022, and 2021 respectively.

Other revenues primarily relate to the Company’s product sales and royalties for peramivir injection (RAPIVAB/RAPIACTA/PERAMIFLU), and for the years ended December 31, 2022 and 2021, galidesivir development contracts with BARDA/HHS and NIAID/HHS. Other revenues for the year ended December 31, 2021 also includes milestone revenue of $15,000 upon receipt from the Japanese National Health Insurance System (“NHI”) of a reimbursement price approval for ORLADEYO.

- Definition

The entire disclosure of revenue from contract with customer to transfer good or service and to transfer nonfinancial asset. Includes, but is not limited to, disaggregation of revenue, credit loss recognized from contract with customer, judgment and change in judgment related to contract with customer, and asset recognized from cost incurred to obtain or fulfill contract with customer. Excludes insurance and lease contracts.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 9
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-9

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
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Reference 3: http://www.xbrl.org/2003/role/disclosureRef
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Reference 4: http://www.xbrl.org/2003/role/disclosureRef
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-URI https://asc.fasb.org//1943274/2147479806/606-10-50-12

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
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-Topic 606
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Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Name Accounting Standards Codification
-Topic 606
-Publisher FASB
-URI https://asc.fasb.org//606/tableOfContent

+ Details

Trade Receivables - Summary of Receivables (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Accounts, Notes, Loans and Financing Receivable [Line Items]
Receivables	$ 2,296	$ 8,268
Billed
Accounts, Notes, Loans and Financing Receivable [Line Items]
Receivables	0	7,959
Unbilled
Accounts, Notes, Loans and Financing Receivable [Line Items]
Receivables	2,296	309
U.S. Department of Health and Human Services, net
Accounts, Notes, Loans and Financing Receivable [Line Items]
Receivables		7,502
U.S. Department of Health and Human Services, net \| Billed
Accounts, Notes, Loans and Financing Receivable [Line Items]
Receivables		7,218
U.S. Department of Health and Human Services, net \| Unbilled
Accounts, Notes, Loans and Financing Receivable [Line Items]
Receivables		284
Royalty receivables from partners
Accounts, Notes, Loans and Financing Receivable [Line Items]
Receivables	2,296	741
Royalty receivables from partners \| Billed
Accounts, Notes, Loans and Financing Receivable [Line Items]
Receivables	0	741
Royalty receivables from partners \| Unbilled
Accounts, Notes, Loans and Financing Receivable [Line Items]
Receivables	$ 2,296	0
Other collaborations
Accounts, Notes, Loans and Financing Receivable [Line Items]
Receivables		25
Other collaborations \| Billed
Accounts, Notes, Loans and Financing Receivable [Line Items]
Receivables		0
Other collaborations \| Unbilled
Accounts, Notes, Loans and Financing Receivable [Line Items]
Receivables		$ 25

Inventory (Tables)

12 Months Ended

Dec. 31, 2023

Inventory Disclosure [Abstract]

Schedule of Inventories

The Company’s inventories consisted of the following (in thousands):


	December 31,
	2023		2022
Raw materials	$	6,449	$	8,906
Work-in-process	17,591		14,990
Finished goods	6,242		4,814
Total inventory	$	30,282	$	28,710
Reserves	(1,599)		(1,177)
Total inventory, net	$	28,683	$	27,533

Lease Obligations (Details Textual) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Lessee, Lease, Description [Line Items]
Accumulated amortization	$ 4,794	$ 3,268
Accumulated amortization	2,293	1,081
Operating cash flows for operating leases	1,920	1,555	$ 1,490
Principal payments on finance lease liabilities	$ 1,165	$ 0	$ 0
Minimum
Lessee, Lease, Description [Line Items]
Lessee, operating lease, term of contract (in years)	1 year
Maximum
Lessee, Lease, Description [Line Items]
Lessee, operating lease, term of contract (in years)	3 years

Stockholders' Equity

12 Months Ended

Dec. 31, 2023

Equity [Abstract]

Stockholders' Equity

Stockholders’ Equity

Sales of Common Stock

On March 1, 2021, the Company filed an automatic shelf registration statement on Form S-3 with the SEC. This shelf registration statement became effective automatically upon filing and allows the Company to sell an indeterminate number of securities, including common stock, preferred stock, depositary shares, purchase contracts, warrants, debt securities, and units, from time to time at prices and on terms to be determined at the time of sale.

On November 19, 2021, concurrent with the Company entering into the 2021 RPI Royalty Purchase Agreement, the Company and RPI entered into the Common Stock Purchase Agreement, pursuant to which the Company issued 3,846 shares of the Company’s common stock to RPI for an aggregate purchase price of $50,000, at a price of $13.00 per share, calculated based on the 20-day volume weighted average price. The $13.00 per share price represented a premium of $1.11 over the closing price of $11.89 of the Company’s common stock on November 19, 2021, the last trading day prior to the execution of the Common Stock Purchase Agreement. The premium of $4,269 paid by RPI on the purchase of the Company’s common stock has been deferred and is being amortized as a component of interest expense of the 2021 RPI royalty financing obligation.

On October 23, 2023, certain entities affiliated with Baker Bros. Advisors LP (the “Baker Entities”) net exercised the remaining balance of the pre-funded warrants held by such Baker Entities that were issued on November 21, 2019. Additionally, certain of the Baker Entities net exercised all of the pre-funded warrants that were issued on June 1, 2020. The exercises resulted in the issuance of 14,997 common shares. Following the exercises, there are no outstanding warrants.

Shares Reserved for Future Issuance of Common Stock

The Company had reserved shares of common stock for issuance as follows (in thousands):


	December 31,
	2023	2022
Shares reserved for exercises of outstanding stock options	41,032	36,520
Shares reserved for vesting of restricted stock units	6,507	4,747
Shares reserved for exercises of warrants	—	15,023
Shares reserved for future issuance under the Stock Incentive Plan	3,376	4,206
Shares reserved for future issuance under the Inducement Equity Incentive Plan	1,651	947
Shares reserved for future issuance under the Employee Stock Purchase Plan	5,454	5,792
Total shares reserved for future issuance	58,020	67,235

- Definition

The entire disclosure for equity.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 13
-Subparagraph (b)
-Publisher FASB
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Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
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Reference 3: http://www.xbrl.org/2003/role/disclosureRef
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-URI https://asc.fasb.org//1943274/2147481112/505-10-50-14

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-Name Accounting Standards Codification
-Section 50
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-URI https://asc.fasb.org//1943274/2147481062/946-235-50-2

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
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-Name Accounting Standards Codification
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Reference 7: http://www.xbrl.org/2003/role/disclosureRef
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Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
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Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Topic 235
-SubTopic 10
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-Section S99
-Paragraph 1
-Subparagraph (SX 210.4-08(e)(1))
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480678/235-10-S99-1

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-Topic 505
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-URI https://asc.fasb.org//505/tableOfContent

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
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Reference 12: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
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-Section 50
-Paragraph 13
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-Publisher FASB
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Reference 13: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
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-Section 50
-Paragraph 13
-Subparagraph (i)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481112/505-10-50-13

Reference 14: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
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-Name Accounting Standards Codification
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-Topic 505
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-Name Accounting Standards Codification
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Reference 17: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
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Reference 18: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
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Reference 19: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
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-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481112/505-10-50-18

+ Details

Trade Receivables (Tables)

12 Months Ended

Dec. 31, 2023

Receivables [Abstract]

Summary of Receivables

Receivables from collaborations were as follows (in thousands):


	December 31, 2023
	Billed		Unbilled		Total
Royalty receivables from partners	$	—	$	2,296	$	2,296
Total receivables from collaborators	$	—	$	2,296	$	2,296


	December 31, 2022
	Billed		Unbilled		Total
U.S. Department of Health and Human Services, net	$	7,218	$	284	$	7,502
Royalty receivables from partners	741		—		741
Other collaborations	—		25		25
Total receivables from collaborators	$	7,959	$	309	$	8,268