0001628280-23-037847 | 10-Q | XBRL Viewer | Biocryst Pharmaceuticals INC

Investments

9 Months Ended

Sep. 30, 2023

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):


	September 30, 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	$	—	$	243,638	$	—	$	243,638
Corporate debt securities	—		2,074		—		2,074
Certificates of deposit	—		973		—		973
Total assets	$	—	$	246,685	$	—	$	246,685


	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 three and nine months ended September 30, 2023 and 2022. There were no liabilities measured at fair value on a recurring basis as of September 30, 2023 and December 31, 2022.

As of September 30, 2023, the Company had 24 securities with a total estimated fair market value of $215,095 in an unrealized loss position. The Company anticipated a full recovery of the amortized cost basis of its debt securities at maturity and an allowance was not recognized.

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


	September 30, 2023
	Amortized Cost		Accrued Interest		Gross Unrealized Gains		Gross Unrealized Losses		Estimated Fair Value
Obligations of U.S. Government and its agencies	$	243,810	$	37	$	3	$	(212)	$	243,638
Corporate debt securities	2,068		6		—		—		2,074
Certificates of deposit	980		6		—		(13)		973
Total investments	$	246,858	$	49	$	3	$	(225)	$	246,685


	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 September 30, 2023 and December 31, 2022 (in thousands):


	September 30, 2023		December 31, 2022
Maturing in one year or less	$	246,685	$	119,543
Maturing after one year through two years	—		18,077
Total investments	$	246,685	$	137,620

Subsequent Event (Details)			3 Months Ended
Subsequent Event (Details)	Nov. 03, 2023 USD ($) payment	Oct. 23, 2023 shares	Jun. 30, 2022 shares
Subsequent Event [Line Items]
Exercise of warrants (in shares) \| shares			253,000
Subsequent Event \| Clearside Biomedical, Inc.
Subsequent Event [Line Items]
Upfront payment	$ 5,000,000
Number of milestone payments \| payment	3
Number of royalties tiers \| payment	3
Subsequent Event \| Clearside Biomedical, Inc. \| Maximum
Subsequent Event [Line Items]
Potential milestone payment	$ 30,000,000
Potential payment if milestone reached	47,500,000
Subsequent Event \| Clearside Biomedical, Inc. \| Annual Net Sales Of $2 Million
Subsequent Event [Line Items]
Annual global net sales	2,000,000,000
Subsequent Event \| Clearside Biomedical, Inc. \| Annual Net Sales Greater Than $1.5 Million
Subsequent Event [Line Items]
Annual global net sales	$ 1,500,000,000
Subsequent Event \| Common Stock
Subsequent Event [Line Items]
Exercise of warrants (in shares) \| shares		14,997,000
Subsequent Event \| Warrants to purchase common stock
Subsequent Event [Line Items]
Warrants outstanding (in shares) \| shares		0

Lease Obligations - Aggregate Lease Expense Under Operating Leases (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
Leases [Abstract]
Aggregate lease expense	$ 1,014	$ 634	$ 2,505	$ 1,854

Investments - Narrative (Details)	Sep. 30, 2023 USD ($) securities
Investments, All Other Investments [Abstract]
Number of securities \| securities	24
Estimated fair market value of securities \| $	$ 215,095

Inventory

9 Months Ended

Sep. 30, 2023

Inventory Disclosure [Abstract]

Inventory

At September 30, 2023 and December 31, 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):


	September 30, 2023		December 31, 2022
Raw materials	$	6,656	$	8,906
Work-in-process	17,739		14,990
Finished goods	6,582		4,814
Total inventory	$	30,977	$	28,710
Reserves	(1,347)		(1,177)
Total inventory, net	$	29,630	$	27,533

Stock-based Compensation - Weighted Average Assumptions for Stock Option Awards Granted to Employees and Directors Under the Plans (Details) - Incentive Plan and Inducement Plan	9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Expected Life in Years	5 years 6 months	5 years 6 months
Expected Volatility	83.90%	84.10%
Expected Dividend Yield	0.00%	0.00%
Risk-Free Interest Rate	3.90%	2.90%

- 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.

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+ Details

Revenue

9 Months Ended

Sep. 30, 2023

Revenue from Contract with Customer [Abstract]

Revenue

The Company recorded the following revenues (in thousands):


	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
ORLADEYO:
U.S.	$	75,268	$	60,106	$	208,934	$	162,743
Outside of U.S.	10,416		5,866		26,173		18,155
Total ORLADEYO	85,684		65,972		235,107		180,898
Other revenues	1,058		9,855		2,904		10,384
Total revenues	$	86,742	$	75,827	$	238,011	$	191,282

ORLADEYO revenues represent total revenues from product sales, collaborative revenues and royalties. Other revenues primarily relate to the Company’s product sales and royalties for peramivir injection (RAPIVAB/RAPIACTA/PERAMIFLU) and galidesivir development contracts with BARDA/HHS and NIAID/HHS.

- 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.

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+ Details

Investments - Schedule of Maturity of Investments (Details) - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Investments [Abstract]
Maturing in one year or less	$ 246,685	$ 119,543
Maturing after one year through two years	0	18,077
Estimated Fair Value	$ 246,685	$ 137,620

Condensed Consolidated Statements of Stockholders' Deficit - USD ($) $ in Thousands	Total	Common Stock	Additional Paid-In Capital	Accumulated Other Comprehensive Income	Accumulated Deficit
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	(74,196)				(74,196)
Other comprehensive income (loss)	9			9
Employee stock purchase plan sales, net	1,504	1	1,503
Exercise of stock options, net	5,853	12	5,841
Stock-based compensation expense	9,601		9,601
Balance at Mar. 31, 2022	(164,215)	1,856	1,115,443	186	(1,281,700)
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	(175,575)
Balance at Sep. 30, 2022	(242,659)	1,864	1,138,654	(98)	(1,383,079)
Balance at Mar. 31, 2022	(164,215)	1,856	1,115,443	186	(1,281,700)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Net loss	(58,859)				(58,859)
Other comprehensive income (loss)	(171)			(171)
Exercise of warrants	3	3
Exercise of stock options, net	145		145
Stock-based compensation expense	9,865		9,865
Balance at Jun. 30, 2022	(213,232)	1,859	1,125,453	15	(1,340,559)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Net loss	(42,520)				(42,520)
Other comprehensive income (loss)	(113)			(113)
Employee stock purchase plan sales, net	1,357	1	1,356
Exercise of stock options, net	1,896	4	1,892
Stock-based compensation expense	9,953		9,953
Balance at Sep. 30, 2022	(242,659)	1,864	1,138,654	(98)	(1,383,079)
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	(53,333)				(53,333)
Other comprehensive income (loss)	559			559
Employee stock purchase plan sales, net	1,575	2	1,573
Exercise of stock options, net	3,502	8	3,494
Stock-based compensation expense	14,007		14,007
Balance at Mar. 31, 2023	(328,287)	1,889	1,177,192	585	(1,507,953)
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	(164,808)
Balance at Sep. 30, 2023	(410,986)	1,898	1,205,744	800	(1,619,428)
Balance at Mar. 31, 2023	(328,287)	1,889	1,177,192	585	(1,507,953)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Net loss	(75,326)				(75,326)
Other comprehensive income (loss)	105			105
Exercise of stock options, net	1,954	6	1,948
Stock-based compensation expense	12,841		12,841
Balance at Jun. 30, 2023	(388,713)	1,895	1,191,981	690	(1,583,279)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Net loss	(36,149)				(36,149)
Other comprehensive income (loss)	110			110
Employee stock purchase plan sales, net	1,020	2	1,018
Exercise of stock options, net	467	1	466
Stock-based compensation expense	12,279		12,279
Balance at Sep. 30, 2023	$ (410,986)	$ 1,898	$ 1,205,744	$ 800	$ (1,619,428)

- Definition

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

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Amount after tax and reclassification adjustments of other comprehensive income (loss).

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+ Details

Stock-based Compensation - Stock Plan Activities (Details) - $ / shares shares in Thousands	3 Months Ended						9 Months Ended
	Sep. 30, 2023	Jun. 30, 2023	Mar. 31, 2023	Sep. 30, 2022	Jun. 30, 2022	Mar. 31, 2022	Sep. 30, 2023
Options Outstanding
Options outstanding, stock option awards exercised (in shares)	(149)	(608)	(801)	(390)	(51)	(1,108)
Incentive Plan
Awards Available
Awards available, beginning balance (in shares)			4,206				4,206
Awards available, plan amendment (in shares)							7,000
Awards available, restricted stock awards granted (in shares)							(314)
Awards available, restricted stock awards cancelled (in shares)							491
Awards available, stock option awards granted (in shares)							(618)
Awards available, stock option awards cancelled (in shares)							1,169
Awards available, ending balance (in shares)	11,934						11,934
Options Outstanding
Options outstanding, beginning balance (in shares)			31,179				31,179
Options outstanding, stock option awards granted ( in shares)							618
Options outstanding, stock option awards exercised (in shares)							(856)
Options outstanding, stock option awards cancelled (in shares)							(1,169)
Options outstanding, ending balance (in shares)	29,772						29,772
Weighted Average Exercise Price
Weighted average exercise price, beginning balance (in usd per share)			$ 8.56				$ 8.56
Weighted average exercise price of stock option awards granted (in usd per share)							9.01
Weighted average exercise price of stock option awards exercised (in usd per share)							5.25
Weighted average exercise price of stock option awards cancelled (in usd per share)							9.93
Weighted average exercise price, ending balance (in usd per share)	$ 8.61						$ 8.61
Inducement Plan
Awards Available
Awards available, beginning balance (in shares)			947				947
Awards available, restricted stock awards granted (in shares)							(457)
Awards available, restricted stock awards cancelled (in shares)							93
Awards available, stock option awards granted (in shares)							(992)
Awards available, stock option awards cancelled (in shares)							609
Awards available, ending balance (in shares)	200						200
Options Outstanding
Options outstanding, beginning balance (in shares)			5,341				5,341
Options outstanding, stock option awards granted ( in shares)							992
Options outstanding, stock option awards exercised (in shares)							(336)
Options outstanding, stock option awards cancelled (in shares)							(609)
Options outstanding, ending balance (in shares)	5,388						5,388
Weighted Average Exercise Price
Weighted average exercise price, beginning balance (in usd per share)			$ 8.80				$ 8.80
Weighted average exercise price of stock option awards granted (in usd per share)							8.64
Weighted average exercise price of stock option awards exercised (in usd per share)							3.67
Weighted average exercise price of stock option awards cancelled (in usd per share)							10.36
Weighted average exercise price, ending balance (in usd per share)	$ 8.91						$ 8.91

Trade Receivables

9 Months Ended

Sep. 30, 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 September 30, 2023 and December 31, 2022, receivables net of reserves related to sales of ORLADEYO were $51,837 and $41,508, respectively. A returns reserve related to sales of ORLADEYO of $124 was recorded as of September 30, 2023. No reserve or allowance amounts were recorded as of December 31, 2022. At September 30, 2023 and December 31, 2022, receivables related to sales of RAPIVAB were $132 and $823, respectively.

Collaborations

Receivables from collaborations were as follows (in thousands):


	September 30, 2023
	Billed		Unbilled		Total
U.S. Department of Health and Human Services, net	$	—	$	113	$	113
Royalty receivables from partners	1,564		—		1,564
Total receivables	$	1,564	$	113	$	1,677


	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	$	7,959	$	309	$	8,268

As of September 30, 2023 and December 31, 2022, the Company maintained a reserve of $522 and $437, respectively, related to royalties associated with Green Cross.

Royalty Monetizations

9 Months Ended

Sep. 30, 2023

Advance Royalties [Abstract]

Royalty Monetizations

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 3.0% of proceeds received by the Company on annual net sales of up to $150,000 in the Other Markets, and (iii) 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, for 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 will be 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 7 herein) through its payoff and termination on April 17, 2023 or, subsequent to that date, the Pharmakon Loan Agreement (as defined in Note 7 herein), as applicable. See “Note 7—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 Condensed 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.

During the three months ended September 30, 2023, the Company adjusted its forecasts related to its BCX10013 program and updated its ORLADEYO forecast based on actual results for the first nine months of 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 RPI Royalty Purchase Agreements. As a result, the effective interest rate related to the 2020 RPI Royalty Purchase Agreement decreased from 22.2% to 22.1%, the effective interest rate related to the 2021 RPI Royalty Purchase Agreement decreased from 10.0% to 0.0%, and the effective interest rate related to the OMERS Royalty Purchase Agreement decreased from 10.6% to 10.2%.

The following table shows the activity within the Royalty financing obligations account (in thousands) as well as the effective interest rate as of September 30, 2023:


	2020 RPI Royalty Agreement			2021 RPI Royalty Agreement			OMERS Royalty Agreement			Total
Balance as of December 31, 2022	$	164,981		$	173,651		$	163,023		$	501,655

Non-cash Interest expense on Royalty financing obligations	9,309			5,680			4,329			19,318
Royalty revenues paid and payable	(6,038)			(524)			—			(6,562)
Balance as of March 31, 2023	$	168,252		$	178,807		$	167,352		$	514,411
Non-cash Interest expense on Royalty financing obligations	9,552			5,440			4,494			19,486
Royalty revenues paid and payable	(7,155)			(621)			—			(7,776)
Balance as of June 30, 2023	$	170,649		$	183,626		$	171,846		$	526,121
Non-cash Interest expense on Royalty financing obligations	9,647			3,068			4,557			17,272
Royalty revenues paid and payable	(7,553)			(654)			—			(8,207)
Balance as of September 30, 2023	$	172,743		$	186,040		$	176,403		$	535,186

Effective interest rate	22.1		%	—		%	10.2		%

The Royalty financing obligations liabilities and the associated deferred issuance costs are amortized using the effective interest method over the term of the arrangement, in accordance with the respective guidance.

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 9—Stockholders’ Equity” for further details on the common stock sale premium.

Condensed Consolidated Balance Sheets (Parenthetical) - $ / shares	Sep. 30, 2023	Dec. 31, 2022
Statement of Financial Position [Abstract]
Preferred stock par value (in usd per share)	$ 0.01	$ 0.01
Preferred stock, shares authorized (in shares)	5,000,000	5,000,000
Preferred stock, shares issued (in shares)	0	0
Preferred stock, shares outstanding (in shares)	0	0
Common stock par 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)	189,803,000	187,906,000
Common stock, shares, outstanding (in shares)	189,803,000	187,906,000

Equity (Tables)

9 Months Ended

Sep. 30, 2023

Equity [Abstract]

Schedule of Stock by Class

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


	As of September 30, 2023	As of December 31, 2022
Shares reserved for exercises of outstanding stock options	35,160	36,520
Shares reserved for vesting of restricted stock units	4,589	4,750
Shares reserved for exercises of warrants	15,023	15,023
Shares reserved for future issuance under the Stock Incentive Plan	11,934	4,206
Shares reserved for future issuance under the Inducement Equity Incentive Plan	200	947
Shares reserved for future issuance under the Employee Stock Purchase Plan	5,454	5,792
Total shares reserved for future issuance	72,360	67,238

- 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.

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+ Details

Significant Accounting Policies and Concentrations of Risk - Summary of Revenues (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
Significant Accounting Policies and Concentration of Risk [Line Items]
Revenues	$ 86,742	$ 75,827	$ 238,011	$ 191,282
Product sales, net
Significant Accounting Policies and Concentration of Risk [Line Items]
Revenues	85,288	75,213	233,957	189,647
Collaborative and other revenues
Significant Accounting Policies and Concentration of Risk [Line Items]
Revenues	$ 1,454	$ 614	$ 4,054	$ 1,635

- 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.

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+ Details

Condensed Consolidated Statements of Stockholders' Deficit (Parenthetical) - shares shares in Thousands	3 Months Ended						9 Months Ended
	Sep. 30, 2023	Jun. 30, 2023	Mar. 31, 2023	Sep. 30, 2022	Jun. 30, 2022	Mar. 31, 2022	Sep. 30, 2023	Sep. 30, 2022
Statement of Stockholders' Equity [Abstract]
Employee stock purchase plan sales, net (in shares)	162		176	145		115	338	260
Exercise of stock options, net (in shares)	149	608	801	390	51	1,108
Exercise of warrants (in shares)					253

Stockholders' Equity

9 Months Ended

Sep. 30, 2023

Stockholders' Equity Note [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 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.

Shares Reserved for Future Issuance of Common Stock

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


	As of September 30, 2023	As of December 31, 2022
Shares reserved for exercises of outstanding stock options	35,160	36,520
Shares reserved for vesting of restricted stock units	4,589	4,750
Shares reserved for exercises of warrants	15,023	15,023
Shares reserved for future issuance under the Stock Incentive Plan	11,934	4,206
Shares reserved for future issuance under the Inducement Equity Incentive Plan	200	947
Shares reserved for future issuance under the Employee Stock Purchase Plan	5,454	5,792
Total shares reserved for future issuance	72,360	67,238

- Definition

The entire disclosure for equity.

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Reference 15: http://www.xbrl.org/2003/role/disclosureRef
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Reference 16: http://www.xbrl.org/2003/role/disclosureRef
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Reference 17: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
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-Section 50
-Paragraph 18
-Subparagraph (a)
-Publisher FASB
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Reference 18: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
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-Name Accounting Standards Codification
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-Paragraph 18
-Subparagraph (b)
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Reference 19: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 18
-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481112/505-10-50-18

+ Details

Stock-based Compensation (Tables)

9 Months Ended

Sep. 30, 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):


	Nine Months Ended September 30,
	2023		2022
Incentive Plan	$	30,934	$	23,687
Inducement Plan	7,346		4,604
ESPP	847		1,130
Stock-based compensation expense	$	39,127	$	29,421

Stock Plan Activities

Related activity under the Incentive Plan is as follows (in thousands):


	Awards Available	Options Outstanding	Weighted Average Exercise Price
Balance December 31, 2022	4,206	31,179	$	8.56

Plan amendment	7,000	—	—
Restricted stock unit awards granted	(314)	—	—
Restricted stock unit awards cancelled	491	—	—
Stock option awards granted	(618)	618	9.01
Stock option awards exercised	—	(856)	5.25
Stock option awards cancelled	1,169	(1,169)	9.93
Balance September 30, 2023	11,934	29,772	$	8.61

Related activity under the Inducement Plan is as follows (in thousands):


	Awards Available	Options Outstanding	Weighted Average Exercise Price
Balance December 31, 2022	947	5,341	$	8.80

Restricted stock unit awards granted	(457)	—	—
Restricted stock unit awards cancelled	93	—	—
Stock option awards granted	(992)	992	8.64
Stock option awards exercised	—	(336)	3.67
Stock option awards cancelled	609	(609)	10.36
Balance September 30, 2023	200	5,388	$	8.91

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

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


	Nine Months Ended September 30,
	2023		2022
Expected Life in Years	5.5		5.5
Expected Volatility	83.9	%	84.1	%
Expected Dividend Yield	0.0	%	0.0	%
Risk-Free Interest Rate	3.9	%	2.9	%

Significant Accounting Policies and Concentrations of Risk - Net Loss Per Share (Details) - shares	9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022
Significant Accounting Policies and Concentration of Risk [Line Items]
Antidilutive securities excluded from computation of earnings per share, amount (in shares)	54,772,000	50,430,000
Outstanding stock options
Significant Accounting Policies and Concentration of Risk [Line Items]
Antidilutive securities excluded from computation of earnings per share, amount (in shares)	35,160,000	32,647,000
Restricted Stock Units (RSUs)
Significant Accounting Policies and Concentration of Risk [Line Items]
Antidilutive securities excluded from computation of earnings per share, amount (in shares)	4,589,000	2,760,000
Warrants to purchase common stock
Significant Accounting Policies and Concentration of Risk [Line Items]
Antidilutive securities excluded from computation of earnings per share, amount (in shares)	15,023,000	15,023,000

Cover - shares	9 Months Ended
Cover - shares	Sep. 30, 2023	Oct. 31, 2023
Cover [Abstract]
Document Type	10-Q
Document Quarterly Report	true
Document Period End Date	Sep. 30, 2023
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
Trading Symbol	BCRX
Security Exchange Name	NASDAQ
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
Entity Shell Company	false
Entity Common Stock, Shares Outstanding		204,809,380
Entity Central Index Key	0000882796
Current Fiscal Year End Date	--12-31
Document Fiscal Year Focus	2023
Document Fiscal Period Focus	Q3
Amendment Flag	false

Significant Accounting Policies and Concentrations of Risk (Tables)

9 Months Ended

Sep. 30, 2023

Accounting Policies [Abstract]

Summary of Revenues

The Company recorded the following revenues for the three and nine months ended September 30, 2023 and 2022:


	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Product sales, net	$	85,288	$	75,213	$	233,957	$	189,647
Collaborative and other revenues	1,454		614		4,054		1,635
Total revenues	$	86,742	$	75,827	$	238,011	$	191,282

The Company recorded the following revenues (in thousands):


	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
ORLADEYO:
U.S.	$	75,268	$	60,106	$	208,934	$	162,743
Outside of U.S.	10,416		5,866		26,173		18,155
Total ORLADEYO	85,684		65,972		235,107		180,898
Other revenues	1,058		9,855		2,904		10,384
Total revenues	$	86,742	$	75,827	$	238,011	$	191,282

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 September 30, 2023 and September 30, 2022, from the computation of diluted net loss per share attributable to common stockholders because including them would have had an anti-dilutive effect:


	September 30, 2023	September 30, 2022
Outstanding stock options	35,160	32,647
Unvested restricted stock unit awards	4,589	2,760
Warrants to purchase common stock	15,023	15,023
Total	54,772	50,430

Investments- Fair Value of the Company's Investments by Type (Details) - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Marketable Securities [Line Items]
Amortized Cost	$ 246,858	$ 138,196
Accrued Interest	49	487
Gross Unrealized Gains	3	0
Gross Unrealized Losses	(225)	(1,063)
Estimated Fair Value	$ 246,685	$ 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	$ 243,810	$ 129,940
Accrued Interest	37	427
Gross Unrealized Gains	3	0
Gross Unrealized Losses	(212)	(996)
Estimated Fair Value	243,638	129,371
Corporate debt securities
Marketable Securities [Line Items]
Amortized Cost	2,068	6,093
Accrued Interest	6	37
Gross Unrealized Gains	0	0
Gross Unrealized Losses	0	(38)
Estimated Fair Value	2,074	6,092
Certificates of deposit
Marketable Securities [Line Items]
Amortized Cost	980	2,163
Accrued Interest	6	23
Gross Unrealized Gains	0	0
Gross Unrealized Losses	(13)	(29)
Estimated Fair Value	$ 973	$ 2,157

Stock-based Compensation

9 Months Ended

Sep. 30, 2023

Share-Based Payment Arrangement [Abstract]

Share-Based Compensation

Stock-Based Compensation

As of September 30, 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):


	Nine Months Ended September 30,
	2023		2022
Incentive Plan	$	30,934	$	23,687
Inducement Plan	7,346		4,604
ESPP	847		1,130
Stock-based compensation expense	$	39,127	$	29,421

There was approximately $111,804 of total unrecognized compensation expense related to non-vested stock option and restricted stock unit awards granted by the Company as of September 30, 2023, which the Company expects to recognize over a weighted-average period of approximately 1.3 years. In addition, the Company has outstanding performance-based stock options and restricted stock unit awards for which no compensation expense is recognized until it is probable the performance conditions will be met.

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 September 30, 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 remaining awards were cancelled.

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.

Related activity under the Incentive Plan is as follows (in thousands):


	Awards Available	Options Outstanding	Weighted Average Exercise Price
Balance December 31, 2022	4,206	31,179	$	8.56

Plan amendment	7,000	—	—
Restricted stock unit awards granted	(314)	—	—
Restricted stock unit awards cancelled	491	—	—
Stock option awards granted	(618)	618	9.01
Stock option awards exercised	—	(856)	5.25
Stock option awards cancelled	1,169	(1,169)	9.93
Balance September 30, 2023	11,934	29,772	$	8.61

For stock option awards granted under the Incentive Plan during the first nine months of 2023 and 2022, the fair value was estimated on the date of grant using a Black-Scholes option pricing model and the assumptions noted in the table following the next subsection. The weighted average grant date fair value of these awards granted during the first nine months of 2023 and 2022 was $6.40 and $7.78, respectively. The fair value of the stock option awards is amortized to expense over the vesting periods using a straight-line expense attribution method. For restricted stock unit awards granted under the Incentive Plan, the fair value of the awards was 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 the first nine months of 2023 and 2022 was $9.18 and $13.18, 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.

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.

Related activity under the Inducement Plan is as follows (in thousands):


	Awards Available	Options Outstanding	Weighted Average Exercise Price
Balance December 31, 2022	947	5,341	$	8.80

Restricted stock unit awards granted	(457)	—	—
Restricted stock unit awards cancelled	93	—	—
Stock option awards granted	(992)	992	8.64
Stock option awards exercised	—	(336)	3.67
Stock option awards cancelled	609	(609)	10.36
Balance September 30, 2023	200	5,388	$	8.91

For stock option awards granted under the Inducement Plan during the first nine months of 2023 and 2022, the fair value was estimated on the date of grant using a Black-Scholes option pricing model and the assumptions noted in the table below. The weighted average grant date fair value of these awards granted during the first nine months of 2023 and 2022 was $6.14 and $9.72, respectively. The fair value of the stock option awards is amortized to expense over the vesting periods using a straight-line expense attribution method. For restricted stock unit awards granted under the Inducement Plan, the fair value of the awards was 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 the first nine months of 2023 and 2022 was $8.35 and $13.38, 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.

The following table summarizes the key assumptions used by the Company to value the stock option awards granted under all plans during the first nine months of 2023 and 2022. Historically, the expected life was based on the average of the assumption that all outstanding stock option awards would be exercised at full vesting and the assumption that all outstanding stock option awards would 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.

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


	Nine Months Ended September 30,
	2023		2022
Expected Life in Years	5.5		5.5
Expected Volatility	83.9	%	84.1	%
Expected Dividend Yield	0.0	%	0.0	%
Risk-Free Interest Rate	3.9	%	2.9	%

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 as of September 30, 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 the six-month purchase dates, 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 nine months ended September 30, 2023 and September 30, 2022, the Company issued 338 and 260 shares under the ESPP, 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.

Subsequent Event	9 Months Ended
Subsequent Event	Sep. 30, 2023
Subsequent Events [Abstract]
Subsequent Event	Subsequent Events 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. On November 3, 2023, the Company announced that it entered into a license agreement (the “Clearside Agreement”) with Clearside Biomedical, Inc. (“Clearside”), enabling the Company to develop its investigational plasma kallikrein inhibitor, avoralstat, with Clearside’s SCS Microinjector® to deliver avoralstat directly to the back of the eye through the suprachoroidal space to treat patients with diabetic macular edema. Under the Clearside Agreement, Clearside will receive a $5,000 upfront license fee from the Company. 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.

Significant Accounting Policies and Concentrations of Risk (Details) - USD ($)		3 Months Ended		9 Months Ended
	Apr. 17, 2023	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022	Dec. 31, 2022
Significant Accounting Policies and Concentration of Risk [Line Items]
Restricted cash		$ 1,584,000		$ 1,584,000		$ 1,472,000
Long-term investment maturity, minimum (in years)				12 months
Inventory adjustments		(170,000)		$ (170,000)
Inventory valuation reserves		1,347,000		1,347,000		1,177,000
Net proceeds from secured term loans				300,000,000	$ 73,072,000
Amortization of debt financing costs and original issue discounts		230,000	$ 258,000	$ 1,536,000	$ 597,000
Antidilutive securities excluded from computation of earnings per share, amount (in shares)				54,772,000	50,430,000
Reclassification from accumulated other comprehensive income, current period, before tax		$ 0	$ 0	$ 0	$ 0
Computer Equipment
Significant Accounting Policies and Concentration of Risk [Line Items]
Property, plant and equipment, useful life (in years)		3 years		3 years
Laboratory Equipment, Office Equipment and Software
Significant Accounting Policies and Concentration of Risk [Line Items]
Property, plant and equipment, useful life (in years)		5 years		5 years
Furniture and Fixtures
Significant Accounting Policies and Concentration of Risk [Line Items]
Property, plant and equipment, useful life (in years)		7 years		7 years
Maximum
Significant Accounting Policies and Concentration 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
Term Loan Tranche A \| Pharmakon Advisors, LP \| Line of Credit
Significant Accounting Policies and Concentration of Risk [Line Items]
Net proceeds from secured term loans	$ 300,000			$ 300,000,000
Collateral for Credit
Significant Accounting Policies and Concentration of Risk [Line Items]
Restricted cash		$ 1,466,000		$ 1,466,000		$ 1,449,000

Investments (Tables)

9 Months Ended

Sep. 30, 2023

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):


	September 30, 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	$	—	$	243,638	$	—	$	243,638
Corporate debt securities	—		2,074		—		2,074
Certificates of deposit	—		973		—		973
Total assets	$	—	$	246,685	$	—	$	246,685


	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

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


	September 30, 2023
	Amortized Cost		Accrued Interest		Gross Unrealized Gains		Gross Unrealized Losses		Estimated Fair Value
Obligations of U.S. Government and its agencies	$	243,810	$	37	$	3	$	(212)	$	243,638
Corporate debt securities	2,068		6		—		—		2,074
Certificates of deposit	980		6		—		(13)		973
Total investments	$	246,858	$	49	$	3	$	(225)	$	246,685


	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 September 30, 2023 and December 31, 2022 (in thousands):


	September 30, 2023		December 31, 2022
Maturing in one year or less	$	246,685	$	119,543
Maturing after one year through two years	—		18,077
Total investments	$	246,685	$	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 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 11: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 12: http://www.xbrl.org/2009/role/commonPracticeRef
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-Publisher FASB
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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
-Topic 320
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-Section 50
-Paragraph 5
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-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481800/320-10-50-5

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

Reference 4: http://www.xbrl.org/2009/role/commonPracticeRef
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-Section 50
-Paragraph 5
-Subparagraph (a)
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-URI https://asc.fasb.org//1943274/2147481800/320-10-50-5

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

Reference 6: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
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-Name Accounting Standards Codification
-Section 50
-Paragraph 5
-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481800/320-10-50-5

Reference 7: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
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-Subparagraph (e)
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Reference 8: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
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-URI https://asc.fasb.org//1943274/2147481800/320-10-50-5

Reference 9: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
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-Paragraph 5
-Subparagraph (f)(2)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481800/320-10-50-5

Reference 10: http://www.xbrl.org/2009/role/commonPracticeRef
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-Name Accounting Standards Codification
-Section 50
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-Subparagraph (f)(3)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481800/320-10-50-5

Reference 11: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
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-Name Accounting Standards Codification
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-URI https://asc.fasb.org//1943274/2147481800/320-10-50-5

Reference 12: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
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Reference 13: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
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-Name Accounting Standards Codification
-Section 50
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Reference 14: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
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Reference 15: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 16: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 17: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 18: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
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+ Details

Trade Receivables (Tables)

9 Months Ended

Sep. 30, 2023

Receivables [Abstract]

Summary of Receivables

Receivables from collaborations were as follows (in thousands):


	September 30, 2023
	Billed		Unbilled		Total
U.S. Department of Health and Human Services, net	$	—	$	113	$	113
Royalty receivables from partners	1,564		—		1,564
Total receivables	$	1,564	$	113	$	1,677


	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	$	7,959	$	309	$	8,268

Lease Obligations

9 Months Ended

Sep. 30, 2023

Leases [Abstract]

Lease Obligations

In June 2021, the Company entered into an operating sublease agreement for approximately 19,978 square feet of office space in Durham, North Carolina (the “Sublease Agreement”) with an expiration of December 2023. In March 2023, the Company signed a lease directly with the landlord commencing in January 2024 for the same space (the “Frontier Agreement”). The Company accounted for the Frontier Agreement as a new agreement and a separate transaction from the Sublease Agreement. In July 2023, the Company’s Sublease Agreement was terminated, and the Company amended the Frontier Agreement to move the commencement date from January 2024 to July 2023 to remain in continuous possession of the office space. The amendment was accounted for as a lease modification, and there was no impact as the lease commenced on the modification date. The Company recorded a right-of-use asset and lease liability of $2,063 in July 2023.

In February 2015, the Company entered into an operating lease agreement for approximately 31,601 square feet of office space in Birmingham, Alabama. In February 2020, the Company entered into an amendment to increase the existing space under the lease by approximately 2,846 rentable square feet (the “First Expansion Premises”). In December 2022, the Company entered into a second amendment to extend the term of the lease and to increase the existing space under the lease by 14,607 rentable square feet (the “Second Expansion Premises”). The second amendment allows for tenant improvement allowances not to exceed an aggregate of $515. The Company will capitalize all necessarily incurred leasehold improvement costs to bring the facilities to the condition for their intended use. In August 2023, the Second Expansion Premises commenced. The second amendment was accounted for as a lease modification, and the right-of-use asset and operating lease liability for the existing premises were remeasured at the modification date. The Company recorded a right-of-use asset of $5,327 and a lease liability of $5,925 in July 2023.

The Company leases certain assets, predominantly under operating leases, which consist of real estate leases, laboratory equipment leases and office equipment leases as of September 30, 2023. Renewal options for the Company’s leases range from 1 to 3 years in length and begin from 2024 through 2030.

Aggregate lease expense was as follows (in thousands):


	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Aggregate lease expense	$	1,014	$	634	$	2,505	$	1,854

Other supplemental information related to leases was as follows:

As of September 30, 2023

As of December 31, 2022

Weighted average remaining lease term

8.0 years

8.1 years

Weighted average discount rate

10.0%

11.0%

The following table summarizes the presentation in the Condensed Consolidated Balance Sheets of the Company’s leases:


	Balance Sheet Location	As of September 30, 2023		As of December 31, 2022
Assets:
Lease assets, net	Other Assets	$	11,380	$	6,806
Liabilities:
Current lease liabilities	Lease financing obligation – current liabilities	$	2,282	$	2,369
Non-current lease liabilities	Lease financing obligation – long-term liabilities	9,966		5,804
Total lease liabilities		$	12,248	$	8,173

Lease assets are recorded net of accumulated amortization of $6,350 and $4,349 as of September 30, 2023 and December 31, 2022, respectively.

Cash paid for amounts included in the measurement of lease liabilities was $834 and $2,293 for the three and nine months ended September 30, 2023, respectively. This compares to cash paid for amounts included in the measurement of lease liabilities of $615 and $1,796 for the three and nine months ended September 30, 2022, respectively.

Maturities of lease liabilities as of September 30, 2023, are as follows (in thousands):


2023 (remaining)	$	853
2024	3,279
2025	2,332
2026	1,846
2027	1,531
Thereafter	9,292
Total lease payments	19,133
Less imputed interest	(6,885)
Total	$	12,248

Significant Accounting Policies and Concentrations of Risk

9 Months Ended

Sep. 30, 2023

Accounting Policies [Abstract]

Significant Accounting Policies and Concentrations of Risk

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 pharmaceuticals 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, has the exclusive right to commercialize ORLADEYO for the prevention of HAE attacks in exchange for certain milestone and 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 September 30, 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 2023 expenses will exceed its 2023 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 largely be 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 issue securities, including common stock, preferred stock, depositary shares, purchase contracts, warrants, debt securities and units, through private placement transactions or registered public offerings in the future. 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 condensed 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 condensed consolidated financial statements.

The Company’s condensed consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States (“U.S. GAAP”) for interim financial reporting and the instructions to Form 10-Q and do not include all of the information and footnotes required by U.S. GAAP for complete financial statements. Such financial statements reflect all adjustments that are, in management’s opinion, necessary to present fairly, in all material respects, the Company’s condensed consolidated financial position, results of operations, and cash flows. There were no adjustments other than normal recurring adjustments.

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

These financial statements should be read in conjunction with the financial statements for the year ended December 31, 2022 and the notes thereto included in the Company’s 2022 Annual Report on Form 10-K. Interim operating results are not necessarily indicative of operating results for the full year. The condensed consolidated balance sheet as of December 31, 2022 has been derived from the audited consolidated financial statements included in the Company’s most recent Annual Report on Form 10-K.

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 condensed 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 three and nine months ended September 30, 2023 and 2022:


	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Product sales, net	$	85,288	$	75,213	$	233,957	$	189,647
Collaborative and other revenues	1,454		614		4,054		1,635
Total revenues	$	86,742	$	75,827	$	238,011	$	191,282

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 sales of RAPIVAB to the U.S. Department of Health and Human Services (“HHS”) under the Company’s procurement contract. 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 its product.

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 products 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 Condensed Consolidated Statements of Comprehensive Loss rather than as a reduction in expenses. Under the Company’s contracts with the Biomedical Advanced Research and Development Authority within HHS (“BARDA/HHS”) and the National Institute of Allergy and Infectious Diseases (“NIAID/HHS”), revenue is 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, certificates of deposit, money market accounts or investments in debt instruments 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 $1,584 and $1,472 as of September 30, 2023 and December 31, 2022, respectively, and primarily consisted of $1,466 and $1,449 as of September 30, 2023 and December 31, 2022, respectively, for a letter of credit the Company is required to maintain associated with the lease execution and build-out of its Birmingham research facilities.

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. Unrealized gains and losses on investments are recognized in comprehensive loss, unless an unrealized loss is considered to be other than temporary, in which case the unrealized loss is charged to operations. The Company periodically reviews its investments for other than temporary declines in fair value below cost basis and whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. 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. Realized gains and losses are reflected in interest and other income in the Condensed 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.

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 HHS, and royalty receivables from the Company’s partners, including Shionogi & Co., Ltd., Green Cross, and Torii.

Monthly invoices are submitted to HHS related to reimbursable research and development costs. The Company is 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 labeling, including raw material, active product ingredient, and the drug product. Finished goods include packaged and labeled 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. During the nine months ended September 30, 2023, the Company evaluated its inventory levels and associated expiration dating relative to the latest sales forecasts for ORLADEYO, RAPIVAB, and peramivir and estimated those

inventories at risk of obsolescence. Accordingly, the Company recorded an increase to the inventory valuation reserve of $170 for a total reserve of $1,347 as of September 30, 2023.

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 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.

In accordance with U.S. GAAP, 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 does not identify costs that it has begun to incur or if it underestimates or overestimates the level of these costs, actual expenses could differ from such estimates. As of September 30, 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, if required, based upon the Company’s ongoing review of the level of services actually performed.

Additionally, the Company has license agreements with third parties which require fees related to sublicense agreements or maintenance fees. The Company expenses sublicense payments as incurred unless they are related to revenues that have been deferred, in which case the expenses are deferred and recognized over the related revenue recognition period. The Company expenses maintenance payments as incurred.

Deferred collaboration expenses represent sublicense payments paid to the Company’s academic partners upon receipt of consideration from various commercial partners, and other consideration paid to the Company’s academic partners for modification to existing license agreements. These deferred expenses would not have been incurred without receipt of such payments or modifications from the Company’s commercial partners and are being expensed in proportion to the related revenue being recognized. The Company believes that this accounting treatment appropriately matches expenses with the associated revenue.

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 expense is 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.

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, predominantly under operating leases, which consist of real estate leases, laboratory equipment leases and office equipment leases as of September 30, 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 Condensed 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 Condensed Consolidated Balance Sheets.

Stock-Based Compensation

All share-based payments, including grants of stock option awards and restricted stock unit awards, are recognized in the Company’s Condensed 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 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 royalty financing obligations (Note 6) and the secured term loan borrowings under the Athyrium Credit Agreement during 2022 and the term loan borrowings under both the Athyrium Credit Agreement and the Pharmakon Loan Agreement during 2023 (Note 7). On April 17, 2023, the Company entered into the Pharmakon Loan Agreement and received initial funding in the form of a term loan of $300,000, the proceeds of which were primarily used to repay the term loan borrowings under the Athyrium Credit Agreement. Accordingly, interest expense for both the three and nine months ended September 30, 2023 includes interest expense from both the Athyrium Credit Agreement and the Pharmakon Loan Agreement. Costs directly associated with the borrowings have been capitalized and are netted against the corresponding debt liabilities on the Condensed Consolidated Balance Sheets. These costs are being amortized to interest expense over the terms of the corresponding borrowings using the effective interest rate method. Amortization of deferred financing costs included in interest expense was $230 and $1,536 for the three and nine months ended September 30, 2023, respectively, and $258 and $597 for the three and nine months ended September 30, 2022, respectively. When utilizing the effective interest method, in periods in which payment-in-kind (“PIK”) interest was designated and was added to the outstanding principal balance of the borrowing, the amortization of the deferred debt fees and issuance costs was accretive. The quarter ended December 31, 2022 was the last period eligible for the PIK Interest Payment designation under the Athyrium Credit Agreement.

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 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”), 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 15 in the case of development conducted on foreign soil.

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 September 30, 2023 and September 30, 2022, from the computation of diluted net loss per share attributable to common stockholders because including them would have had an anti-dilutive effect:


	September 30, 2023	September 30, 2022
Outstanding stock options	35,160	32,647
Unvested restricted stock unit awards	4,589	2,760
Warrants to purchase common stock	15,023	15,023
Total	54,772	50,430

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 income and recorded as interest and other income on the Condensed Consolidated Statements of Comprehensive Loss. There were no realized gains or losses reclassified out of accumulated other comprehensive income for the three and nine months ended September 30, 2023 and 2022.

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, for 2022, sales of RAPIVAB (peramivir injection) under the Company’s procurement contract with the Assistant Secretary for Preparedness and Response within HHS.

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.

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 Condensed 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

There have been no new accounting pronouncements adopted by the Company or new accounting pronouncements issued by the Financial Accounting Standards Board during the nine months ended September 30, 2023, as compared to the recent accounting pronouncements described in Note 1 of the Company’s Annual Report on Form 10-K for the year ended December 31, 2022, that the Company believes are of significance or potential significance to the Company.

Stockholders' Equity (Details) - Common Stock Purchase Agreement with RPI - USD ($) $ / shares in Units, shares in Thousands, $ in Thousands	Nov. 19, 2021	Nov. 17, 2021
Equity Disclosure [Line Items]
Stock issued during period, shares, new issues (in shares)	3,846
Proceeds from issuance of common stock	$ 50,000
Shares issued, price per share (in usd per share)	$ 13.00
Shares issued, price per share, premium (in usd per share)	1.11
Shares issued, price per share, closing price (in usd per share)	$ 11.89
Shares issued, common stock, premium		$ 4,269

Royalty Monetizations (Details Textual) - USD ($) $ in Thousands	Nov. 19, 2021	Dec. 07, 2020	Sep. 30, 2023
RPI 2019 Intermediate Finance Trust
Royalty Monetizations [Line Items]
Shares issued, common stock, premium			$ 4,269
RPI 2019 Intermediate Finance Trust \| ORLADEYO
Royalty 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
Royalty 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
Royalty 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
Royalty 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
Royalty 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
Royalty 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
Royalty Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of annual net sales in other markets		0.00%
RPI 2019 Intermediate Finance Trust \| BCX10013
Royalty 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 \| Future Royalties Payable
Royalty Monetizations [Line Items]
Proceeds from issuance of debt		$ 125,000
RPI 2021 and 2020 Intermediate Finance Trust \| Future Royalties Payable
Royalty Monetizations [Line Items]
Proceeds from issuance of debt	$ 150,000
The 2021 RPI Royalty Purchase Agreement \| Annual Net Sales Under $350,000
Royalty 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
Royalty Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of annual net sales in key territories	1.75%
The 2021 RPI Royalty Purchase Agreement \| Annual Net Sales Over $550,000
Royalty Monetizations [Line Items]
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
Royalty 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
Royalty Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of sublicense revenue in other markets	2.00%
The 2021 RPI Royalty Purchase Agreement \| ORLADEYO \| Annual Net Sales Over $230,000
Royalty Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of annual net sales in other markets	0.00%
The 2021 RPI Royalty Purchase Agreement \| BCX10013 \| Annual Net Sales Under $1.5 Million
Royalty Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of annual net sales in key territories	3.00%
The 2021 RPI Royalty Purchase Agreement \| BCX10013 \| Annual Net Sales Between $1.5 and $3 Millions
Royalty Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of annual net sales in key territories	2.00%
The 2021 RPI Royalty Purchase Agreement \| BCX10013 \| Annual Net Sales Over $3 Million
Royalty Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of annual net sales in other markets	0.00%
OMERS Capital Markets \| ORLADEYO
Royalty 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
OMERS Capital Markets \| ORLADEYO \| Annual Net Sales Under $350,000
Royalty 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
Royalty 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
Royalty 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
Royalty 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
Royalty 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
Royalty Monetizations [Line Items]
Royalty purchase agreement, royalties, percentage of sublicense revenue in other markets	0.00%

Trade Receivables (Details Textual) - Trade Accounts Receivable - USD ($)	Sep. 30, 2023	Dec. 31, 2022
Accounts, Notes, Loans and Financing Receivable [Line Items]
Accounts receivable, after allowance for credit loss, total	$ 124,000	$ 0
ORLADEYO
Accounts, Notes, Loans and Financing Receivable [Line Items]
Accounts receivable, after allowance for credit loss, total	51,837,000	41,508,000
Outside of U.S.
Accounts, Notes, Loans and Financing Receivable [Line Items]
Accounts receivable, after allowance for credit loss, total	132,000	823,000
Green Cross Corporation
Accounts, Notes, Loans and Financing Receivable [Line Items]
Accounts receivable, after allowance for credit loss, total	$ 522,000	$ 437,000

Lease Obligations - Other Supplemental Information Related to Leases (Details)	Sep. 30, 2023	Dec. 31, 2022
Leases [Abstract]
Weighted average remaining lease term (in years)	8 years	8 years 1 month 6 days
Weighted average discount rate	10.00%	11.00%

Lease Obligations -Balance Sheets of the Company's Operating Leases (Details) - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Assets:
Other assets [extensible enumeration]	Other assets
Operating lease assets, net	$ 11,380	$ 6,806
Liabilities:
Lease financing obligation	2,282	2,369
Lease financing obligation	9,966	5,804
Total lease liabilities	$ 12,248	$ 8,173

Condensed Consolidated Balance Sheets - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Assets
Cash and cash equivalents	$ 150,905	$ 304,767
Restricted cash	1,584	1,472
Investments	246,685	119,543
Trade receivables	53,646	50,599
Inventory, net	29,630	27,533
Prepaid expenses and other current assets	20,016	12,586
Total current assets	502,466	516,500
Property and equipment, net	8,999	8,617
Long-term investments	0	18,077
Other assets	11,459	6,806
Total assets	522,924	550,000
Liabilities and Stockholders’ Deficit
Accounts payable	10,567	14,356
Accrued expenses	77,392	87,565
Deferred revenue	522	1,224
Lease financing obligation	2,282	2,369
Total current liabilities	90,763	105,514
Lease financing obligation	9,966	5,804
Royalty financing obligations	535,186	501,655
Secured term loans	297,995	231,624
Stockholders’ deficit:
Preferred stock, $0.01 par value; shares authorized - 5,000; no shares issued and outstanding	0	0
Common stock, $0.01 par value; shares authorized - 450,000; shares issued and outstanding – 189,803 as of September 30, 2023 and 187,906 as of December 31, 2022	1,898	1,879
Additional paid-in capital	1,205,744	1,158,118
Accumulated other comprehensive income	800	26
Accumulated deficit	(1,619,428)	(1,454,620)
Total stockholders’ deficit	(410,986)	(294,597)
Total liabilities and stockholders’ deficit	$ 522,924	$ 550,000

Inventory (Details) - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Inventory Disclosure [Abstract]
Raw materials	$ 6,656	$ 8,906
Work-in-process	17,739	14,990
Finished goods	6,582	4,814
Total inventory	30,977	28,710
Reserves	(1,347)	(1,177)
Total inventory, net	$ 29,630	$ 27,533

Insider Trading Arrangements	3 Months Ended
Insider Trading Arrangements	Sep. 30, 2023
Trading Arrangements, by Individual
Rule 10b5-1 Arrangement Adopted	false
Non-Rule 10b5-1 Arrangement Adopted	false
Rule 10b5-1 Arrangement Terminated	false
Non-Rule 10b5-1 Arrangement Terminated	false

Condensed Consolidated Statements of Cash Flows - USD ($) $ in Thousands	9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022
Cash flows from operating activities:
Net loss	$ (164,808)	$ (175,575)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation and amortization	1,232	1,041
Inventory obsolescence	236	0
Stock-based compensation expense	39,127	29,419
Non-cash interest expense on royalty financing obligations and secured term loan and amortization of debt issuance costs	66,286	55,119
Amortization of premium/discount on investments	(6,814)	(818)
Loss on extinguishment of debt	(29,019)	0
Changes in operating assets and liabilities:
Receivables	(3,125)	(13,827)
Inventory	(2,357)	(11,263)
Prepaid expenses and other assets	(7,530)	(1,132)
Accounts payable and accrued expenses	(36,678)	(27,639)
Interest payable	0	6,345
Deferred revenue	(811)	329
Net cash used in operating activities	(86,223)	(138,001)
Cash flows from investing activities:
Acquisitions of property and equipment	(1,614)	(825)
Purchase of investments	(357,419)	(244,283)
Sales and maturities of investments	256,008	39,655
Net cash used in investing activities	(103,025)	(205,453)
Cash flows from financing activities:
Net proceeds from common stock issued under stock-based compensation plans	8,518	10,756
Net proceeds from secured term loans	300,000	73,072
Repayment of Athyrium secured term loans principal	(240,452)	0
Prepayment and repayment fees on Athyrium secured term loans	(21,261)	0
Payment of debt issuance costs on Pharmakon Tranche A term loan	(11,147)	0
Net cash provided by financing activities	35,658	83,828
Effect of exchange rate on cash, cash equivalents, and restricted cash	(160)	235
Decrease in cash, cash equivalents and restricted cash	(153,750)	(259,391)
Cash, cash equivalents and restricted cash at beginning of period	306,239	507,734
Cash, cash equivalents and restricted cash at end of period	$ 152,489	$ 248,343

Pay vs Performance Disclosure - USD ($) $ in Thousands	3 Months Ended						9 Months Ended
Pay vs Performance Disclosure - USD ($) $ in Thousands	Sep. 30, 2023	Jun. 30, 2023	Mar. 31, 2023	Sep. 30, 2022	Jun. 30, 2022	Mar. 31, 2022	Sep. 30, 2023	Sep. 30, 2022
Pay vs Performance Disclosure
Net loss	$ (36,149)	$ (75,326)	$ (53,333)	$ (42,520)	$ (58,859)	$ (74,196)	$ (164,808)	$ (175,575)

- Definition

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

+ References

+ Details

Lease Obligations - Maturities of Operating Lease Liabilities (Details) - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Leases [Abstract]
2023 (remaining)	$ 853
2024	3,279
2025	2,332
2026	1,846
2027	1,531
Thereafter	9,292
Total lease payments	19,133
Less imputed interest	(6,885)
Total	$ 12,248	$ 8,173
Operating lease, liability, statement of financial position [extensible enumeration]	Accrued expenses

Significant Accounting Policies and Concentrations of Risk (Policies)

9 Months Ended

Sep. 30, 2023

Accounting Policies [Abstract]

The Company

Basis of Presentation

Use of Estimates

Revenue Recognition

The Company recorded the following revenues for the three and nine months ended September 30, 2023 and 2022:


	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Product sales, net	$	85,288	$	75,213	$	233,957	$	189,647
Collaborative and other revenues	1,454		614		4,054		1,635
Total revenues	$	86,742	$	75,827	$	238,011	$	191,282

Product Sales, Net

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 its product.

Collaborative and Other Revenues

Cash and Cash Equivalents

Restricted Cash

Restricted Cash Total restricted cash was $1,584 and $1,472 as of September 30, 2023 and December 31, 2022, respectively, and primarily consisted of $1,466 and $1,449 as of September 30, 2023 and December 31, 2022, respectively, for a letter of credit the Company is required to maintain associated with the lease execution and build-out of its Birmingham research facilities.

Investments

Trade Receivables

Inventory

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

inventories at risk of obsolescence. Accordingly, the Company recorded an increase to the inventory valuation reserve of $170 for a total reserve of $1,347 as of September 30, 2023.

Property and Equipment

Accrued Expenses

Cost of Product Sales

Research and Development Expenses

Selling, General and Administrative Expenses

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, predominantly under operating leases, which consist of real estate leases, laboratory equipment leases and office equipment leases as of September 30, 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.

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

Stock-Based Compensation

Interest Expense and Deferred Financing Costs

Interest Expense and Royalty Financing Obligations

Income Taxes

Net Loss Per Share


	September 30, 2023	September 30, 2022
Outstanding stock options	35,160	32,647
Unvested restricted stock unit awards	4,589	2,760
Warrants to purchase common stock	15,023	15,023
Total	54,772	50,430

Accumulated Other Comprehensive Income

Significant Customers and Other Risks

Significant Customers

continue these distribution activities could adversely impact the Company’s business, results of operations and financial condition.

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

Lease Obligations (Tables)

9 Months Ended

Sep. 30, 2023

Leases [Abstract]

Aggregate Lease Expense Under Operating Leases

Aggregate lease expense was as follows (in thousands):


	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Aggregate lease expense	$	1,014	$	634	$	2,505	$	1,854

Other Supplemental Information Related to Leases

Other supplemental information related to leases was as follows:

As of September 30, 2023

As of December 31, 2022

Weighted average remaining lease term

8.0 years

8.1 years

Weighted average discount rate

10.0%

11.0%

Balance Sheets of the Company's Operating Leases

The following table summarizes the presentation in the Condensed Consolidated Balance Sheets of the Company’s leases:


	Balance Sheet Location	As of September 30, 2023		As of December 31, 2022
Assets:
Lease assets, net	Other Assets	$	11,380	$	6,806
Liabilities:
Current lease liabilities	Lease financing obligation – current liabilities	$	2,282	$	2,369
Non-current lease liabilities	Lease financing obligation – long-term liabilities	9,966		5,804
Total lease liabilities		$	12,248	$	8,173

Maturities of Operating Lease Liabilities

Maturities of lease liabilities as of September 30, 2023, are as follows (in thousands):


2023 (remaining)	$	853
2024	3,279
2025	2,332
2026	1,846
2027	1,531
Thereafter	9,292
Total lease payments	19,133
Less imputed interest	(6,885)
Total	$	12,248

Collaborative and Other Relationships (Details) $ in Thousands		3 Months Ended		9 Months Ended
	Nov. 05, 2019 USD ($)	Sep. 30, 2023 USD ($)	Sep. 30, 2022 USD ($)	Sep. 30, 2023 USD ($) dose	Sep. 30, 2022 USD ($)
Collaborative and Other Relationships [Line Items]
Revenues		$ 86,742	$ 75,827	$ 238,011	$ 191,282
Torii Pharmaceutical Co.
Collaborative and Other Relationships [Line Items]
Revenues	$ 22,000
Potential milestone payments receivable if regulatory approval before specified date	$ 15,000
Maximum customary reduction on royalty rate	50.00%
Royalty payments receivable, expiration term from first commercial (in years)	10 years
Torii Pharmaceutical Co. \| Minimum
Collaborative and Other Relationships [Line Items]
Royalty rate if maintains sakigake designation	20.00%
Torii Pharmaceutical Co. \| Maximum
Collaborative and Other Relationships [Line Items]
Royalty rate if maintains sakigake designation	40.00%
U.S. Department of Health and Human Services, net
Collaborative and Other Relationships [Line Items]
Contract term (in years)				5 years
U.S. Department of Health and Human Services, net \| Outside of U.S.
Collaborative and Other Relationships [Line Items]
Maximum number of products, doses \| dose				50,000
Number of product delivered, doses \| dose				49,980

Collaborative and Other Relationships	9 Months Ended
Collaborative and Other Relationships	Sep. 30, 2023
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Collaborative and Other Relationships	Collaborative and Other Relationships ORLADEYO Torii Pharmaceutical Co., Ltd. On November 5, 2019, the Company entered into a Commercialization and License Agreement with Torii (the “Torii Agreement”), granting Torii the exclusive right to commercialize ORLADEYO for the prevention of HAE attacks in Japan. Under the Torii Agreement, the Company received an upfront, non-refundable payment of $22,000. The Japanese National Health Insurance System’s (“NHI”) approval of the addition of ORLADEYO to the NHI drug price list in April 2021 triggered a $15,000 milestone payment from Torii to the Company, which was received in May 2021. In addition, under the Torii Agreement, the Company is 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 are subject to customary reductions in certain circumstances, but may not be reduced by more than 50% of the amount that otherwise would have been payable to the Company in the applicable calendar quarter. Torii’s royalty payment obligations commenced upon the first commercial sale of ORLADEYO in Japan 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 is responsible for supplying Torii with its required amounts of ORLADEYO. The activities of the parties pursuant to the Torii Agreement are overseen by a joint steering committee, composed of an equal number of representatives from each party to coordinate the development and commercialization of ORLADEYO in Japan. Torii launched ORLADEYO in Japan on April 23, 2021. The Company identified performance obligations 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. Peramivir Injection (RAPIVAB, RAPIACTA, PERAMIFLU) Shionogi & Co., Ltd. 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. The Company developed peramivir under a license from the University of Alabama at Birmingham (“UAB”) and will owe sublicense payments to UAB on any future milestone payments and/or royalties received by the Company from Shionogi. 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. Green Cross Corporation 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. Government Collaborations The Company has previously entered into contracts with the U.S. Government, including the procurement contract with HHS for up to 50,000 doses of RAPIVAB over a five-year period to supply the Strategic National Stockpile for use in a public health emergency and contracts with NIAID/HHS and BARDA/HHS for the development of galidesivir. As of September 30, 2023, the Company has delivered a total of 49,980 RAPIVAB doses of the 50,000 RAPIVAB doses available under the procurement contract, effectively completing the contract with HHS, and all of the Company’s government funding for galidesivir has expired.

Inventory (Tables)

9 Months Ended

Sep. 30, 2023

Inventory Disclosure [Abstract]

Summary of Inventories

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


	September 30, 2023		December 31, 2022
Raw materials	$	6,656	$	8,906
Work-in-process	17,739		14,990
Finished goods	6,582		4,814
Total inventory	$	30,977	$	28,710
Reserves	(1,347)		(1,177)
Total inventory, net	$	29,630	$	27,533

Debt (Details)			3 Months Ended		9 Months Ended
Debt (Details)	Apr. 17, 2023 USD ($)	Dec. 07, 2020 USD ($)	Sep. 30, 2023 USD ($)	Sep. 30, 2022 USD ($)	Sep. 30, 2023 USD ($)	Sep. 30, 2022 USD ($)	Jul. 29, 2022 USD ($)
Debt Instrument [Line Items]
Net proceeds from secured term loans					$ 300,000,000	$ 73,072,000
Amortization of debt issuance costs					1,069,000	(597,000)
Loss on extinguishment of debt			$ 0	$ 0	29,019,000	0
Pharmakon Advisors, LP
Debt Instrument [Line Items]
Debt instrument capitalized interest rate	0.50
Pharmakon Advisors, LP \| Secured Overnight Financing Rate (SOFR) Overnight Index Swap Rate
Debt Instrument [Line Items]
Debt Instrument, SOFR 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]
Debt instrument, 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 Fourth Anniversary
Debt Instrument [Line Items]
Debt instrument, prepayment fee, percentage of principal voluntary payments					1.00%
Pharmakon Advisors, LP \| Line of Credit \| After Third Anniversary
Debt Instrument [Line Items]
Debt instrument, prepayment fee, percentage of principal voluntary payments					2.00%
Athyrium
Debt Instrument [Line Items]
Periodic payment, interest						$ 15,311
Paid-in-kind interest					$ 8,476,000
Athyrium \| Senior Credit Facility
Debt Instrument [Line Items]
Repayments of lines of credit	$ 241,787
MidCap Financial Services, LLC \| Senior Credit Facility
Debt Instrument [Line Items]
Repayments of lines of credit		$ 43,298,000
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				$ 300,000,000
Debt instrument, term	18 months
Effective interest rate			13.24%		13.24%
Interest expense, debt, total					$ 17,350,000
Periodic payment, interest					8,675,000
Fair value			$ 308,675,000		308,675,000
Debt instrument, unamortized discount (premium) and debt issuance costs, net, total			11,147,000		11,147,000
Amortization of debt issuance costs			$ 230,000		467,000
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
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
Credit Agreement \| Athyrium
Debt Instrument [Line Items]
Debt instrument, face amount		$ 200,000,000
Debt instrument, term	17 days
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%
Long-term debt, total	$ 240,452,000
Interest expense	1,355
Fee amount	17,261,000
Deferred financing costs	11,758,000
Loss on extinguishment of debt					$ 29,019,000
Credit Agreement \| Athyrium \| Prior to the Second Anniversary
Debt Instrument [Line Items]
Debt instrument, prepayment fee, percentage of principal voluntary payments		102.00%
Debt instrument, prepayment fee, percentage of accrued interest		0.50%
Credit Agreement \| Athyrium \| Between the Second and Third Anniversaries
Debt Instrument [Line Items]
Debt instrument, prepayment fee, percentage of principal voluntary payments		2.00%
Credit Agreement \| Athyrium \| Between the Third and Fourth Anniversaries
Debt Instrument [Line Items]
Debt instrument, prepayment fee, percentage of principal voluntary payments		1.00%
Credit Agreement \| Athyrium \| After Fourth Anniversary
Debt Instrument [Line Items]
Debt instrument, prepayment fee, percentage of principal voluntary payments		0.00%
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
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
Credit Agreement \| Athyrium \| Term Loans
Debt Instrument [Line Items]
Effective interest rate			12.68%		12.68%
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

Royalty Monetizations (Tables)

9 Months Ended

Sep. 30, 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 September 30, 2023:


	2020 RPI Royalty Agreement			2021 RPI Royalty Agreement			OMERS Royalty Agreement			Total
Balance as of December 31, 2022	$	164,981		$	173,651		$	163,023		$	501,655

Non-cash Interest expense on Royalty financing obligations	9,309			5,680			4,329			19,318
Royalty revenues paid and payable	(6,038)			(524)			—			(6,562)
Balance as of March 31, 2023	$	168,252		$	178,807		$	167,352		$	514,411
Non-cash Interest expense on Royalty financing obligations	9,552			5,440			4,494			19,486
Royalty revenues paid and payable	(7,155)			(621)			—			(7,776)
Balance as of June 30, 2023	$	170,649		$	183,626		$	171,846		$	526,121
Non-cash Interest expense on Royalty financing obligations	9,647			3,068			4,557			17,272
Royalty revenues paid and payable	(7,553)			(654)			—			(8,207)
Balance as of September 30, 2023	$	172,743		$	186,040		$	176,403		$	535,186

Effective interest rate	22.1		%	—		%	10.2		%

Stock-based Compensation (Details Textual) $ / shares in Units, $ in Thousands	1 Months Ended		3 Months Ended				9 Months Ended		12 Months Ended
	Jan. 31, 2022 shares	Dec. 31, 2014 shares	Sep. 30, 2023 USD ($) plan shares	Mar. 31, 2023 shares	Sep. 30, 2022 shares	Mar. 31, 2022 shares	Sep. 30, 2023 USD ($) plan $ / shares shares	Sep. 30, 2022 $ / shares shares	Dec. 31, 2022 shares
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Number of plans \| plan			3				3
Total unrecognized compensation expense related to non-vested stock options and RSU awards \| $			$ 111,804				$ 111,804
Weighted-average term to recognize non-vested stock options and RSU awards							1 year 3 months 18 days
Employee stock purchase plan sales, net (in shares)			162,000	176,000	145,000	115,000	338,000	260,000
Outstanding stock options \| Non-employee Directors
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Award vesting period (in years)							1 year
Incentive Plan
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Number of common stock shares available for purchase (in shares)			11,934,000				11,934,000		4,206,000
Incentive Plan \| Outstanding stock options
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Weighted average grant date fair value stock option awards (in dollars per share) \| $ / shares							$ 6.40	$ 7.78
Incentive Plan \| Outstanding stock options \| Maximum
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Stock option awards remaining contractual term (in years)							10 years
Incentive Plan \| Outstanding stock options \| 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 (in years)							4 years
Incentive Plan \| Outstanding stock options \| Thirty-six Equal Monthly Installments \| Non-employee Directors
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Award vesting period (in years)							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%
Performance-based stock options issued in period (in shares)		1,250,000
Incentive Plan \| Performance-based Restricted Stock Units
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Award vesting period (in years)	3 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]
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]
Performance-based restricted stock unit awards issued (in shares)	221,000								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 \| Restricted Stock Units (RSUs)
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Weighted average grant date fair value (in dollars per share) \| $ / shares							$ 9.18	13.18
Inducement Plan
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Award vesting period (in years)							4 years
Expiration period (in years)							10 years
Weighted average grant date fair value stock option awards (in dollars per share) \| $ / shares							$ 6.14	9.72
Number of common stock shares available for purchase (in shares)			200,000				200,000		947,000
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 \| Outstanding stock options
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Expected Dividend Yield							0.00%
Inducement Plan \| Restricted Stock Units (RSUs)
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Weighted average grant date fair value (in dollars per share) \| $ / shares							$ 8.35	$ 13.38
Employee Stock Purchase Plan
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Number of shares of common stock authorized to be purchased (in shares)			7,975,000				7,975,000
Number of common stock shares available for purchase (in shares)			5,454,000				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)							3,000
Maximum number of shares per employee, amount \| $			$ 25				$ 25

Investments - Fair Value, Recurring Basis (Details) - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Marketable Securities [Line Items]
Estimated Fair Value	$ 246,685	$ 137,620
Obligations of U.S. Government and its agencies
Marketable Securities [Line Items]
Estimated Fair Value	243,638	129,371
Corporate debt securities
Marketable Securities [Line Items]
Estimated Fair Value	2,074	6,092
Certificates of deposit
Marketable Securities [Line Items]
Estimated Fair Value	973	2,157
Quoted Price in Active Markets (Level 1)
Marketable Securities [Line Items]
Estimated Fair Value	0	0
Quoted Price in Active Markets (Level 1) \| Obligations of U.S. Government and its agencies
Marketable Securities [Line Items]
Estimated Fair Value	0	0
Quoted Price in Active Markets (Level 1) \| Corporate debt securities
Marketable Securities [Line Items]
Estimated Fair Value	0	0
Quoted Price in Active Markets (Level 1) \| Certificates of deposit
Marketable Securities [Line Items]
Estimated Fair Value	0	0
Significant Other Observable Inputs (Level 2)
Marketable Securities [Line Items]
Estimated Fair Value	246,685	137,620
Significant Other Observable Inputs (Level 2) \| Obligations of U.S. Government and its agencies
Marketable Securities [Line Items]
Estimated Fair Value	243,638	129,371
Significant Other Observable Inputs (Level 2) \| Corporate debt securities
Marketable Securities [Line Items]
Estimated Fair Value	2,074	6,092
Significant Other Observable Inputs (Level 2) \| Certificates of deposit
Marketable Securities [Line Items]
Estimated Fair Value	973	2,157
Significant Unobservable Inputs (Level 3)
Marketable Securities [Line Items]
Estimated Fair Value	0	0
Significant Unobservable Inputs (Level 3) \| Obligations of U.S. Government and its agencies
Marketable Securities [Line Items]
Estimated Fair Value	0	0
Significant Unobservable Inputs (Level 3) \| Corporate debt securities
Marketable Securities [Line Items]
Estimated Fair Value	0	0
Significant Unobservable Inputs (Level 3) \| Certificates of deposit
Marketable Securities [Line Items]
Estimated Fair Value	$ 0	$ 0

Stock-based Compensation - Share-Based Compensation (Details) - USD ($) $ in Thousands	9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Share-based payment arrangement, expense	$ 39,127	$ 29,421
Incentive Plan
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Share-based payment arrangement, expense	30,934	23,687
Inducement Plan
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Share-based payment arrangement, expense	7,346	4,604
Employee Stock Purchase Plan
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Share-based payment arrangement, expense	$ 847	$ 1,130

- 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

Royalty Monetizations - Schedule of Royalty Financing Obligations (Details) - USD ($) $ in Thousands	3 Months Ended
	Sep. 30, 2023	Jun. 30, 2023	Mar. 31, 2023
Royalty Montizations [Roll Forward]
Balance	$ 526,121	$ 514,411	$ 501,655
Non-cash Interest expense on Royalty financing obligations	17,272	19,486	19,318
Royalty revenues paid and payable	(8,207)	(7,776)	(6,562)
Balance	535,186	526,121	514,411
2020 RPI Royalty Agreement
Royalty Montizations [Roll Forward]
Balance	170,649	168,252	164,981
Non-cash Interest expense on Royalty financing obligations	9,647	9,552	9,309
Royalty revenues paid and payable	(7,553)	(7,155)	(6,038)
Balance	$ 172,743	$ 170,649	168,252
Effective interest rate	22.10%	22.20%
2021 RPI Royalty Agreement
Royalty Montizations [Roll Forward]
Balance	$ 183,626	$ 178,807	173,651
Non-cash Interest expense on Royalty financing obligations	3,068	5,440	5,680
Royalty revenues paid and payable	(654)	(621)	(524)
Balance	$ 186,040	$ 183,626	178,807
Effective interest rate	0.00%	10.00%
OMERS Royalty Agreement
Royalty Montizations [Roll Forward]
Balance	$ 171,846	$ 167,352	163,023
Non-cash Interest expense on Royalty financing obligations	4,557	4,494	4,329
Royalty revenues paid and payable	0	0	0
Balance	$ 176,403	$ 171,846	$ 167,352
Effective interest rate	10.20%	10.60%

Revenue (Tables)

9 Months Ended

Sep. 30, 2023

Revenue from Contract with Customer [Abstract]

Summary of Revenues

The Company recorded the following revenues for the three and nine months ended September 30, 2023 and 2022:


	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Product sales, net	$	85,288	$	75,213	$	233,957	$	189,647
Collaborative and other revenues	1,454		614		4,054		1,635
Total revenues	$	86,742	$	75,827	$	238,011	$	191,282

The Company recorded the following revenues (in thousands):


	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
ORLADEYO:
U.S.	$	75,268	$	60,106	$	208,934	$	162,743
Outside of U.S.	10,416		5,866		26,173		18,155
Total ORLADEYO	85,684		65,972		235,107		180,898
Other revenues	1,058		9,855		2,904		10,384
Total revenues	$	86,742	$	75,827	$	238,011	$	191,282

Lease Obligations (Details Textual)	3 Months Ended		9 Months Ended
Lease Obligations (Details Textual)	Sep. 30, 2023 USD ($)	Sep. 30, 2022 USD ($)	Sep. 30, 2023 USD ($)	Sep. 30, 2022 USD ($)	Jul. 31, 2023 USD ($)	Dec. 31, 2022 USD ($) ft²	Jun. 30, 2021 ft²	Feb. 29, 2020 ft²	Feb. 28, 2015 ft²
Lessee, Lease, Description [Line Items]
Operating lease assets, net	$ 11,380,000		$ 11,380,000			$ 6,806,000
Operating lease, liability	12,248,000		12,248,000			8,173,000
Operating lease, right-of-use asset, accumulated amortization	6,350,000		6,350,000			$ 4,349,000
Operating lease, payments	$ 834,000	$ 615,000	$ 2,293,000	$ 1,796,000
NORTH CAROLINA \| Durham Office Space
Lessee, Lease, Description [Line Items]
Area of real estate \| ft²							19,978
Operating lease assets, net					$ 2,063
Operating lease, liability					2,063
ALABAMA \| Birmingham Office Space
Lessee, Lease, Description [Line Items]
Area of real estate \| ft²						14,607		2,846	31,601
Operating lease assets, net					5,327
Operating lease, liability					$ 5,925
Tenant improvement allowance						$ 515
Minimum
Lessee, Lease, Description [Line Items]
Lessee, operating lease, term of contract (in years)	1 year		1 year
Maximum
Lessee, Lease, Description [Line Items]
Lessee, operating lease, term of contract (in years)	3 years		3 years

Condensed Consolidated Statements of Comprehensive Loss - USD ($) shares in Thousands, $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
Revenues
Revenues	$ 86,742	$ 75,827	$ 238,011	$ 191,282
Expenses:
Cost of product sales	1,099	3,543	2,924	4,025
Research and development	46,879	52,740	146,514	180,090
Selling, general and administrative	50,648	36,919	149,512	109,218
Royalty	37	70	100	73
Total operating expenses	98,663	93,272	299,050	293,406
Loss from operations	(11,921)	(17,445)	(61,039)	(102,124)
Interest and other income	4,184	1,760	11,312	2,423
Interest expense	(27,345)	(24,775)	(83,656)	(72,634)
Foreign currency losses, net	(737)	(538)	(665)	(583)
Loss on extinguishment of debt	0	0	(29,019)	0
Loss before income taxes	(35,819)	(40,998)	(163,067)	(172,918)
Income tax expense	330	1,522	1,741	2,657
Net loss	(36,149)	(42,520)	(164,808)	(175,575)
Foreign currency translation adjustment	(185)	695	(67)	877
Unrealized gain (loss) on available for sale investments	295	(808)	841	(1,152)
Comprehensive loss	$ (36,039)	$ (42,633)	$ (164,034)	$ (175,850)
Basic net loss per common share (in usd per share)	$ (0.19)	$ (0.23)	$ (0.87)	$ (0.95)
Diluted net loss per common share (in usd per share)	$ (0.19)	$ (0.23)	$ (0.87)	$ (0.95)
Weighted average shares outstanding, basic (in shares)	189,644	186,180	189,095	185,566
Weighted average shares outstanding, diluted (in shares)	189,644	186,180	189,095	185,566

Trade Receivables - Summary of Receivables (Details) - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Accounts, Notes, Loans and Financing Receivable [Line Items]
Total receivables	$ 1,677	$ 8,268
Billed
Accounts, Notes, Loans and Financing Receivable [Line Items]
Total receivables	1,564	7,959
Unbilled
Accounts, Notes, Loans and Financing Receivable [Line Items]
Total receivables	113	309
U.S. Department of Health and Human Services, net
Accounts, Notes, Loans and Financing Receivable [Line Items]
Total receivables	113	7,502
U.S. Department of Health and Human Services, net \| Billed
Accounts, Notes, Loans and Financing Receivable [Line Items]
Total receivables	0	7,218
U.S. Department of Health and Human Services, net \| Unbilled
Accounts, Notes, Loans and Financing Receivable [Line Items]
Total receivables	113	284
Royalty receivables from partners
Accounts, Notes, Loans and Financing Receivable [Line Items]
Total receivables	1,564	741
Royalty receivables from partners \| Billed
Accounts, Notes, Loans and Financing Receivable [Line Items]
Total receivables	1,564	741
Royalty receivables from partners \| Unbilled
Accounts, Notes, Loans and Financing Receivable [Line Items]
Total receivables	$ 0	0
Other collaborations
Accounts, Notes, Loans and Financing Receivable [Line Items]
Total receivables		25
Other collaborations \| Billed
Accounts, Notes, Loans and Financing Receivable [Line Items]
Total receivables		0
Other collaborations \| Unbilled
Accounts, Notes, Loans and Financing Receivable [Line Items]
Total receivables		$ 25

Debt	9 Months Ended
Debt	Sep. 30, 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 and to pay associated transaction costs and fees, and intends to use 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 13.24% for the three months ended September 30, 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 September 30, 2023, the Company had total borrowings of $300,000 under the Pharmakon Loan Agreement. Total quarterly interest expense on the Tranche A Loan for the nine months ended September 30, 2023 totaled $17,350. As allowable under the Pharmakon Loan Agreement, the Company has designated and accounted for 50% of the quarterly interest payments for the nine months ended September 30, 2023 as a Pharmakon PIK Interest Payment and the amount of $8,675 has been added to the outstanding principal balance of the borrowing. The remaining 50% of the quarterly interest payments of $8,675 have been paid at the end of each quarterly period. As of September 30, 2023, borrowings, including the Pharmakon PIK Interest Payments, totaled $308,675. 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 $230 and $467 was recognized for the three and nine months ended September 30, 2023, respectively. 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 loans” 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 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; (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 monetization 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 12.68% during the period in which the debt was outstanding for the three months ended September 30, 2022. Subject to certain exceptions, the Athyrium Credit Agreement would have required the Company to make mandatory prepayments of the Athyrium Term Loans with the proceeds of certain asset sales, certain ORLADEYO out-licensing or royalty monetization transactions (excluding the Royalty Sales), extraordinary receipts, debt issuances, or upon a change of control of the Company and specified other events. The Company could have made voluntary prepayments in whole or in part. Prepayments were subject to a premium equal to, (i) with respect to any voluntary prepayment and certain mandatory prepayments paid on or prior to the second anniversary of the applicable Athyrium Term Loan borrowing date, the amount, if any, by which (a) the sum of (1) 102.00% of the principal amount of the Athyrium Term Loan being prepaid plus (2) the present value of all interest that would have accrued on the principal amount of the Athyrium Term Loan being prepaid through and including the second anniversary of the date of the borrowing of such Athyrium Term Loan, plus 0.50%, exceeds (b) the principal amount of the Athyrium Term Loan being prepaid; (ii) with respect to any prepayment made between the second and third anniversaries of the applicable Athyrium Term Loan borrowing date, 2.00% of the principal amount of the Athyrium Term Loan being prepaid; (iii) with respect to any prepayment made between the third and fourth anniversaries of the applicable Athyrium Term Loan borrowing date, 1.00% of the principal amount of the Athyrium Term Loan being prepaid; and (iv) with respect to any prepayment made after the fourth anniversary of the applicable Athyrium Term Loan borrowing date, 0.00% of the principal amount of the Athyrium Term Loan being prepaid. Upon the prepayment or repayment, including at maturity, 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. Quarterly interest payments under the Athyrium Credit Agreement for the nine months ended September 30, 2023 totaled $8,476. Quarterly interest payments under the Athyrium Credit Agreement for the nine months ended September 30, 2022 totaled $15,311 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 Loan 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 $597, was recognized for the nine months ended September 30, 2023 and 2022, 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 $1,355 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. Upon repayment of the Athyrium Term Loans, the Company incurred certain unaccrued prepayment and final payment fees to Athyrium of $17,261. 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 Condensed Consolidated Statements of Comprehensive Loss for the nine months ended September 30, 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

Reference 3: http://fasb.org/us-gaap/role/ref/legacyRef
-Topic 470
-Name Accounting Standards Codification
-Publisher FASB
-URI https://asc.fasb.org//470/tableOfContent

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

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1B
-Subparagraph (g)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481139/470-20-50-1B

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1B
-Subparagraph (i)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481139/470-20-50-1B

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1C
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481139/470-20-50-1C

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1C
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481139/470-20-50-1C

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1C
-Subparagraph (c)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481139/470-20-50-1C

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1E
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481139/470-20-50-1E

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1I
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481139/470-20-50-1I

Reference 12: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1I
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481139/470-20-50-1I

Reference 13: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 1I
-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481139/470-20-50-1I

+ Details

Revenue (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
Revenue (Details) - USD ($) $ in Thousands	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
Disaggregation of Revenue [Line Items]
Revenues	$ 86,742	$ 75,827	$ 238,011	$ 191,282
Product sales, net
Disaggregation of Revenue [Line Items]
Revenues	85,288	75,213	233,957	189,647
Product sales, net \| U.S.
Disaggregation of Revenue [Line Items]
Revenues	75,268	60,106	208,934	162,743
Product sales, net \| Outside of U.S.
Disaggregation of Revenue [Line Items]
Revenues	10,416	5,866	26,173	18,155
ORLADEYO
Disaggregation of Revenue [Line Items]
Revenues	85,684	65,972	235,107	180,898
Other revenues
Disaggregation of Revenue [Line Items]
Revenues	$ 1,058	$ 9,855	$ 2,904	$ 10,384

- 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 - Shares Reserved for Future Issuance (Details) - shares	Sep. 30, 2023	Dec. 31, 2022
Equity Disclosure [Line Items]
Total shares reserved for future issuance	72,360,000	67,238,000
Outstanding stock options
Equity Disclosure [Line Items]
Total shares reserved for future issuance	35,160,000	36,520,000
Restricted Stock Units (RSUs)
Equity Disclosure [Line Items]
Total shares reserved for future issuance	4,589,000	4,750,000
Warrants to purchase common stock
Equity Disclosure [Line Items]
Total shares reserved for future issuance	15,023,000	15,023,000
Incentive Plan
Equity Disclosure [Line Items]
Total shares reserved for future issuance	11,934,000	4,206,000
Inducement Plan
Equity Disclosure [Line Items]
Total shares reserved for future issuance	200,000	947,000
Employee Stock Purchase Plan
Equity Disclosure [Line Items]
Total shares reserved for future issuance	5,454,000	5,792,000