0001628280-24-021069 | 10-Q | XBRL Viewer | Biocryst Pharmaceuticals INC

Stockholders' Equity (Tables)

3 Months Ended

Mar. 31, 2024

Stockholders' Equity Note [Abstract]

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


	March 31, 2024	December 31, 2023
Shares reserved for exercises of outstanding stock options	39,580	41,032
Shares reserved for vesting of restricted stock units	6,124	6,507
Shares reserved for future issuance under the Stock Incentive Plan	4,628	3,376
Shares reserved for future issuance under the Inducement Equity Incentive Plan	1,903	1,651
Shares reserved for future issuance under the Employee Stock Purchase Plan	5,203	5,454
Total shares reserved for future issuance	57,438	58,020

- 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

Lease Obligations

3 Months Ended

Mar. 31, 2024

Leases [Abstract]

Lease Obligations

The Company leases certain assets under operating and finance leases, which consist of real estate leases, laboratory equipment leases and office equipment leases as of March 31, 2024. Renewal options for the Company’s leases are three years in length and begin from 2025 through 2030.

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


	Three Months Ended March 31,
	2024		2023
Operating lease expense	$	590	$	433
Finance lease expense:
Amortization of right-of-use assets	$	407	$	256
Interest on lease liabilities	77		38
Total finance lease expense	$	484	$	294

Other supplemental information related to leases was as follows:


	March 31, 2024		December 31, 2023
Weighted average remaining lease term:
Operating leases	9.4 years		9.7 years
Finance leases	2.8 years		3.0 years
Weighted average discount rate:
Operating leases	10.89	%	10.88	%
Finance leases	7.59	%	7.46	%

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


	Balance Sheet Location	March 31, 2024		December 31, 2023
Operating lease assets:
Operating lease assets, net	Other Assets	$	8,864	$	8,682
Operating lease liabilities:
Current operating lease liabilities	Operating lease liabilities – current liabilities	$	1,098	$	1,058
Non-current operating lease liabilities	Operating lease liabilities – long-term liabilities	8,596		8,390
Total operating lease liabilities		$	9,694	$	9,448

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


	Balance Sheet Location	March 31, 2024		December 31, 2023
Finance lease assets:
Finance lease assets, net	Other Assets	$	4,018	$	4,322
Finance lease liabilities:
Current finance lease liabilities	Finance lease liabilities – current liabilities	$	1,642	$	1,590
Non-current finance lease liabilities	Finance lease liabilities – long-term liabilities	2,506		2,845
Total finance lease liabilities		$	4,148	$	4,435

Operating lease assets are recorded net of accumulated amortization of $5,120 and $4,794 as of March 31, 2024 and December 31, 2023, respectively. Finance lease assets are recorded net of accumulated amortization of $2,700 and $2,293 as of March 31, 2024 and December 31, 2023, respectively.

Maturities of lease liabilities as of March 31, 2024 are as follows (in thousands):


	Operating Leases		Finance Leases
2024 (remaining)	$	1,589	$	1,418
2025	1,850		1,766
2026	1,661		933
2027	1,584		459
2028	1,416		31
Thereafter	7,997		—
Total lease payments	16,097		4,607
Less imputed interest	(6,403)		(459)
Total	$	9,694	$	4,148

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


	Three Months Ended March 31,
	2024		2023
Cash paid for amounts included in the measurement of lease liabilities:
Operating cash flows for finance leases	$	77	$	282
Operating cash flows for operating leases	$	525	$	423
Financing cash flows for finance leases	$	390	$	—

Operating lease assets obtained in exchange for operating lease liabilities:	$	156	$	95
Finance lease assets obtained in exchange for finance lease liabilities:	$	103	$	610
Non-cash increase to operating lease assets due to remeasurement of operating lease liabilities:	$	365	$	—

Investments - Fair Value, Recurring Basis (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Marketable Securities [Line Items]
Estimated Fair Value	$ 252,221	$ 278,344
Obligations of U.S. Government and its agencies
Marketable Securities [Line Items]
Estimated Fair Value	251,240	277,358
Certificates of deposit
Marketable Securities [Line Items]
Estimated Fair Value	981	986
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) \| 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	252,221	278,344
Significant Other Observable Inputs (Level 2) \| Obligations of U.S. Government and its agencies
Marketable Securities [Line Items]
Estimated Fair Value	251,240	277,358
Significant Other Observable Inputs (Level 2) \| Certificates of deposit
Marketable Securities [Line Items]
Estimated Fair Value	981	986
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) \| Certificates of deposit
Marketable Securities [Line Items]
Estimated Fair Value	$ 0	$ 0

Pay vs Performance Disclosure - USD ($) $ in Thousands	3 Months Ended
Pay vs Performance Disclosure - USD ($) $ in Thousands	Mar. 31, 2024	Mar. 31, 2023
Pay vs Performance Disclosure
Net loss	$ (35,379)	$ (53,333)

- Definition

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

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+ Details

Lease Obligations (Tables)

3 Months Ended

Mar. 31, 2024

Leases [Abstract]

Aggregate Lease Expense Under Operating Leases

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


	Three Months Ended March 31,
	2024		2023
Operating lease expense	$	590	$	433
Finance lease expense:
Amortization of right-of-use assets	$	407	$	256
Interest on lease liabilities	77		38
Total finance lease expense	$	484	$	294

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


	Three Months Ended March 31,
	2024		2023
Cash paid for amounts included in the measurement of lease liabilities:
Operating cash flows for finance leases	$	77	$	282
Operating cash flows for operating leases	$	525	$	423
Financing cash flows for finance leases	$	390	$	—

Operating lease assets obtained in exchange for operating lease liabilities:	$	156	$	95
Finance lease assets obtained in exchange for finance lease liabilities:	$	103	$	610
Non-cash increase to operating lease assets due to remeasurement of operating lease liabilities:	$	365	$	—

Other Supplemental Information Related to Leases

Other supplemental information related to leases was as follows:


	March 31, 2024		December 31, 2023
Weighted average remaining lease term:
Operating leases	9.4 years		9.7 years
Finance leases	2.8 years		3.0 years
Weighted average discount rate:
Operating leases	10.89	%	10.88	%
Finance leases	7.59	%	7.46	%

Balance Sheets of the Company's Operating Leases

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


	Balance Sheet Location	March 31, 2024		December 31, 2023
Operating lease assets:
Operating lease assets, net	Other Assets	$	8,864	$	8,682
Operating lease liabilities:
Current operating lease liabilities	Operating lease liabilities – current liabilities	$	1,098	$	1,058
Non-current operating lease liabilities	Operating lease liabilities – long-term liabilities	8,596		8,390
Total operating lease liabilities		$	9,694	$	9,448

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


	Balance Sheet Location	March 31, 2024		December 31, 2023
Finance lease assets:
Finance lease assets, net	Other Assets	$	4,018	$	4,322
Finance lease liabilities:
Current finance lease liabilities	Finance lease liabilities – current liabilities	$	1,642	$	1,590
Non-current finance lease liabilities	Finance lease liabilities – long-term liabilities	2,506		2,845
Total finance lease liabilities		$	4,148	$	4,435

Maturities of Operating Lease Liabilities

Maturities of lease liabilities as of March 31, 2024 are as follows (in thousands):


	Operating Leases		Finance Leases
2024 (remaining)	$	1,589	$	1,418
2025	1,850		1,766
2026	1,661		933
2027	1,584		459
2028	1,416		31
Thereafter	7,997		—
Total lease payments	16,097		4,607
Less imputed interest	(6,403)		(459)
Total	$	9,694	$	4,148

Trade Receivables (Details Textual) - Trade Accounts Receivable - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
ORLADEYO
Accounts, Notes, Loans and Financing Receivable [Line Items]
Accounts receivable, after allowance for credit loss, total	$ 56,404	$ 54,149
Outside of U.S.
Accounts, Notes, Loans and Financing Receivable [Line Items]
Accounts receivable, after allowance for credit loss, total	$ 604	$ 505

Condensed Consolidated Balance Sheets (Parenthetical) - $ / shares	Mar. 31, 2024	Dec. 31, 2023
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)	206,347,000	205,771,000
Common stock, shares, outstanding (in shares)	206,347,000	205,771,000

Investments (Tables)

3 Months Ended

Mar. 31, 2024

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):


	March 31, 2024
	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	$	—	$	251,240	$	—	$	251,240
Certificates of deposit	—		981		—		981
Total assets	$	—	$	252,221	$	—	$	252,221


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

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):


	March 31, 2024
	Amortized Cost		Accrued Interest		Gross Unrealized Gains		Gross Unrealized Losses		Estimated Fair Value
Obligations of U.S. Government and its agencies	$	251,425	$	45	$	1	$	(231)	$	251,240
Certificates of deposit	980		6		—		(5)		981
Total investments	$	252,405	$	51	$	1	$	(236)	$	252,221


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

Schedule of the Maturity of Investments

The following table summarizes the scheduled maturity for the Company’s investments at March 31, 2024 and December 31, 2023 (in thousands):


	March 31, 2024		December 31, 2023
Maturing in one year or less	$	252,221	$	278,344
Maturing after one year through two years	—		—
Total investments	$	252,221	$	278,344

- 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).

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- Definition

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Reference 1: http://www.xbrl.org/2009/role/commonPracticeRef
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+ Details

Royalty Financing Obligations

3 Months Ended

Mar. 31, 2024

Advance Royalties [Abstract]

Royalty Financing Obligations

ORLADEYO and Factor D Inhibitors

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

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

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

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

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

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

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

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

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

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

Under the Royalty Purchase Agreements, the Company has agreed to specified affirmative and negative covenants, including covenants regarding periodic reporting of information by the Company to RPI and OMERS, third-party audits of royalties paid under the Royalty Purchase Agreements, and restrictions on the ability of the Company or any of its subsidiaries to incur indebtedness other than certain royalty sales and as was permitted to be incurred under the terms of the Athyrium Credit Agreement (as defined in Note 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. On a quarterly basis, the Company assesses the projected royalty payments relative to the projected interest accretion for the next twelve months to determine if the royalty liability balance is reduced relative to the current outstanding liability, which would signify a repayment of the principal. In such case of excess payments relative to interest accretion for the next twelve months, the excess payments are considered to be a short-term liability and classified within current liabilities on the Company’s Condensed Consolidated Balance Sheets.

During the three months ended March 31, 2024, there was no significant impact on the amount and timing of expected royalties under the Royalty Purchase Agreements based on the Company’s latest forecasts related to its research and development programs and ORLADEYO sales.

The following table shows the royalty financing obligations activity for the three months ended March 31, 2024 (in thousands) as well as the effective interest rate as of March 31, 2024:


	2020 RPI Royalty Agreement			2021 RPI Royalty Agreement			OMERS Royalty Agreement			Total
Balance as of December 31, 2023	$	174,480		$	185,345		$	171,774		$	531,599
Non-cash Interest expense on Royalty financing obligations	9,916			—			4,271			14,187
Royalty revenues paid and payable	(7,842)			(679)			(6,691)			(15,212)
Balance as of March 31, 2024	$	176,554		$	184,666		$	169,354		$	530,574

Effective interest rate	22.3		%	—		%	9.8		%

The Royalty financing obligations liabilities and the associated deferred issuance costs are amortized using the effective interest method over the term of the arrangement.

Concurrent with entering into the 2021 RPI Royalty Purchase Agreement, the Company and RPI entered into a Common Stock Purchase Agreement 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.

Significant Accounting Policies and Concentrations of Risk (Policies)

3 Months Ended

Mar. 31, 2024

Accounting Policies [Abstract]

The Company

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

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

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

Basis of Presentation

The 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. Certain prior year amounts have been reclassified to conform to the current year presentation.

These financial statements should be read in conjunction with the financial statements for the year ended December 31, 2023 and the notes thereto included in the Company’s 2023 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, 2023 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 months ended March 31, 2024 and 2023 (in thousands):


	Three Months Ended March 31,
	2024		2023
Product sales, net	$	89,272	$	68,166
Collaborative and other revenues	3,489		612
Total revenues	$	92,761	$	68,778

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, and sales of peramivir. In the United States, the Company ships ORLADEYO directly to patients through a single specialty pharmacy, which is considered its customer. In the European Union, United Kingdom and elsewhere, the Company sells ORLADEYO to specialty distributors as well as hospitals and pharmacies, which collectively are considered its customers.

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

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

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

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

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

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

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

Collaborative and Other Revenues

The Company has collaboration and license agreements with a number of third parties. 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.

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

Cash and Cash Equivalents

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

Restricted Cash

Total restricted cash was $1,798 and $1,804 as of March 31, 2024 and December 31, 2023, respectively, and primarily consisted of $1,501 and $1,493 as of March 31, 2024 and December 31, 2023, respectively, for a letter of credit the Company is required to maintain associated with its Birmingham lease.

Investments

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

The Company classifies all of its investments as available-for-sale. Available-for-sale investments are reported at fair value at each balance sheet date, and include any unrealized holding gains and losses in accumulated other comprehensive income, unless an unrealized loss is considered to be other than temporary, in which case the unrealized loss is charged to operations. The Company reviews its investments for other than temporary declines in fair value below cost basis at the end of each reporting period and whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. Factors considered to determine whether an unrealized loss is temporary include whether a decline in fair value below the amortized cost basis is due to credit-related factors or non-credit-related factors, the financial condition and near-term prospects of the Company, and the Company’s intent and ability to hold the investment to allow for an anticipated recovery in fair value. A credit-related impairment is recognized as an allowance on the balance sheet with a corresponding adjustment to earnings. Any impairment that is not credit-related is recognized in other comprehensive income, net of applicable taxes unless deemed other than temporary. Realized gains and losses are reflected in interest and other income in the 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.

Fair Value Measurements

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

Assets measured at fair value on a recurring basis include investments (Note 3). There were no liabilities measured at fair value on a recurring basis as of March 31, 2024 and December 31, 2023. The carrying amounts reflected in the Condensed Consolidated Balance Sheets for cash and cash equivalents, trade receivables, prepaid expenses and other current assets, accounts payable and accrued expenses approximate their fair values due to the short-term nature of these assets and liabilities.

Trade Receivables

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

Receivables from collaborations are recorded for amounts due to the Company related to royalty receivables from the Company’s partners, including Shionogi & Co., Ltd., Green Cross, and Torii (Note 11).

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

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

Property and Equipment

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

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

Accrued Expenses

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

The Company bases its expenses related to clinical trials on its estimates of the services received and efforts expended pursuant to contracts with multiple research institutions and CROs that conduct and manage clinical trials on the Company’s behalf. The financial terms of these agreements are subject to negotiation, vary from contract to contract and may result in uneven payment flows. Payments under some of these contracts depend on factors such as the successful enrollment of patients and the completion of clinical trial milestones. In accruing service fees, the Company estimates the time period over which services will be performed and the level of effort expended in each period. If the actual timing of the performance of services or the level of effort varies from the estimate, the Company will adjust the accrual accordingly. If the Company underestimates or overestimates the level of these costs, actual expenses could differ from such estimates. As of March 31, 2024 and December 31, 2023, the carrying value of accrued expenses approximates their fair value due to their short-term settlement.

Cost of Product Sales

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

Research and Development Expenses

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

Company over the service periods specified in the contracts, and estimates are adjusted based upon the Company’s ongoing review of the level of services actually performed.

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

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

Selling, General and Administrative Expenses

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

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

Leases

The Company leases certain assets, under operating and finance leases, which consist of real estate leases, laboratory equipment leases and office equipment leases as of March 31, 2024. 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 Company reduces stock-based compensation expense for estimated forfeitures. The estimation of share-based payment awards that will ultimately vest requires judgment, and to the extent actual results or updated estimates differ from the Company’s current estimates, such

amounts will be recorded as a cumulative adjustment in the period estimates are revised. In addition, the Company has outstanding performance-based stock options and restricted stock units for which no compensation expense is recognized until “performance” is deemed to have occurred. Significant management judgment is also required in determining estimates of future stock price volatility to be used in the valuation of the options. Actual results, and future changes in estimates, may differ substantially from the Company’s current estimates.

Interest Expense, Deferred Financing Costs and Royalty Financing Obligations

Interest expense primarily relates to the royalty financing obligations (Note 6) and the term loan borrowings under the Pharmakon Loan Agreement (Note 7) during the three months ended March 31, 2024 and to the secured term loan borrowings under the Athyrium Credit Agreement (Note 7) during the three months ended March 31, 2023.

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. When utilizing the effective interest method, in periods in which payment-in-kind (“PIK”) interest was designated and added to the outstanding principal balance of the borrowing, the amortization of the deferred debt fees and issuance costs was accretive.

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 fifteen in the case of development conducted on foreign soil.

Foreign Currency

The functional currency of each of the Company’s foreign subsidiaries is primarily the local currency of the country in which the subsidiary operates. The Company’s asset and liability accounts are translated at the current exchange rate as of the balance sheet date. Revenue and expense accounts are translated at the average exchange rate over the period.

Adjustments resulting from the translation of the financial statements of the Company’s foreign subsidiaries into U.S. dollars are accumulated as a separate component of stockholders’ equity within accumulated other comprehensive income. Gains or losses resulting from transactions denominated in foreign currencies are included in foreign currency losses, net, within the Condensed Consolidated Statement of Comprehensive Loss.

Net Loss Per Share

Basic net loss per share is based upon the weighted average number of common shares outstanding during the period. Diluted net loss per share is computed by dividing net loss by the weighted average number of common shares outstanding during the period, after giving consideration to the dilutive effect of potentially dilutive common shares. The Company has generated a net loss in all periods presented, so the diluted net loss per share is equivalent to basic net loss per share for all periods presented herein because common equivalent shares from unexercised stock options, warrants and common shares expected to be issued under the Company’s equity compensation plans would be anti-dilutive. The Company excluded the following potential common shares, presented based on amounts outstanding as of March 31, 2024 and March 31, 2023, from the computation of diluted net loss per share attributable to common stockholders because including them would have had an anti-dilutive effect:


	March 31,
	2024	2023
Outstanding stock options	39,580	35,858
Unvested restricted stock unit awards	6,124	4,654
Warrants to purchase common stock	—	15,023
Total	45,704	55,535

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 months ended March 31, 2024 and 2023.

Significant Customers and Other Risks

Significant Customers

The Company’s primary source of revenue and cash flow are the sales of ORLADEYO in the United States and other global markets.

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.

Recently Adopted Accounting Pronouncements And New Accounting Pronouncements Not Yet Adopted

Recently Adopted Accounting Pronouncements

There have been no new accounting pronouncements adopted by the Company during the three months ended March 31, 2024.

New Accounting Pronouncements Not Yet Adopted

In December 2023, the Financial Accounting Standards Board issued Accounting Standards Update (“ASU”) 2023-09, Income Taxes (Topic 740): Improvements to Income Tax Disclosures, which requires public entities, on an annual basis, to provide disclosure of specific categories in the rate reconciliation, as well as disclosure of income taxes paid disaggregated by jurisdiction. ASU 2023-09 is effective for fiscal years beginning after December 15, 2024, with early adoption permitted. The Company is currently evaluating the impact of adopting ASU 2023-09.

Stock-based Compensation (Tables)

3 Months Ended

Mar. 31, 2024

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):


	Three Months Ended March 31,
	2024		2023
Incentive Plan	$	11,566	$	11,061
Inducement Plan	1,923		2,475
ESPP	163		471
Stock-based compensation expense	$	13,652	$	14,007

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


	Shares (in thousands)	Weighted Average Exercise Price per Share		Weighted Average Remaining Contractual Term (in years)	Aggregate Intrinsic Value (in thousands)
Outstanding at December 31, 2023	35,505	$	8.24
Granted	—	—
Exercised	(48)	3.27			$	103
Cancelled or Forfeited	(1,068)	9.60
Outstanding at March 31, 2024	34,389	$	8.20	6.61	$	6,951
Exercisable at March 31, 2024	20,922	$	7.88	5.30	$	6,951
Vested and expected to vest at March 31, 2024	31,784	$	8.13	6.49	$	6,951

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


	Shares (in thousands)	Weighted Average Exercise Price per Share		Weighted Average Remaining Contractual Term (in years)	Aggregate Intrinsic Value (in thousands)
Outstanding at December 31, 2023	5,527	$	8.77
Granted	92	5.25
Exercised	(128)	3.09			$	274
Cancelled or Forfeited	(300)	10.86
Outstanding at March 31, 2024	5,191	$	8.73	7.53	$	2,454
Exercisable at March 31, 2024	2,628	$	7.53	6.76	$	2,128
Vested and expected to vest at March 31, 2024	4,684	$	8.59	7.46	$	2,373

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

Stock Incentive Plan

There were no stock option awards granted under the Incentive Plan during the three months ended March 31, 2024. The following table summarizes the key assumptions used by the Company to value the stock option awards granted under the Incentive Plan during the three months ended March 31, 2023:


	Three Months Ended March 31, 2023
Expected Life in Years	5.5
Expected Volatility	84.2		%
Expected Dividend Yield	0.0		%
Risk-Free Interest Rate	3.6		%
Weighted average grant date fair value per share	$	7.47

Inducement Equity Incentive Plan

The following table summarizes the key assumptions used by the Company to value the stock option awards granted under the Inducement Plan during the three months ended March 31, 2024 and 2023:


	Three Months Ended March 31,
	2024			2023
Expected Life in Years	5.8			5.5
Expected Volatility	82.8		%	84.2		%
Expected Dividend Yield	0.0		%	0.0		%
Risk-Free Interest Rate	4.1		%	3.7		%
Weighted average grant date fair value per share	$	3.77		$	6.85

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

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


	Shares (in thousands)	Weighted Average Grant Date Fair Value
Unvested at December 31, 2023	5,592	$	8.67
Granted	128	5.30
Vested	(96)	12.30
Forfeited	(313)	8.65
Unvested at March 31, 2024	5,311	$	8.52

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


	Shares (in thousands)	Weighted Average Grant Date Fair Value
Unvested at December 31, 2023	915	$	9.90
Granted	63	5.25
Vested	(59)	11.96
Forfeited	(106)	10.41
Unvested at March 31, 2024	813	$	9.32

Stock-based Compensation (Details Textual) $ / shares in Units, $ in Thousands	3 Months Ended
	Mar. 31, 2024 USD ($) plan $ / shares shares	Mar. 31, 2023 $ / shares shares
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Number of plans \| plan	3
Employee stock purchase plan sales, net (in shares) \| shares	251,000	176,000
Incentive Plan
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Total unrecognized compensation expense related to non-vested stock options and RSU awards \| $	$ 62,593
Weighted-average term to recognize non-vested stock options and RSU awards	1 year 7 months 6 days
Performance-based stock options issued in period (in shares) \| shares	0
Incentive Plan \| Unvested restricted stock unit awards
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Restricted stock units, grants in period, weighted average grant date fair value (in dollars per share) \| $ / shares	$ 5.30	$ 10.32
Total unrecognized compensation expense related to non-vested stock options and RSU awards \| $	$ 33,457
Weighted-average term to recognize non-vested stock options and RSU awards	1 year 9 months 18 days
Inducement Plan
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Total unrecognized compensation expense related to non-vested stock options and RSU awards \| $	$ 12,326
Weighted-average term to recognize non-vested stock options and RSU awards	1 year 4 months 24 days
Inducement Plan \| Unvested restricted stock unit awards
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Restricted stock units, grants in period, weighted average grant date fair value (in dollars per share) \| $ / shares	$ 5.25	$ 9.64
Total unrecognized compensation expense related to non-vested stock options and RSU awards \| $	$ 5,588
Weighted-average term to recognize non-vested stock options and RSU awards	1 year 8 months 12 days
ESPP
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Number of shares of common stock authorized to be purchased (in shares) \| shares	7,975,000
Number of common stock shares available for purchase (in shares) \| shares	5,203,000

Trade Receivables

3 Months Ended

Mar. 31, 2024

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 March 31, 2024 and December 31, 2023, receivables, net of reserves, related to sales of ORLADEYO were $56,404 and $54,149, respectively. At March 31, 2024 and December 31, 2023, receivables related to sales of RAPIVAB were $604 and $505, respectively.

Collaborations

Receivables from collaborations were as follows (in thousands):


	March 31, 2024
	Billed		Unbilled		Total
Royalty receivables from partners	$	86	$	3,492	$	3,578
Total receivables from collaborators	$	86	$	3,492	$	3,578


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

As of March 31, 2024 and December 31, 2023,

Significant Accounting Policies and Concentrations of Risk

3 Months Ended

Mar. 31, 2024

Accounting Policies [Abstract]

Significant Accounting Policies and Concentrations of Risk

The Company

Basis of Presentation

Use of Estimates

Revenue Recognition

The Company recorded the following revenues for the three months ended March 31, 2024 and 2023 (in thousands):


	Three Months Ended March 31,
	2024		2023
Product sales, net	$	89,272	$	68,166
Collaborative and other revenues	3,489		612
Total revenues	$	92,761	$	68,778

Product Sales, Net

Collaborative and Other Revenues

Cash and Cash Equivalents

Restricted Cash

Investments

sheet date are classified as current. Investments with a maturity beyond 12 months from the balance sheet date are classified as long-term.

Fair Value Measurements

Trade Receivables

Inventory

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

Property and Equipment

Accrued Expenses

Cost of Product Sales

Research and Development Expenses

Company over the service periods specified in the contracts, and estimates are adjusted based upon the Company’s ongoing review of the level of services actually performed.

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 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, Deferred Financing Costs and Royalty Financing Obligations

Income Taxes

Foreign Currency

Net Loss Per Share


	March 31,
	2024	2023
Outstanding stock options	39,580	35,858
Unvested restricted stock unit awards	6,124	4,654
Warrants to purchase common stock	—	15,023
Total	45,704	55,535

Accumulated Other Comprehensive Income

Significant Customers and Other Risks

Significant Customers

The Company’s primary source of revenue and cash flow are the sales of ORLADEYO in the United States and other global markets.

Risks from Third-Party Manufacturing and Distribution Concentration

Credit Risk

Recently Adopted Accounting Pronouncements

There have been no new accounting pronouncements adopted by the Company during the three months ended March 31, 2024.

New Accounting Pronouncements Not Yet Adopted

Significant Accounting Policies and Concentrations of Risk (Details) - USD ($) shares in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023	Dec. 31, 2023
Significant Accounting Policies and Concentration of Risk [Line Items]
Restricted cash	$ 1,798,000		$ 1,804,000
Long-term investment maturity, minimum (in years)	12 months
Antidilutive securities excluded from computation of earnings per share, amount (in shares)	45,704	55,535
Reclassification from accumulated other comprehensive income, current period, before tax	$ 0	$ 0
Computer Equipment
Significant Accounting Policies and Concentration of Risk [Line Items]
Property, plant and equipment, useful life (in 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
Furniture and Fixtures
Significant Accounting Policies and Concentration of Risk [Line Items]
Property, plant and equipment, useful life (in years)	5 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
Collateral for Credit
Significant Accounting Policies and Concentration of Risk [Line Items]
Restricted cash	$ 1,501,000		$ 1,493,000

Stock-based Compensation - Restricted Stock Unit Activity (Details) - Unvested restricted stock unit awards - $ / shares shares in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Incentive Plan
Share-Based Compensation Arrangement by Share-Based Payment Award, Equity Instruments Other than Options, Nonvested, Number of Shares [Roll Forward]
Unvested RSU beginning balance (in shares)	5,592
Issued RSU (in shares)	128
Vested RSU (in shares)	(96)
Forfeited RSU (in shares)	(313)
Unvested RSU ending balance (in shares)	5,311
Share-Based Compensation Arrangement by Share-Based Payment Award, Equity Instruments Other than Options, Nonvested, Weighted Average Grant Date Fair Value [Abstract]
Nonvested RSU weighted average grant date fair value beginning balance (in dollars per share)	$ 8.67
Restricted stock units, grants in period, weighted average grant date fair value (in dollars per share)	5.30	$ 10.32
Restricted stock units, vested in period, weighted average grant date fair value (in dollars per share)	12.30
Restricted stock units, forfeited in period, weighted average grant date fair value (in dollars per share)	8.65
Nonvested RSU weighted average grant date fair value ending balance (in dollars per share)	$ 8.52
Inducement Plan
Share-Based Compensation Arrangement by Share-Based Payment Award, Equity Instruments Other than Options, Nonvested, Number of Shares [Roll Forward]
Unvested RSU beginning balance (in shares)	915
Issued RSU (in shares)	63
Vested RSU (in shares)	(59)
Forfeited RSU (in shares)	(106)
Unvested RSU ending balance (in shares)	813
Share-Based Compensation Arrangement by Share-Based Payment Award, Equity Instruments Other than Options, Nonvested, Weighted Average Grant Date Fair Value [Abstract]
Nonvested RSU weighted average grant date fair value beginning balance (in dollars per share)	$ 9.90
Restricted stock units, grants in period, weighted average grant date fair value (in dollars per share)	5.25	$ 9.64
Restricted stock units, vested in period, weighted average grant date fair value (in dollars per share)	11.96
Restricted stock units, forfeited in period, weighted average grant date fair value (in dollars per share)	10.41
Nonvested RSU weighted average grant date fair value ending balance (in dollars per share)	$ 9.32

Condensed Consolidated Balance Sheets - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Assets
Cash and cash equivalents	$ 84,333	$ 110,643
Restricted cash	1,798	1,804
Investments	252,221	278,344
Trade receivables	60,586	56,950
Inventory, net	29,781	28,683
Prepaid expenses and other current assets	18,381	19,542
Total current assets	447,100	495,966
Property and equipment, net	7,831	7,910
Other assets	12,961	13,084
Total assets	467,892	516,960
Liabilities and Stockholders’ Deficit
Accounts payable	11,910	20,893
Accrued expenses	79,249	102,882
Operating lease liabilities	1,098	1,058
Finance lease liabilities	1,642	1,590
Royalty financing obligations	26,022	23,565
Total current liabilities	119,921	149,988
Operating lease liabilities	8,596	8,390
Finance lease liabilities	2,506	2,845
Royalty financing obligations	504,552	508,034
Secured term loan	308,484	303,231
Stockholders’ deficit:
Preferred stock, $0.01 par value; shares authorized - 5,000; no shares issued and outstanding at March 31, 2024 and December 31, 2023	0	0
Common stock, $0.01 par value; shares authorized - 450,000; shares issued and outstanding – 206,347 at March 31, 2024 and 205,771 at December 31, 2023	2,063	2,058
Additional paid-in capital	1,237,537	1,222,236
Accumulated other comprehensive income	771	1,337
Accumulated deficit	(1,716,538)	(1,681,159)
Total stockholders’ deficit	(476,167)	(455,528)
Total liabilities and stockholders’ deficit	$ 467,892	$ 516,960

Condensed Consolidated Statements of Comprehensive Loss - USD ($) shares in Thousands, $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Revenues
Revenues	$ 92,761	$ 68,778
Expenses:
Cost of product sales	1,265	931
Research and development	46,493	48,388
Selling, general and administrative	59,364	47,867
Royalty	127	7
Total operating expenses	107,249	97,193
Loss from operations	(14,488)	(28,415)
Interest income	4,031	3,378
Interest expense	(24,506)	(27,396)
Foreign currency losses, net	(51)	(229)
Loss before income taxes	(35,014)	(52,662)
Income tax expense	365	671
Net loss	(35,379)	(53,333)
Foreign currency translation adjustment	(253)	59
Unrealized (loss) gain on available for sale investments	(313)	500
Net comprehensive loss	$ (35,945)	$ (52,774)
Basic net loss per common share (in usd per share)	$ (0.17)	$ (0.28)
Diluted net loss per common share (in usd per share)	$ (0.17)	$ (0.28)
Weighted average shares outstanding, basic (in shares)	206,064	188,509
Weighted average shares outstanding, diluted (in shares)	206,064	188,509

Lease Obligations - Maturities of Lease Liabilities (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Lessee, Operating Lease, Liability, to be Paid, Fiscal Year Maturity [Abstract]
2024 (remaining)	$ 1,589
2025	1,850
2026	1,661
2027	1,584
2028	1,416
Thereafter	7,997
Total lease payments	16,097
Less imputed interest	(6,403)
Total	9,694	$ 9,448
Finance Lease, Liability, to be Paid, Fiscal Year Maturity [Abstract]
2024 (remaining)	1,418
2025	1,766
2026	933
2027	459
2028	31
Thereafter	0
Total lease payments	4,607
Less imputed interest	(459)
Total	$ 4,148	$ 4,435

Subsequent Event	3 Months Ended
Subsequent Event	Mar. 31, 2024
Subsequent Events [Abstract]
Subsequent Event	Subsequent Events The Company considers events or transactions that occur after the balance sheet date but prior to the issuance of the condensed consolidated financial statements to provide additional evidence for certain estimates or to identify matters that require additional disclosure. The Company has concluded that no subsequent events have occurred that require disclosure.

Lease Obligations - Supplemental Cash Flows (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Leases [Abstract]
Operating cash flows for finance leases	$ 77	$ 282
Operating cash flows for operating leases	525	423
Financing cash flows for finance leases	390	0
Operating lease assets obtained in exchange for operating lease liabilities:	156	95
Finance lease assets obtained in exchange for finance lease liabilities:	103	610
Non-cash increase to operating lease assets due to remeasurement of operating lease liabilities:	$ 365	$ 0

Investments

3 Months Ended

Mar. 31, 2024

Investments [Abstract]

Investments

Fair value is an exit price, representing the amount that would be received from the sale of an asset or paid to transfer a liability in an orderly transaction between market participants. Fair value is determined based on observable and unobservable inputs. Observable inputs reflect readily obtainable data from independent sources, while unobservable inputs reflect certain market assumptions. As a basis for considering such assumptions, U.S. GAAP establishes a three-tier value hierarchy, which prioritizes the inputs used to develop the assumptions and for measuring fair value as follows: (Level 1) observable inputs such as quoted prices in active markets for identical assets; (Level 2) inputs other than the quoted prices in active markets that are observable either directly or indirectly; and (Level 3) unobservable inputs for which there is little

or no market data, which requires the Company to develop its own assumptions. This hierarchy requires the Company to use observable market data, when available, and to minimize the use of unobservable inputs when determining fair value.

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

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


	March 31, 2024
	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	$	—	$	251,240	$	—	$	251,240
Certificates of deposit	—		981		—		981
Total assets	$	—	$	252,221	$	—	$	252,221


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

As of March 31, 2024, the Company had 14 securities with a total estimated fair market value of $171,437 in an unrealized loss position. The Company believes the individual unrealized losses represent temporary declines primarily resulting from interest rate changes. The Company does not have intent to sell these investments, and it is more likely than not that the investments will be held until recovery of their amortized cost basis. As such, no allowance was recognized.

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


	March 31, 2024
	Amortized Cost		Accrued Interest		Gross Unrealized Gains		Gross Unrealized Losses		Estimated Fair Value
Obligations of U.S. Government and its agencies	$	251,425	$	45	$	1	$	(231)	$	251,240
Certificates of deposit	980		6		—		(5)		981
Total investments	$	252,405	$	51	$	1	$	(236)	$	252,221


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

The following table summarizes the scheduled maturity for the Company’s investments at March 31, 2024 and December 31, 2023 (in thousands):


	March 31, 2024		December 31, 2023
Maturing in one year or less	$	252,221	$	278,344
Maturing after one year through two years	—		—
Total investments	$	252,221	$	278,344

Royalty Financing Obligations (Tables)

3 Months Ended

Mar. 31, 2024

Advance Royalties [Abstract]

Schedule of Royalty Financing Obligations

The following table shows the royalty financing obligations activity for the three months ended March 31, 2024 (in thousands) as well as the effective interest rate as of March 31, 2024:


	2020 RPI Royalty Agreement			2021 RPI Royalty Agreement			OMERS Royalty Agreement			Total
Balance as of December 31, 2023	$	174,480		$	185,345		$	171,774		$	531,599
Non-cash Interest expense on Royalty financing obligations	9,916			—			4,271			14,187
Royalty revenues paid and payable	(7,842)			(679)			(6,691)			(15,212)
Balance as of March 31, 2024	$	176,554		$	184,666		$	169,354		$	530,574

Effective interest rate	22.3		%	—		%	9.8		%

Workforce Reduction

3 Months Ended

Mar. 31, 2024

Restructuring and Related Activities [Abstract]

Workforce Reduction

In January 2024, the Company announced a reduction of workforce. The majority of the impacted employees had a termination date in January 2024, with certain employees exiting later in 2024. The Company notified the impacted employees in January 2024.

The Company incurred costs related to employee severance, benefits, and related costs which were accounted for as ongoing terminations benefits under ASC Topic 712, Nonretirement Postemployment Benefits. As of December 31, 2023, it was considered probable that payment would be owed and the amount of payment was considered to be reasonably estimable, which resulted in the recognition of $3,380 of costs related to the workforce reduction during the year ended December 31, 2023, of which $3,026 was recognized in research and development expenses and $354 was recognized in selling, general and administrative expenses in the Consolidated Statement of Comprehensive Loss. As of March 31, 2024, $1,904 of the costs were paid, and the remaining $1,476 are included within accrued expenses on the Condensed Consolidated Balance Sheet. The remaining unpaid costs are expected to be disbursed during the period of April 1, 2024 through December 31, 2024.

In addition, the employees impacted by the workforce reduction received an amount equal to the bonus amount the employee would have received through continued employment with the Company, which was considered a one-time termination benefit pursuant to ASC Topic 420, Exit or Disposal Costs. As a result, $1,264 has been recognized during the three months ended March 31, 2024, the period in which the communication occurred, of which $1,201 has been recognized in research and development expenses, and $63 has been recognized in selling, general and administrative expenses in the Condensed Consolidated Statement of Comprehensive Loss. All of these costs were paid during the three months ended March 31, 2024.

The following table summarizes the accrued liability activity recorded in connection with the workforce reduction for the three months ended March 31, 2024 (in thousands):


Balance at December 31, 2023	$	3,380
Workforce reduction expense recorded during the three months ended March 31, 2024	1,264
Amounts paid during the three months ended March 31, 2024	(3,168)
Balance at March 31, 2024	$	1,476

The Company does not expect to incur any additional significant costs related to this workforce reduction.

Investments - Narrative (Details) $ in Thousands	Mar. 31, 2024 USD ($) security
Investments, All Other Investments [Abstract]
Number of securities \| security	14
Estimated fair market value of securities \| $	$ 171,437

Workforce Reduction (Details) - USD ($) $ in Thousands	3 Months Ended	12 Months Ended
Workforce Reduction (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Restructuring Cost and Reserve [Line Items]
Restructuring costs	$ 1,264
Restructuring reserve	1,476	$ 3,380
Employee Severance
Restructuring Cost and Reserve [Line Items]
Restructuring costs		3,380
Payments for restructuring, net	1,904
Restructuring reserve	1,476
Employee Severance \| Research and Developement
Restructuring Cost and Reserve [Line Items]
Restructuring costs		3,026
Employee Severance \| Selling, General and Administrative Expenses
Restructuring Cost and Reserve [Line Items]
Restructuring costs		$ 354
One-time Termination Benefits
Restructuring Cost and Reserve [Line Items]
Restructuring costs	1,264
One-time Termination Benefits \| Research and Developement
Restructuring Cost and Reserve [Line Items]
Restructuring costs	1,201
One-time Termination Benefits \| Selling, General and Administrative Expenses
Restructuring Cost and Reserve [Line Items]
Restructuring costs	$ 63

- 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

Lease Obligations - Aggregate Lease Expense Under Operating Leases (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Leases [Abstract]
Operating lease expense	$ 590	$ 433
Amortization of right-of-use assets	407	256
Interest on lease liabilities	77	38
Total finance lease expense	$ 484	$ 294

Investments- Fair Value of the Company's Investments by Type (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Marketable Securities [Line Items]
Amortized Cost	$ 252,405	$ 278,131
Accrued Interest	51	135
Gross Unrealized Gains	1	150
Gross Unrealized Losses	(236)	(72)
Estimated Fair Value	$ 252,221	$ 278,344
Debt Securities, Available-for-Sale, Accrued Interest, after Allowance for Credit Loss, Statement of Financial Position [Extensible Enumeration]	Investments	Investments
Obligations of U.S. Government and its agencies
Marketable Securities [Line Items]
Amortized Cost	$ 251,425	$ 277,151
Accrued Interest	45	121
Gross Unrealized Gains	1	150
Gross Unrealized Losses	(231)	(64)
Estimated Fair Value	251,240	277,358
Certificates of deposit
Marketable Securities [Line Items]
Amortized Cost	980	980
Accrued Interest	6	14
Gross Unrealized Gains	0	0
Gross Unrealized Losses	(5)	(8)
Estimated Fair Value	$ 981	$ 986

Workforce Reduction (Tables)

3 Months Ended

Mar. 31, 2024

Restructuring and Related Activities [Abstract]

Restructuring and Related Costs

The following table summarizes the accrued liability activity recorded in connection with the workforce reduction for the three months ended March 31, 2024 (in thousands):


Balance at December 31, 2023	$	3,380
Workforce reduction expense recorded during the three months ended March 31, 2024	1,264
Amounts paid during the three months ended March 31, 2024	(3,168)
Balance at March 31, 2024	$	1,476

Trade Receivables (Tables)

3 Months Ended

Mar. 31, 2024

Receivables [Abstract]

Summary of Receivables

Receivables from collaborations were as follows (in thousands):


	March 31, 2024
	Billed		Unbilled		Total
Royalty receivables from partners	$	86	$	3,492	$	3,578
Total receivables from collaborators	$	86	$	3,492	$	3,578


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

Lease Obligations (Details Textual) - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Leases [Abstract]
Lessee, operating lease, term of contract (in years)	3 years
Operating lease, right-of-use asset, accumulated amortization	$ 5,120	$ 4,794
Finance lease, right-of-use asset, accumulated amortization	$ 2,700	$ 2,293

Investments - Schedule of Maturity of Investments (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Investments [Abstract]
Maturing in one year or less	$ 252,221	$ 278,344
Maturing after one year through two years	0	0
Estimated Fair Value	$ 252,221	$ 278,344

Stockholders' Equity

3 Months Ended

Mar. 31, 2024

Stockholders' Equity Note [Abstract]

Stockholders' Equity

Stockholders’ Equity

Sales of Common Stock

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

Shares Reserved for Future Issuance of Common Stock

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


	March 31, 2024	December 31, 2023
Shares reserved for exercises of outstanding stock options	39,580	41,032
Shares reserved for vesting of restricted stock units	6,124	6,507
Shares reserved for future issuance under the Stock Incentive Plan	4,628	3,376
Shares reserved for future issuance under the Inducement Equity Incentive Plan	1,903	1,651
Shares reserved for future issuance under the Employee Stock Purchase Plan	5,203	5,454
Total shares reserved for future issuance	57,438	58,020

- Definition

The entire disclosure for equity.

+ References

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

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

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

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
-SubTopic 235
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481062/946-235-50-2

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
-SubTopic 235
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481062/946-235-50-2

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Topic 946
-SubTopic 505
-Name Accounting Standards Codification
-Section 50
-Paragraph 6
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481004/946-505-50-6

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 815
-SubTopic 40
-Name Accounting Standards Codification
-Section 50
-Paragraph 6
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480237/815-40-50-6

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.3-04)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480008/505-10-S99-1

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

Reference 10: http://fasb.org/us-gaap/role/ref/legacyRef
-Topic 505
-Name Accounting Standards Codification
-Publisher FASB
-URI https://asc.fasb.org//505/tableOfContent

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 13
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481112/505-10-50-13

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

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

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

Reference 15: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 14
-Subparagraph (c)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147481112/505-10-50-14

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

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

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

Significant Accounting Policies and Concentrations of Risk - Net Loss Per Share (Details) - shares shares in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Significant Accounting Policies and Concentration of Risk [Line Items]
Antidilutive securities excluded from computation of earnings per share, amount (in shares)	45,704	55,535
Outstanding stock options
Significant Accounting Policies and Concentration of Risk [Line Items]
Antidilutive securities excluded from computation of earnings per share, amount (in shares)	39,580	35,858
Unvested restricted stock unit awards
Significant Accounting Policies and Concentration of Risk [Line Items]
Antidilutive securities excluded from computation of earnings per share, amount (in shares)	6,124	4,654
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)	0	15,023

Stock-based Compensation - Option Activity (Details) $ / shares in Units, $ in Thousands	3 Months Ended
	Mar. 31, 2024 USD ($) $ / shares shares
Incentive Plan
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Outstanding [Roll Forward]
Options, grants in period, gross (in shares)	0
Outstanding stock options \| Incentive Plan
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Outstanding [Roll Forward]
Options outstanding, beginning balance (in shares)	35,505,000
Options, grants in period, gross (in shares)	0
Options exercised (in shares)	(48,000)
Options cancelled or forfeited (in shares)	(1,068,000)
Options outstanding, ending balance (in shares)	34,389,000
Options exercisable (in shares)	20,922,000
Number of stock option awards vested and expected to vest (in shares)	31,784,000
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Outstanding, Weighted Average Exercise Price [Abstract]
Weighted average exercise price, beginning balance (in usd per share) \| $ / shares	$ 8.24
Weighted average exercise price of stock option awards granted (in usd per share) \| $ / shares	0
Weighted average exercise price of stock option awards exercised (in usd per share) \| $ / shares	3.27
Weighted average exercise price of stock option awards cancelled (in usd per share) \| $ / shares	9.60
Weighted average exercise price, ending balance (in usd per share) \| $ / shares	8.20
Weighted average exercise price, stock option awards, exercisable (in dollars per share) \| $ / shares	7.88
Weighted average exercise price of stock option awards vested and expected to vest (in dollars per share) \| $ / shares	$ 8.13
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Additional Disclosures [Abstract]
Weighted average remaining contractual life \| $	$ 103
Options, exercises in period, intrinsic value	6 years 7 months 9 days
Options outstanding, aggregate intrinsic value \| $	$ 6,951
Options, exercisable, weighted average remaining contractual term (year)	5 years 3 months 18 days
Stock option awards vested and expected to vest, weighted average remaining contractual life (year) \| $	$ 6,951
Options, aggregate intrinsic value	6 years 5 months 26 days
Options, exercisable, aggregate intrinsic value \| $	$ 6,951
Outstanding stock options \| Inducement Plan
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Outstanding [Roll Forward]
Options outstanding, beginning balance (in shares)	5,527,000
Options, grants in period, gross (in shares)	92,000
Options exercised (in shares)	(128,000)
Options cancelled or forfeited (in shares)	(300,000)
Options outstanding, ending balance (in shares)	5,191,000
Options exercisable (in shares)	2,628,000
Number of stock option awards vested and expected to vest (in shares)	4,684,000
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Outstanding, Weighted Average Exercise Price [Abstract]
Weighted average exercise price, beginning balance (in usd per share) \| $ / shares	$ 8.77
Weighted average exercise price of stock option awards granted (in usd per share) \| $ / shares	5.25
Weighted average exercise price of stock option awards exercised (in usd per share) \| $ / shares	3.09
Weighted average exercise price of stock option awards cancelled (in usd per share) \| $ / shares	10.86
Weighted average exercise price, ending balance (in usd per share) \| $ / shares	8.73
Weighted average exercise price, stock option awards, exercisable (in dollars per share) \| $ / shares	7.53
Weighted average exercise price of stock option awards vested and expected to vest (in dollars per share) \| $ / shares	$ 8.59
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Additional Disclosures [Abstract]
Weighted average remaining contractual life \| $	$ 274
Options, exercises in period, intrinsic value	7 years 6 months 10 days
Options outstanding, aggregate intrinsic value \| $	$ 2,454
Options, exercisable, weighted average remaining contractual term (year)	6 years 9 months 3 days
Stock option awards vested and expected to vest, weighted average remaining contractual life (year) \| $	$ 2,128
Options, aggregate intrinsic value	7 years 5 months 15 days
Options, exercisable, aggregate intrinsic value \| $	$ 2,373

Significant Accounting Policies and Concentrations of Risk (Tables)

3 Months Ended

Mar. 31, 2024

Accounting Policies [Abstract]

Summary of Revenues

The Company recorded the following revenues for the three months ended March 31, 2024 and 2023 (in thousands):


	Three Months Ended March 31,
	2024		2023
Product sales, net	$	89,272	$	68,166
Collaborative and other revenues	3,489		612
Total revenues	$	92,761	$	68,778

The Company recorded the following revenues for the three months ended March 31, 2024 and 2023 (in thousands):


	Three Months Ended March 31,
	2024		2023
ORLADEYO:
U.S.	$	79,966	$	60,849
Outside of U.S.	8,901		7,565
Total ORLADEYO	88,867		68,414
Other revenues	3,894		364
Total revenues	$	92,761	$	68,778

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 March 31, 2024 and March 31, 2023, from the computation of diluted net loss per share attributable to common stockholders because including them would have had an anti-dilutive effect:


	March 31,
	2024	2023
Outstanding stock options	39,580	35,858
Unvested restricted stock unit awards	6,124	4,654
Warrants to purchase common stock	—	15,023
Total	45,704	55,535

Trade Receivables - Summary of Receivables (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Accounts, Notes, Loans and Financing Receivable [Line Items]
Total receivables	$ 3,578	$ 2,296
Billed
Accounts, Notes, Loans and Financing Receivable [Line Items]
Total receivables	86	0
Unbilled
Accounts, Notes, Loans and Financing Receivable [Line Items]
Total receivables	3,492	2,296
Royalty receivables from partners
Accounts, Notes, Loans and Financing Receivable [Line Items]
Total receivables	3,578	2,296
Royalty receivables from partners \| Billed
Accounts, Notes, Loans and Financing Receivable [Line Items]
Total receivables	86	0
Royalty receivables from partners \| Unbilled
Accounts, Notes, Loans and Financing Receivable [Line Items]
Total receivables	$ 3,492	$ 2,296

Revenue (Details) - USD ($) $ in Thousands	3 Months Ended
Revenue (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Mar. 31, 2023
Disaggregation of Revenue [Line Items]
Revenues	$ 92,761	$ 68,778
Product sales, net
Disaggregation of Revenue [Line Items]
Revenues	89,272	68,166
Product sales, net \| U.S.
Disaggregation of Revenue [Line Items]
Revenues	79,966	60,849
Product sales, net \| Outside of U.S.
Disaggregation of Revenue [Line Items]
Revenues	8,901	7,565
ORLADEYO
Disaggregation of Revenue [Line Items]
Revenues	88,867	68,414
Other revenues
Disaggregation of Revenue [Line Items]
Revenues	$ 3,894	$ 364

- Definition

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

+ References + Details

- Definition

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

+ References

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

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

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

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

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
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-Name Accounting Standards Codification
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-Publisher FASB
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Reference 6: http://www.xbrl.org/2003/role/disclosureRef
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-Subparagraph (b)
-Publisher FASB
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Reference 7: http://www.xbrl.org/2003/role/disclosureRef
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-Name Accounting Standards Codification
-Section 50
-Paragraph 40
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482810/280-10-50-40

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 280
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-Name Accounting Standards Codification
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-Subparagraph (a)
-Publisher FASB
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Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Topic 280
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 32
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482810/280-10-50-32

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

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

+ Details

Revenue

3 Months Ended

Mar. 31, 2024

Revenue from Contract with Customer [Abstract]

Revenue

The Company recorded the following revenues for the three months ended March 31, 2024 and 2023 (in thousands):


	Three Months Ended March 31,
	2024		2023
ORLADEYO:
U.S.	$	79,966	$	60,849
Outside of U.S.	8,901		7,565
Total ORLADEYO	88,867		68,414
Other revenues	3,894		364
Total revenues	$	92,761	$	68,778

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).

- Definition

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

+ References

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

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

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
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-Name Accounting Standards Codification
-Section 50
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-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-15

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 12
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-12

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
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-Paragraph 12
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-Publisher FASB
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Reference 6: http://www.xbrl.org/2003/role/disclosureRef
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-Section 50
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-Subparagraph (c)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-12

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
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-Section 50
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-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147479806/606-10-50-12

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
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-Name Accounting Standards Codification
-Section 50
-Paragraph 12
-Subparagraph (e)
-Publisher FASB
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Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Topic 606
-SubTopic 10
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-Section 50
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-Subparagraph (b)(2)
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-URI https://asc.fasb.org//1943274/2147479806/606-10-50-13

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

+ Details

Workforce Reduction - Restructuring and Related Costs (Details) $ in Thousands	3 Months Ended
	Mar. 31, 2024 USD ($)
Restructuring Reserve [Roll Forward]
Restructuring costs, beginning balance	$ 3,380
Restructuring costs	1,264
Payments for restructuring	(3,168)
Restructuring costs, ending balance	$ 1,476

Lease Obligations - Balance Sheets of the Company's Operating Leases (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Assets:
Operating Lease, Right-of-Use Asset, Statement of Financial Position [Extensible Enumeration]	Other assets	Other assets
Operating lease assets, net	$ 8,864	$ 8,682
Operating Lease, Liability [Abstract]
Operating lease liabilities	1,098	1,058
Operating lease liabilities	8,596	8,390
Total	$ 9,694	$ 9,448

Stock-based Compensation - Share-Based Compensation (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Stock-based compensation expense	$ 13,652	$ 14,007
Incentive Plan
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Stock-based compensation expense	11,566	11,061
Inducement Plan
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Stock-based compensation expense	1,923	2,475
ESPP
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Stock-based compensation expense	$ 163	$ 471

- 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

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
-SubTopic 10
-Name Accounting Standards Codification
-Section 35
-Paragraph 1D
-Publisher FASB
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Reference 2: http://www.xbrl.org/2003/role/disclosureRef
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Reference 3: http://www.xbrl.org/2003/role/disclosureRef
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Reference 4: http://www.xbrl.org/2003/role/disclosureRef
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Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
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Reference 6: http://www.xbrl.org/2003/role/disclosureRef
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Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Topic 718
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+ Details

Condensed Consolidated Statements of Stockholders' Deficit - USD ($) shares in Thousands, $ in Thousands	Total	Common Stock	Additional Paid-in Capital	Accumulated Other Comprehensive Income	Accumulated Deficit
Common stock, shares outstanding, beginning balance (in shares) at Dec. 31, 2022		187,906
Beginning 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
Exercise of stock options, net (in shares)		702
Exercise of stock options, net	3,502	$ 7	3,495
Vesting of restricted stock units (in shares)		99
Vesting of restricted stock units	$ 0	$ 1	(1)
Employee stock purchase plan sales, net (in shares)	176	176
Employee stock purchase plan sales	$ 1,575	$ 2	1,573
Stock-based compensation expense	14,007		14,007
Common stock, shares outstanding, ending balance (in shares) at Mar. 31, 2023		188,883
Ending balance at Mar. 31, 2023	$ (328,287)	$ 1,889	1,177,192	585	(1,507,953)
Common stock, shares outstanding, beginning balance (in shares) at Dec. 31, 2023	205,771	205,771
Beginning balance at Dec. 31, 2023	$ (455,528)	$ 2,058	1,222,236	1,337	(1,681,159)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Net loss	(35,379)				(35,379)
Other comprehensive income (loss)	(566)			(566)
Exercise of stock options, net (in shares)		176
Exercise of stock options, net	552	$ 2	550
Vesting of restricted stock units (in shares)		155
Vesting of restricted stock units	0	$ 1	(1)
Shares withheld for taxes for vesting of restricted stock units (in shares)		(8)
Shares withheld for taxes for vesting of restricted stock units	$ (38)		(38)
Employee stock purchase plan sales, net (in shares)	251	251
Employee stock purchase plan sales	$ 1,129	$ 2	1,127
Issuance of shares to directors in lieu of cash retainer (in shares)		2
Issuance of shares to directors in lieu of cash retainer	11		11
Stock-based compensation expense	$ 13,652		13,652
Common stock, shares outstanding, ending balance (in shares) at Mar. 31, 2024	206,347	206,347
Ending balance at Mar. 31, 2024	$ (476,167)	$ 2,063	$ 1,237,537	$ 771	$ (1,716,538)

Lease Obligations - Other Supplemental Information Related to Leases (Details)	Mar. 31, 2024	Dec. 31, 2023
Lessee, Operating Lease, Liability, to be Paid, Fiscal Year Maturity [Abstract]
Weighted average remaining lease term (in years)	9 years 4 months 24 days	9 years 8 months 12 days
Operating leases	10.89%	10.88%
Finance Lease, Liability, to be Paid, Fiscal Year Maturity [Abstract]
Weighted average remaining lease term (in years)	2 years 9 months 18 days	3 years
Finance leases	7.59%	7.46%

Revenue (Tables)

3 Months Ended

Mar. 31, 2024

Revenue from Contract with Customer [Abstract]

Summary of Revenues

The Company recorded the following revenues for the three months ended March 31, 2024 and 2023 (in thousands):


	Three Months Ended March 31,
	2024		2023
Product sales, net	$	89,272	$	68,166
Collaborative and other revenues	3,489		612
Total revenues	$	92,761	$	68,778

The Company recorded the following revenues for the three months ended March 31, 2024 and 2023 (in thousands):


	Three Months Ended March 31,
	2024		2023
ORLADEYO:
U.S.	$	79,966	$	60,849
Outside of U.S.	8,901		7,565
Total ORLADEYO	88,867		68,414
Other revenues	3,894		364
Total revenues	$	92,761	$	68,778

Stock-based Compensation - Weighted Average Assumptions for Stock Option Awards Granted to Employees and Directors Under the Plans (Details) - $ / shares	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Incentive Plan
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Expected life in years		5 years 6 months
Expected Volatility		84.20%
Expected Dividend Yield		0.00%
Risk-Free Interest Rate		3.60%
Weighted average grant date fair value stock option awards (in dollars per share)		$ 7.47
Inducement Plan
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Expected life in years	5 years 9 months 18 days	5 years 6 months
Expected Volatility	82.80%	84.20%
Expected Dividend Yield	0.00%	0.00%
Risk-Free Interest Rate	4.10%	3.70%
Weighted average grant date fair value stock option awards (in dollars per share)	$ 3.77	$ 6.85

- 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

Condensed Consolidated Statements of Cash Flows - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Cash flows from operating activities:
Net loss	$ (35,379)	$ (53,333)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation and amortization	306	405
Inventory obsolescence	0	236
Stock-based compensation expense	13,652	14,007
Non-cash interest expense on royalty financing obligations and secured term loan and amortization of debt issuance costs	19,440	20,216
Amortization of premium/discount on investments	(3,181)	(1,385)
Changes in operating assets and liabilities:
Receivables	(3,839)	2,019
Inventory	(1,220)	131
Prepaid expenses and other assets	1,106	(2,547)
Accounts payable and accrued expenses	(44,569)	(27,226)
Deferred revenue	0	(35)
Net cash used in operating activities	(53,684)	(47,512)
Cash flows from investing activities:
Acquisitions of property and equipment	(235)	(160)
Purchase of investments	(88,009)	(148,637)
Sales and maturities of investments	117,000	41,688
Net cash provided by (used in) investing activities	28,756	(107,109)
Cash flows from financing activities:
Net proceeds from common stock issued under stock-based compensation plans	1,681	5,077
Net proceeds from common stock issued to directors in lieu of cash retainer	11	0
Withholding taxes paid on stock-based awards	(2,350)	0
Principal payments on finance lease liabilities	(390)	0
Net cash (used in) provided by financing activities	(1,048)	5,077
Effect of exchange rate on cash, cash equivalents, and restricted cash	(340)	(96)
Decrease in cash, cash equivalents and restricted cash	(26,316)	(149,640)
Cash, cash equivalents and restricted cash at beginning of period	112,447	306,239
Cash, cash equivalents and restricted cash at end of period	86,131	156,599
Supplemental Cash Flow Information [Abstract]
Cash paid for interest	4,987	7,142
Cash paid for taxes	186	142
Taxes withheld on stock-based awards included in accrued expenses	$ 1,846	$ 2,128

Inventory (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Inventory Disclosure [Abstract]
Raw materials	$ 7,526	$ 6,449
Work-in-process	16,864	17,591
Finished goods	6,990	6,242
Total inventory	31,380	30,282
Reserves	(1,599)	(1,599)
Total inventory, net	$ 29,781	$ 28,683

Inventory

3 Months Ended

Mar. 31, 2024

Inventory Disclosure [Abstract]

Inventory

At March 31, 2024 and December 31, 2023, 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):


	March 31, 2024		December 31, 2023
Raw materials	$	7,526	$	6,449
Work-in-process	16,864		17,591
Finished goods	6,990		6,242
Total inventory	$	31,380	$	30,282
Reserves	(1,599)		(1,599)
Total inventory, net	$	29,781	$	28,683

Stock-based Compensation

3 Months Ended

Mar. 31, 2024

Share-Based Payment Arrangement [Abstract]

Share-Based Compensation

Stock-Based Compensation

As of March 31, 2024, 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 22, 2024, subject to stockholder approval at the Company’s annual meeting of stockholders to be held on June 12, 2024. 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):


	Three Months Ended March 31,
	2024		2023
Incentive Plan	$	11,566	$	11,061
Inducement Plan	1,923		2,475
ESPP	163		471
Stock-based compensation expense	$	13,652	$	14,007

Stock Incentive Plan

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


	Shares (in thousands)	Weighted Average Exercise Price per Share		Weighted Average Remaining Contractual Term (in years)	Aggregate Intrinsic Value (in thousands)
Outstanding at December 31, 2023	35,505	$	8.24
Granted	—	—
Exercised	(48)	3.27			$	103
Cancelled or Forfeited	(1,068)	9.60
Outstanding at March 31, 2024	34,389	$	8.20	6.61	$	6,951
Exercisable at March 31, 2024	20,922	$	7.88	5.30	$	6,951
Vested and expected to vest at March 31, 2024	31,784	$	8.13	6.49	$	6,951

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


	Shares (in thousands)	Weighted Average Grant Date Fair Value
Unvested at December 31, 2023	5,592	$	8.67
Granted	128	5.30
Vested	(96)	12.30
Forfeited	(313)	8.65
Unvested at March 31, 2024	5,311	$	8.52

For restricted stock unit awards granted under the Incentive Plan, the fair value of the awards is determined based on the market value of the Company’s shares on the grant date. The weighted average grant date fair value of these awards granted during the first three months of 2024 and 2023 was $5.30 and $10.32, respectively. The fair value of the restricted stock unit awards is amortized to expense over the vesting periods using a straight-line expense attribution method.

As of March 31, 2024, total unrecognized compensation cost related to unvested restricted stock unit awards granted under the Incentive Plan was $33,457, which is expected to be recognized over a weighted average period of 1.8 years.

Inducement Equity Incentive Plan

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


	Shares (in thousands)	Weighted Average Exercise Price per Share		Weighted Average Remaining Contractual Term (in years)	Aggregate Intrinsic Value (in thousands)
Outstanding at December 31, 2023	5,527	$	8.77
Granted	92	5.25
Exercised	(128)	3.09			$	274
Cancelled or Forfeited	(300)	10.86
Outstanding at March 31, 2024	5,191	$	8.73	7.53	$	2,454
Exercisable at March 31, 2024	2,628	$	7.53	6.76	$	2,128
Vested and expected to vest at March 31, 2024	4,684	$	8.59	7.46	$	2,373

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


	Shares (in thousands)	Weighted Average Grant Date Fair Value
Unvested at December 31, 2023	915	$	9.90
Granted	63	5.25
Vested	(59)	11.96
Forfeited	(106)	10.41
Unvested at March 31, 2024	813	$	9.32

For restricted stock unit awards granted under the Inducement Plan, the fair value of the awards is determined based on the market value of the Company’s shares on the grant date. The weighted average grant date fair value of these awards granted during the first three months of 2024 and 2023 was $5.25 and $9.64, respectively. The fair value of the restricted stock unit awards is amortized to expense over the vesting periods using a straight-line expense attribution method.

As of March 31, 2024, total unrecognized compensation cost related to unvested restricted stock unit awards granted under the Inducement Plan was $5,588, which is expected to be recognized over a weighted average period of 1.7 years.

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

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

Stock Incentive Plan


	Three Months Ended March 31, 2023
Expected Life in Years	5.5
Expected Volatility	84.2		%
Expected Dividend Yield	0.0		%
Risk-Free Interest Rate	3.6		%
Weighted average grant date fair value per share	$	7.47

As of March 31, 2024, total unrecognized compensation cost related to unvested stock option awards granted under the Incentive Plan was $62,593, which is expected to be recognized over a weighted average period of 1.6 years.

Inducement Equity Incentive Plan

The following table summarizes the key assumptions used by the Company to value the stock option awards granted under the Inducement Plan during the three months ended March 31, 2024 and 2023:


	Three Months Ended March 31,
	2024			2023
Expected Life in Years	5.8			5.5
Expected Volatility	82.8		%	84.2		%
Expected Dividend Yield	0.0		%	0.0		%
Risk-Free Interest Rate	4.1		%	3.7		%
Weighted average grant date fair value per share	$	3.77		$	6.85

As of March 31, 2024, total unrecognized compensation cost related to unvested stock option awards granted under the Inducement Plan was $12,326, which is expected to be recognized over a weighted average period of 1.4 years.

Employee Stock Purchase Plan

The Company has reserved a total of 7,975 shares of common stock to be purchased under the ESPP, of which 5,203 shares remain available for purchase as of March 31, 2024. During the three months ended March 31, 2024 and March 31, 2023, the Company issued 251 and 176 shares under the ESPP, respectively.

Royalty Financing Obligations - Schedule of Royalty Financing Obligations (Details) $ in Thousands	3 Months Ended
	Mar. 31, 2024 USD ($)
Royalty Montizations [Roll Forward]
Balance	$ 531,599
Non-cash Interest expense on Royalty financing obligations	14,187
Royalty revenues paid and payable	(15,212)
Balance	530,574
2020 RPI Royalty Agreement
Royalty Montizations [Roll Forward]
Balance	174,480
Non-cash Interest expense on Royalty financing obligations	9,916
Royalty revenues paid and payable	(7,842)
Balance	$ 176,554
Effective interest rate	22.30%
2021 RPI Royalty Agreement
Royalty Montizations [Roll Forward]
Balance	$ 185,345
Non-cash Interest expense on Royalty financing obligations	0
Royalty revenues paid and payable	(679)
Balance	$ 184,666
Effective interest rate	0.00%
OMERS Royalty Agreement
Royalty Montizations [Roll Forward]
Balance	$ 171,774
Non-cash Interest expense on Royalty financing obligations	4,271
Royalty revenues paid and payable	(6,691)
Balance	$ 169,354
Effective interest rate	9.80%

Debt (Details) $ in Thousands			3 Months Ended
Debt (Details) $ in Thousands	Apr. 17, 2023 USD ($)	Dec. 07, 2020 USD ($)	Mar. 31, 2024 USD ($)	Mar. 31, 2023 USD ($)	Jul. 29, 2022 USD ($)
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
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 \| 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
Term Loan Tranche A \| Pharmakon Advisors, LP \| Line of Credit
Debt Instrument [Line Items]
Debt instrument, face amount	$ 300,000
Proceeds from Issuance of Long-Term Debt			$ 300,000
Debt instrument, term	18 months
Effective interest rate			13.34%
Interest expense, debt, total			$ 9,974
Periodic payment, interest			4,987
Fair value			318,650
Debt instrument, unamortized discount (premium) and debt issuance costs, net, total			11,147
Amortization of debt issuance costs			$ 266
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
Proceeds from Issuance of Long-Term Debt	$ 25,805
Credit Agreement \| Athyrium
Debt Instrument [Line Items]
Debt instrument, face amount		$ 200,000
Debt instrument, term	17 days
Debt instrument, LIBOR floor		1.75%
Debt instrument, LIBOR cap		3.50%
Paid-in-kind interest				$ 7,142
Long-term debt, total	$ 240,452
Interest expense	1,335
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]
Proceeds from Issuance of Long-Term Debt		$ 125,000
Amortization of debt issuance costs				$ 898
Credit Agreement \| Athyrium \| Term B Loan
Debt Instrument [Line Items]
Debt instrument, face amount					$ 25,000
Credit Agreement \| Athyrium \| Term C Loan
Debt Instrument [Line Items]
Debt instrument, face amount					50,000
Credit Agreement \| Athyrium \| Term B And C Loan
Debt Instrument [Line Items]
Debt instrument, face amount					$ 75,000
Credit Agreement \| Athyrium \| Term Loans
Debt Instrument [Line Items]
Effective interest rate				11.88%
Term Loan Tranche B \| Pharmakon Advisors, LP \| Line of Credit
Debt Instrument [Line Items]
Debt instrument, face amount	50,000
Term Loan Tranche C \| Pharmakon Advisors, LP \| Line of Credit
Debt Instrument [Line Items]
Debt instrument, face amount	50,000
Term Loan Tranche D \| Pharmakon Advisors, LP \| Line of Credit
Debt Instrument [Line Items]
Debt instrument, face amount	$ 50,000

Stockholders' Equity - Shares Reserved for Future Issuance (Details) - shares shares in Thousands	Mar. 31, 2024	Dec. 31, 2023
Equity Disclosure [Line Items]
Total shares reserved for future issuance	57,438	58,020
Outstanding stock options
Equity Disclosure [Line Items]
Total shares reserved for future issuance	39,580	41,032
Unvested restricted stock unit awards
Equity Disclosure [Line Items]
Total shares reserved for future issuance	6,124	6,507
Incentive Plan
Equity Disclosure [Line Items]
Total shares reserved for future issuance	4,628	3,376
Inducement Plan
Equity Disclosure [Line Items]
Total shares reserved for future issuance	1,903	1,651
ESPP
Equity Disclosure [Line Items]
Total shares reserved for future issuance	5,203	5,454

Inventory (Tables)

3 Months Ended

Mar. 31, 2024

Inventory Disclosure [Abstract]

Summary of Inventories

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


	March 31, 2024		December 31, 2023
Raw materials	$	7,526	$	6,449
Work-in-process	16,864		17,591
Finished goods	6,990		6,242
Total inventory	$	31,380	$	30,282
Reserves	(1,599)		(1,599)
Total inventory, net	$	29,781	$	28,683

Lease Obligations - Balance Sheets of the Company's Finance Leases (Details) - USD ($) $ in Thousands	Mar. 31, 2024	Dec. 31, 2023
Leases [Abstract]
Finance Lease, Right-of-Use Asset, Statement of Financial Position [Extensible Enumeration]	Other assets	Other assets
Finance lease assets, net	$ 4,018	$ 4,322
Finance lease liabilities	1,642	1,590
Finance lease liabilities	2,506	2,845
Total	$ 4,148	$ 4,435

Insider Trading Arrangements	3 Months Ended
Insider Trading Arrangements	Mar. 31, 2024
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

Royalty Financing Obligations (Details Textual) - USD ($) $ in Thousands	Nov. 19, 2021	Dec. 07, 2020
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%

Collaborative and Other Relationships	3 Months Ended
Collaborative and Other Relationships	Mar. 31, 2024
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Collaborative and Other Relationships	Collaborative and Other Relationships ORLADEYO Torii Pharmaceutical Co., Ltd. (“Torii”) On November 5, 2019, the Company entered into a Commercialization and License Agreement with Torii (the “Original Torii Agreement”), granting Torii the exclusive right to commercialize ORLADEYO for the prevention of HAE attacks in Japan. Under the Original Torii Agreement, the Company received an upfront, non-refundable payment of $22,000. The Company received an additional milestone payment of $15,000 in the second quarter of 2021 upon receipt from the Japanese National Health Insurance System of a reimbursement price approval for ORLADEYO. In addition, the Company was entitled to receive tiered royalty payments, ranging from 20% to 40% of annual net sales of ORLADEYO in Japan during each calendar year. Torii’s royalty payment obligations were subject to customary reductions in certain circumstances, but could not be reduced by more than 50% of the amount that otherwise would have been payable to the Company in the applicable calendar quarter. The Company identified performance obligations under the Original Torii Agreement related to (i) the license to develop and commercialize ORLADEYO, (ii) regulatory approval support, and (iii) reimbursement pricing approval support. These were each determined to be distinct from the other performance obligations. The Company allocated the $22,000 upfront consideration to the identified performance obligations using estimation approaches to determine the standalone selling prices under ASC Topic 606. Specifically, in determining the value related to the license, a valuation approach utilizing risk adjusted discounted cash flow projections was used, and an expected cost plus margin approach was utilized for the other performance obligations. On November 30, 2023, the Company entered into an Amended and Restated Commercialization and License Agreement with Torii (as amended, the “Torii Agreement”). Under the Torii Agreement, the Company is entitled to receive tiered royalty payments, ranging from 20% to 80% of annual net sales of ORLADEYO in Japan during each calendar year. The Company is now responsible for all commercial promotion activities to support ORLADEYO sales in Japan, and Torii is responsible for HAE disease awareness activities in Japan. The Company will receive a 20% royalty on annual Japanese sales below a prespecified threshold and an 80% royalty on annual Japanese sales above the prespecified threshold. Torii’s updated royalty payment obligations commenced upon November 30, 2023 and expire upon the later of (i) the tenth anniversary of the date of first commercial sale of ORLADEYO in Japan, (ii) the expiration of the Company’s patents covering ORLADEYO, and (iii) the expiration of regulatory exclusivity for ORLADEYO in Japan. The Company determined that the Torii Agreement represented a contract modification to be accounted for as if it were part of the Original Torii Agreement under ASC Topic 606. As the performance obligations under the Original Torii agreement had been fully satisfied, the Company was not required to adjust revenue previously recognized. Peramivir Injection (RAPIVAB, RAPIACTA, PERAMIFLU) Shionogi & Co., Ltd. (“Shionogi”) In February 2007, the Company entered into an exclusive license agreement with Shionogi to develop and commercialize peramivir in Japan for the treatment of seasonal and potentially life-threatening human influenza. Under the terms of the agreement, Shionogi obtained rights to injectable formulations of peramivir in Japan. In October 2008, the Company and Shionogi amended the license agreement to expand the territory covered by the agreement to include Taiwan. Shionogi has commercially launched peramivir under the commercial name RAPIACTA in Japan and Taiwan. The Company developed peramivir under a license from UAB and will owe sublicense payments to UAB on any future milestone payments and/or royalties received by the Company from Shionogi. Green Cross Corporation (“Green Cross”) In June 2006, the Company entered into an agreement with Green Cross to develop and commercialize peramivir in Korea. Under the terms of the agreement, Green Cross is responsible for all development, regulatory, and commercialization costs in Korea and the Company is entitled to share in profits resulting from the sale of peramivir in Korea, including the sale of peramivir to the Korean government for stockpiling purposes. Furthermore, Green Cross will pay the Company a premium over its cost to supply peramivir for development and any future marketing of peramivir products in Korea. Other Collaborations Clearside Biomedical, Inc. (“Clearside”) On November 3, 2023, the Company announced that it entered into a license agreement (the “Clearside Agreement”) with Clearside, enabling the Company to develop its investigational plasma kallikrein inhibitor, avoralstat, with Clearside’s SCS Microinjector® to deliver avoralstat to the back of the eye through the suprachoroidal space to treat patients with diabetic macular edema. Under the Clearside Agreement, Clearside received a $5,000 upfront license fee from the Company. 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 - Summary of Revenues (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2023
Significant Accounting Policies and Concentration of Risk [Line Items]
Revenues	$ 92,761	$ 68,778
Product sales, net
Significant Accounting Policies and Concentration of Risk [Line Items]
Revenues	89,272	68,166
Collaborative and other revenues
Significant Accounting Policies and Concentration of Risk [Line Items]
Revenues	$ 3,489	$ 612

- Definition

+ References

+ Details

Debt	3 Months Ended
Debt	Mar. 31, 2024
Debt Disclosure [Abstract]
Debt	Debt Pharmakon Loan Agreement On April 17, 2023, the Company entered into a $450,000 Loan Agreement (the “Pharmakon Loan Agreement”) with BioPharma Credit Investments V (Master) LP and BPCR Limited Partnership, as lenders, and BioPharma Credit PLC, as collateral agent for the lenders. Certain of the Company’s wholly-owned subsidiaries are guarantors to the Pharmakon Loan Agreement. The Pharmakon Loan Agreement provides for an initial term loan in the principal amount of $300,000 (the “Tranche A Loan”) funded on April 17, 2023 (the “Tranche A Closing Date”). The Company used a portion of the proceeds from the Tranche A Loan to repay the $241,787 of outstanding indebtedness (principal and interest due as of April 17, 2023) under the then-existing Athyrium Credit Agreement (defined below) and to pay associated transaction costs and fees, and used the remaining net proceeds of $25,805 for other general corporate purposes. The Pharmakon Loan Agreement also provides for three additional term loan tranches, at the Company’s option, in principal amounts of $50,000 each (each a “Subsequent Tranche Loan” and, collectively with the Tranche A Loan, the “Pharmakon Term Loans” and each, a “Pharmakon Term Loan”), which may be requested on or prior to September 30, 2024. The maturity date of the Pharmakon Loan Agreement is April 17, 2028 (the “Maturity Date”), the fifth anniversary of the Tranche A Closing Date. The Pharmakon Loan Agreement provides for quarterly interest-only payments until the Maturity Date, with the unpaid principal amount of the outstanding Pharmakon Term Loans due and payable on the Maturity Date. During the first 18 months following the Tranche A Closing Date, the Company has the option to make a portion of the applicable interest payment on the Tranche A Loan in-kind (a “Pharmakon PIK Interest Payment”) by capitalizing as principal up to 50% of the amount of interest accrued on the Tranche A Loan during the applicable interest period. The Pharmakon Term Loans will bear interest at a rate equal to the three-month Secured Overnight Financing Rate (“SOFR”) rate, which shall be no less than 1.75%, plus 7.00%, per annum or, for each interest period in which a Pharmakon PIK Interest Payment is made, with respect to the Tranche A Loan, SOFR plus 7.25%, per annum. The Tranche A Loan accrued interest at an effective interest rate of 13.34% for the three months ended March 31, 2024. 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 March 31, 2024, the Company had total borrowings of $300,000 under the Pharmakon Loan Agreement. Interest expense on the Tranche A Loan for the three months ended March 31, 2024 was $9,974. As allowable under the Pharmakon Loan Agreement, the Company has designated and accounted for 50% of the quarterly interest payments for the three months ended March 31, 2024 as a Pharmakon PIK Interest Payment and the amount of $4,987 has been added to the outstanding principal balance of the borrowing. The remaining 50% of the quarterly interest payments of $4,987 have been paid at the end of each quarterly period. As of March 31, 2024, borrowings, including the Pharmakon PIK Interest Payments, totaled $318,650. 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 $266 was recognized for the three months ended March 31, 2024. Athyrium Credit Agreement On December 7, 2020, the Company entered into a $200,000 Credit Agreement (the “Athyrium Credit Agreement”) with Athyrium Opportunities III Co-Invest 1 LP (“Athyrium”), as lender and as administrative agent for the lenders. Certain of the Company’s direct and indirect subsidiaries were guarantors to the Athyrium Credit Agreement. The Athyrium Credit Agreement provided for an initial term loan in the principal amount of $125,000 (the “Term A Loan”), which was received by the Company on December 7, 2020 and is recorded in “Secured term loan” on the Company’s balance sheet. The Company used a portion of the proceeds from the Term A Loan to repay $43,298 of outstanding indebtedness, including accrued interest, under its prior credit facility with MidCap Financial Trust. The Athyrium Credit Agreement also provided for two additional term loans, at the Company’s option, in the respective principal amounts of $25,000 (the “Term B Loan”) and $50,000 (the “Term C Loan” and, collectively with the Term A Loan and the Term B Loan, the “Athyrium Term Loans”). Having achieved all required revenue-based milestones, the Company exercised its option to draw upon the additional funding available under the Athyrium Credit Agreement, borrowing the principal amounts of $25,000 under the Term B Loan and $50,000 under the Term C Loan. Both the Term B Loan and the Term C Loan were funded on July 29, 2022 in the aggregate principal amount of $75,000. The Term B Loan and the Term C Loan were subject to all the provisions under the Athyrium Credit Agreement. On November 19, 2021, the Company entered into an amendment to the Athyrium Credit Agreement to, among other things, (i) permit the Company to enter into the 2021 RPI Royalty Purchase Agreement, the OMERS Royalty Purchase Agreement, and the other definitive documentation related thereto and to perform its obligations thereunder; and (ii) require the Company to pay to Athyrium, for the account of the lenders, a make-whole premium plus certain fees set forth in the Athyrium Credit Agreement in the event that the Company prepaid or repaid, or was required to prepay or repay, voluntarily or pursuant to mandatory prepayment obligations under the Athyrium Credit Agreement (e.g., with the proceeds of certain asset sales, certain ORLADEYO out-licensing or royalty financing transactions (excluding the Royalty Sales), extraordinary receipts, debt issuances, or upon a change of control of the Company and specified other events, subject to certain exceptions), all of the then-outstanding Athyrium Term Loans, in each case, subject to certain exceptions set forth in the Athyrium Credit Agreement. The Athyrium Credit Agreement provided for quarterly interest-only payments until the maturity date, with the unpaid principal amount of the outstanding Athyrium Term Loans due and payable on the maturity date. For each of the first eight full fiscal quarters following December 7, 2020, the Company had the option to make the applicable interest payment-in-kind (an “Athyrium PIK Interest Payment”) by capitalizing the entire amount of interest accrued during the applicable interest period with the unpaid original principal amount outstanding on the last day of such period. The Athyrium Term Loans accrued interest at a rate equal to the three-month LIBOR rate, which was no less than 1.75% and no more than 3.50% (“LIBOR”), plus 8.25%, or for each interest period in which an Athyrium PIK Interest Payment was made, LIBOR plus 10.25%. The quarter ended December 31, 2022 was the last period eligible for the Athyrium PIK Interest Payment designation. The Athyrium Term Loans accrued interest at an effective interest rate of 11.88% for the three months ended March 31, 2023. Quarterly interest payments under the Athyrium Credit Agreement for the three months ended March 31, 2023 totaled $7,142. Deferred financing amortization of $898 was recognized for the three months ended March 31, 2023. On April 17, 2023, the outstanding principal of the Athyrium Term Loans, including the Athyrium PIK Interest Payments of $240,452 along with interest accrued of $1,335 for the first 17 days of the quarterly interest period ended June 30, 2023, was repaid with the funding received through the Pharmakon Loan Agreement

- 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

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Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Topic 235
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Reference 4: http://www.xbrl.org/2003/role/disclosureRef
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Reference 6: http://www.xbrl.org/2003/role/disclosureRef
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Reference 7: http://www.xbrl.org/2003/role/disclosureRef
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+ Details

Cover - shares	3 Months Ended
Cover - shares	Mar. 31, 2024	Apr. 30, 2024
Cover [Abstract]
Document Type	10-Q
Document Quarterly Report	true
Document Period End Date	Mar. 31, 2024
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		206,377,088
Entity Central Index Key	0000882796
Current Fiscal Year End Date	--12-31
Document Fiscal Year Focus	2024
Document Fiscal Period Focus	Q1
Amendment Flag	false

Stockholders' Equity (Details)	Oct. 23, 2023 shares
Common Stock
Equity Disclosure [Line Items]
Exercise of warrants (in shares)	14,997,000
Warrants to purchase common stock
Equity Disclosure [Line Items]
Warrants outstanding (in shares)	0

Collaborative and Other Relationships (Details)			3 Months Ended
Collaborative and Other Relationships (Details)	Nov. 03, 2023 USD ($) payment	Nov. 05, 2019 USD ($)	Mar. 31, 2024 USD ($)	Mar. 31, 2023 USD ($)	Nov. 30, 2023
Collaborative and Other Relationships [Line Items]
Revenues			$ 92,761,000	$ 68,778,000
Torii Pharmaceutical Co.
Collaborative and Other Relationships [Line Items]
Revenues		$ 22,000,000
Potential milestone payments receivable if regulatory approval before specified date		$ 15,000,000
Maximum customary reduction on royalty rate		50.00%
Torii Pharmaceutical Co. \| Minimum
Collaborative and Other Relationships [Line Items]
Royalty rate if maintains sakigake designation		20.00%
Royalty rate					20.00%
Torii Pharmaceutical Co. \| Maximum
Collaborative and Other Relationships [Line Items]
Royalty rate if maintains sakigake designation		40.00%
Royalty rate					80.00%
Clearside Biomedical, Inc.
Collaborative and Other Relationships [Line Items]
Upfront payment	$ 5,000,000
Number of milestone payments \| payment	3
Number of royalties tiers \| payment	3
Clearside Biomedical, Inc. \| Annual Net Sales Of $2 Million
Collaborative and Other Relationships [Line Items]
Annual global net sales	$ 2,000,000,000
Clearside Biomedical, Inc. \| Annual Net Sales Greater Than $1.5 Million
Collaborative and Other Relationships [Line Items]
Annual global net sales	1,500,000,000
Clearside Biomedical, Inc. \| Maximum
Collaborative and Other Relationships [Line Items]
Potential milestone payment	30,000,000
Potential payment if milestone reached	$ 47,500,000