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Item 2 — Management's Discussion and Analysis
Biocryst Pharmaceuticals, Inc. · 10-Q · Q2 FY2026 · Period ended Jun 30, 2026
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The following Management’s Discussion and Analysis (“MD&A”) is intended to help the reader understand our results of operations and financial condition. This MD&A is provided as a supplement to, and should be read in conjunction with, our unaudited condensed consolidated financial statements and the accompanying notes to the financial statements and other disclosures included in this report (including the “Cautionary Note Regarding Forward-Looking Statements” at the beginning of this report and the “Risk Factors” section in Part II, Item 1A of this report).
Overview
We are a global biotechnology company focused on developing and commercializing medicines for hereditary angioedema (“HAE”) and other rare diseases, driven by our deep commitment to improving the lives of people living with these conditions. We have built a robust commercial infrastructure to support the successful commercialization of ORLADEYO, an oral, once-daily therapy discovered and developed internally for the prevention of HAE attacks. Our business strategy includes leveraging this established commercial platform to successfully commercialize a pipeline of potential first-in-class or best-in-class oral small-molecule and injectable protein therapeutics targeting a range of rare diseases. In June 2026, we announced that we would discontinue our internal discovery programs and close our Discovery Center of Excellence facility in Birmingham, Alabama. We plan to focus on identifying and advancing high-value opportunities through external innovation, rigorous scientific evaluation, and disciplined capital allocation. By utilizing our existing commercial capabilities and focusing on rare disease markets, we believe that we can most effectively optimize our costs and strategically allocate resources to support long-term, sustainable growth.
Products and Product Candidates
ORLADEYO® (berotralstat)
ORLADEYO is an oral, once-daily therapy discovered and developed by us for the prevention of HAE attacks. A capsule formulation of ORLADEYO is approved in the United States and other global markets for the prevention of HAE attacks in adults and pediatric patients 12 years and older. In addition, in December 2025, the U.S. Food and Drug Administration (“FDA”) approved an oral pellet formulation of once-daily ORLADEYO for prophylactic therapy in pediatric patients with HAE aged 2 to <12 years.
Based on proprietary analyses of HAE prevalence and market research studies with HAE patients, physicians, and payors in the United States and Europe, and over five years of commercialization experience with ORLADEYO, we anticipate that the global commercial market for ORLADEYO has the potential to reach a global peak of $1 billion in annual net ORLADEYO revenues. These expectations are subject to numerous risks and uncertainties that may cause our actual results, performance, or achievements to be materially different. There can be no assurance that our commercialization methods and strategies will succeed, or that the market for ORLADEYO will develop in line with our current expectations. See “Risk Factors—Risks Relating to Our Business—Risks Relating to Product Development and Commercialization—There can be no assurance that our or our partners’ commercialization efforts, methods, and strategies for our products or technologies will succeed, and our future revenue generation is uncertain” in Part II, Item 1A of this report for further discussion of these risks.
Revenue from sales of ORLADEYO for the three and six months ended June 30, 2026 is discussed under “Results of Operations” in this MD&A. Revenue from sales of ORLADEYO in future periods is subject to uncertainties and will depend on several factors, including, but not limited to, the success of our and our partners’ commercialization efforts in the United States and elsewhere, the number of new patients switching to ORLADEYO, patient retention and demand, the number of physicians prescribing ORLADEYO, the rate of monthly prescriptions, reimbursement from third-party and government payors, the number of patients receiving free product, our pricing strategy, and market trends. We monitor and analyze this data on an ongoing basis as we continue to commercialize ORLADEYO and adjust our forecasts accordingly. In addition, on May 6, 2026, we announced that we identified a manufacturing issue that delayed the initial product fulfillment of the oral pellet formulation of ORLADEYO. On June 29, 2026, we announced that the issue was resolved, and the week of August 3, 2026, we began shipping the ORLADEYO oral pellets in the United States to pediatric patients with HAE aged 2 to <12 years.
Navenibart (STAR-0215)
On January 23, 2026, we completed the previously announced merger (the “Merger”) with Astria Therapeutics, Inc. (“Astria”). Pursuant to the Merger, we acquired Astria’s lead product candidate navenibart, an injectable monoclonal antibody designed to inhibit plasma kallikrein for the prevention of HAE attacks. Navenibart is currently in Phase 3 clinical
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development, and the FDA has granted Fast Track and Orphan Drug designations to navenibart for the treatment of HAE. The goal for navenibart is to develop an injectable prophylactic therapy with potential best-in-class dosing every 3- or 6-months, which could offer significant improvements over existing injectable options and address key unmet needs in the HAE patient community.
BCX17725 (Netherton syndrome)
BCX17725 is a potent and selective investigational protein therapeutic KLK5 inhibitor, currently in Phase 1 clinical development, and designed to provide best-in-class, potentially disease-modifying, treatment for people with Netherton syndrome. Netherton syndrome is a serious, rare, lifelong genetic disorder causing disruption of the skin barrier with premature separation of the skin layers, chronic inflammation and vulnerability to serious infections, caused by lack of normal function of a natural inhibitor of KLK5. People with Netherton syndrome often have itchy, red, scaly, inflamed skin, fragile hair, and are more likely to develop severe food allergies, asthma and eczema. Netherton syndrome can be life-threatening, especially during infancy when patients are vulnerable to dehydration and recurrent infections. Currently, there are no approved treatments that target the underlying cause of Netherton syndrome. BCX17725 is designed to replace missing functions of the natural KLK5 inhibitor, which could restore the normal skin barrier and result in improved skin function, including protection from severe inflammatory and infectious complications of the disease.
RAPIVAB®/RAPIACTA®/PERAMIFLU® (peramivir injection)
RAPIVAB (peramivir injection) is approved in the United States for the treatment of acute uncomplicated influenza for patients six months and older. Peramivir injection is also approved in Canada (RAPIVAB), Australia (RAPIVAB), Japan (RAPIACTA), Taiwan (RAPIACTA), and Korea (PERAMIFLU).
Revenues and Expenses
Our revenues are difficult to predict and depend on several factors, including those discussed in the “Risk Factors” section in Part II, Item 1A of this report. For example, our revenues depend, in part, on regulatory approval decisions for our products and product candidates, the effectiveness of our and our collaborative partners’ commercialization efforts, market acceptance of our products, particularly ORLADEYO, and the resources dedicated to our products and product candidates by us and our collaborative partners, as well as entering into or modifying licensing agreements for our product candidates. Furthermore, revenues related to our collaborative development activities are dependent upon the progress toward, and the achievement of, developmental milestones by us or our collaborative partners.
Our operating expenses are also difficult to predict and depend primarily on research and development activities, including clinical research activities, and the ongoing requirements of our development programs, as well as the costs of commercialization, drug manufacturing, direction from regulatory agencies and the factors discussed in the “Risk Factors” section in Part II, Item 1A of this report. Management may be able to control the timing and level of research and development and selling, general and administrative expenses, but many of these expenditures will occur irrespective of our actions due to contractually committed activities and/or payments. In addition, in June 2026, we announced the Birmingham Closure Plan (as defined below). While we expect costs associated with the Birmingham Closure Plan to include costs related to contract termination, lease termination, employee termination benefits and severance, among other costs, management’s analysis of the Birmingham Closure Plan’s execution and related impact is still ongoing.
As a result of these factors, we believe that period-to-period comparisons are not necessarily meaningful, and you should not rely on them as an indication of future performance. Due to the foregoing factors, it is possible that our operating results will be below the expectations of market analysts and investors. In such event, the prevailing market price of our common stock could be materially adversely affected.
Recent Developments
ORLADEYO (berotralstat)
In May 2026, we presented new real-world evidence underscoring the ongoing burden of pediatric HAE, with studies highlighting the significant healthcare and quality-of-life burden associated with HAE in pediatric patients and their caregivers. In addition, on June 12, 2026, we announced that new clinical data and real-world evidence demonstrated that treatment with ORLADEYO was associated with consistent reductions in HAE attack burden and healthcare utilization across diverse patient populations.
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We announced on August 5, 2026 that initial product shipments of ORLADEYO oral pellets to pediatric patients began the week of August 3, marking a new paradigm in the treatment of HAE in pediatrics. In addition, to support the growing scale of ORLADEYO across both adults and pediatrics, we have engaged a new commercial pharmacy partner for ORLADEYO to serve as our sole source specialty pharmacy for ORLADEYO shipments to patients in the United States beginning in the third quarter of 2026.
Navenibart (STAR-0215)
On May 4, 2026, we announced that we entered into a licensing agreement granting an Irish affiliate of Neopharmed Gentili S.p.A. (“Neopharmed”) exclusive rights to commercialize navenibart for HAE in Europe (the “Neopharmed License Agreement”). We received upfront consideration of $70.0 million and will be eligible to receive up to $275.0 million in future regulatory and sales milestone payments. We will also receive tiered royalties on net sales ranging from 18% to 30%. Navenibart is an investigational product that has not yet received regulatory approval in the United States or Europe.
On May 6, 2026, we announced that patient enrollment in ALPHA-ORBIT, the ongoing pivotal study of navenibart for the prophylaxis of HAE, was on track to be completed by the end of June 2026. On June 29, 2026, we announced that enrollment was completed in June 2026, putting navenibart on track to potentially be the first HAE therapy to have both 3- and 6-month dosing with efficacy evaluated through 12 months.
On June 12, 2026, we announced that a post-hoc analysis of the Phase 1b/2 multicenter, dose-ranging, open-label ALPHA-STAR study of navenibart demonstrated that investigational navenibart consistently reduced HAE attack rates across patient subgroups, supporting the ongoing Phase 3 evaluation of navenibart as a potential long-acting therapeutic option for the broad HAE population.
BCX17725 (Netherton syndrome)
On May 6, 2026, we announced that we have begun dosing in Part 4 of the Phase 1 trial of BCX17725 for the treatment of Netherton syndrome, which will enroll up to 12 patients for three months.
Birmingham Closure Plan
On June 25, 2026, our Board of Directors approved a plan to discontinue our internal discovery programs and close the Discovery Center of Excellence facility in Birmingham, Alabama (the “Birmingham Closure Plan”). The Birmingham Closure Plan was approved as part of the ongoing strategic evolution to strengthen our rare disease pipeline, following a comprehensive strategic review and scientific diligence of our research capabilities, programs and priorities. The implementation of the Birmingham Closure Plan is expected to be substantially complete by the end of 2026.
Results of Operations for the three and six months ended June 30, 2026 compared to the three and six months ended June 30, 2025
Revenues
The following table summarizes our revenues for the three months ended June 30, 2026 and 2025:
Three Months Ended June 30,
2026 2025
ORLADEYO $ 158,202 $ 143,527
European ORLADEYO business — 13,310
Total ORLADEYO 158,202 156,837
License and other revenues 60,048 6,516
Total revenues $ 218,250 $ 163,353
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The $54.9 million increase in total revenues for the three months ended June 30, 2026 compared to the three months ended June 30, 2025 was primarily driven by:
•$53.5 million increase in license and other revenues primarily attributable to the recognition of $55.7 million of revenue related to the Neopharmed License Agreement (see “Note 12—Collaborative and Other Relationships” in the Notes to the Condensed Consolidated Financial Statements in Part I, Item 1 of this report for additional information on this agreement), partially offset by a $6.2 million decrease in direct sales of peramivir primarily due to lower direct sales of peramivir to the U.S. Department of Health and Human Services (“HHS”) following the expiration of the initial 12‑month base ordering period in September 2025, after which no additional ordering periods were exercised; and
•$14.7 million increase in ORLADEYO revenue, excluding revenues associated with our European ORLADEYO business, primarily due to a net price increase and an increase in volume of direct sales of ORLADEYO due to continued strong market dynamics; partially offset by:
•$13.3 million decrease in revenues associated with our European ORLADEYO business due to the sale of our European ORLADEYO business to Neopharmed on October 1, 2025.
The following table summarizes our revenues for the six months ended June 30, 2026 and 2025:
Six Months Ended June 30,
2026 2025
ORLADEYO $ 306,549 $ 266,234
European ORLADEYO business — 24,846
Total ORLADEYO 306,549 291,080
License and other revenues 68,114 17,807
Total revenues $ 374,663 $ 308,887
The $65.8 million increase in total revenues for the six months ended June 30, 2026 compared to the six months ended June 30, 2025 was primarily driven by the following:
•$50.3 million increase in license and other revenues primarily attributable to:
◦$55.7 million increase due to the recognition of revenue related to the Neopharmed License Agreement;
◦$5.9 million increase due to revenue related to quarterly royalty payments from BioCryst Ireland Limited; and
◦$2.5 million increase due to the recognition of revenue related to the Kaken License Agreement (see “Note 12—Collaborative and Other Relationships” in the Notes to the Condensed Consolidated Financial Statements in Part I, Item 1 of this report for additional information on this agreement); partially offset by:
◦$13.2 million decrease in direct sales of peramivir primarily due to lower direct sales of peramivir to the HHS following the expiration of the initial 12‑month base ordering period in September 2025, after which no additional ordering periods were exercised.
•$40.3 million increase in ORLADEYO revenue, excluding revenues associated with our European ORLADEYO business, primarily due to a net price increase and an increase in direct sales of ORLADEYO due to continued strong market dynamics; partially offset by:
• $24.8 million decrease in revenues associated with our European ORLADEYO business due to the sale of our European ORLADEYO business to Neopharmed on October 1, 2025.
Cost of product sales
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The following tables summarize our cost of product sales for the three and six months ended June 30, 2026 and 2025:
Three Months Ended June 30,
2026 2025
Cost of product sales - ORLADEYO $ 3,737 $ 1,308
Cost of product sales - peramivir 103 410
European ORLADEYO business — 1,080
Total cost of product sales $ 3,840 $ 2,798
Six Months Ended June 30,
2026 2025
Cost of product sales - ORLADEYO $ 6,433 $ 2,637
Cost of product sales - peramivir 2,784 2,984
European ORLADEYO business — 1,745
Total cost of product sales $ 9,217 $ 7,366
The increase in cost of product sales for the three and six months ended June 30, 2026 compared to three and six months ended June 30, 2025 was primarily due to an increase in ORLADEYO sales to our partners, partially offset by a decrease in cost of product sales associated with our European ORLADEYO business due to the sale of our European ORLADEYO business to Neopharmed on October 1, 2025.
Acquired in-process research and development expense
Acquired in-process research and development expense was $697.8 million for the six months ended June 30, 2026 related to the non-cash charge recognized for the value assigned to the navenibart in-process research and development asset acquired as part of the Merger. See “Note 2—Acquisition of Astria Therapeutics, Inc.” in the Notes to the Condensed Consolidated Financial Statements in Part I, Item 1 of this report for additional information on the Merger.
Research and development expenses
Research and development expenses include all costs incurred to discover, develop and advance our product candidates and related technologies. These costs include direct costs, such as compensation for research and development personnel and costs paid to third-parties for laboratory studies, process development and manufacturing of product candidates, and the conduct and management of clinical trials and other clinical and preclinical activities. Additionally, direct expenses include those costs necessary to discontinue and close out a development program, including termination fees and other commitments. Research and development expenses also include indirect costs, such as lab supplies and services, facility costs, depreciation of lab equipment and other overhead of our research and development activities. Research and development expenses vary based on the number of programs in development and the stage of development of each program. Later stage clinical programs generally require higher spending than earlier stage programs due to the longer length of time of the clinical trials and the higher patient enrollment.
We do not maintain or evaluate internal research and development costs on a program-by-program basis, and certain costs may benefit multiple programs. Beginning in the quarter ended September 30, 2025, we no longer allocate non-program specific external costs or internal costs to programs. These costs are separately presented on the respective line items listed below. Research and development expenses have been reclassified for the three and six months ended June 30, 2025 for comparability. There is no impact on total research and development expenses.
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The following table summarizes our research and development expenses for the three months ended June 30, 2026 and 2025:
Three Months Ended June 30,
2026 2025
Navenibart $ 17,328 $ —
BCX17725 5,240 3,316
Berotralstat 1,611 3,796
Avoralstat 351 3,305
STAR-0310 91 —
Research, discovery and preclinical programs 3,441 3,560
Compensation and related personnel costs 11,390 12,053
Stock-based compensation 5,473 9,327
Other non-program specific and indirect costs 7,044 6,743
European ORLADEYO business (excluding stock-based compensation) — 1,286
Total research and development expenses $ 51,969 $ 43,386
The increase in research and development expenses for the three months ended June 30, 2026 compared to three months ended June 30, 2025 was primarily driven by the following:
•$17.3 million increase in navenibart due to the acquisition of the Phase 3 product candidate in connection with the Merger; and
•$1.9 million increase in BCX17725 primarily due to an increase in drug manufacturing activities.
These increases were partially offset by the following:
•$3.9 million decrease in stock-based compensation expense primarily due to research and development related turnover and prior period acceleration of expense pursuant to the BioCryst Pharmaceuticals, Inc. Equity Award Retirement Policy, partially offset by a modification to extend the post-termination exercise period of certain vested stock option awards at the time of retirement for certain individuals to the original expiration date, resulting in $2.4 million of incremental expense in the second quarter of 2026 (see “Note 11—Stock-Based Compensation” in the Notes to the Condensed Consolidated Financial Statements in Part I, Item 1 of this report);
•$3.0 million decrease in avoralstat due to the discontinuation of the program in the first quarter of 2026;
•$2.2 million decrease in berotralstat primarily attributed to a decrease in manufacturing and other costs incurred to support FDA approval in pediatric patients in 2025; and
•$1.3 million decrease in research and development expenses associated with our European ORLADEYO business (excluding stock-based compensation) due to the sale of our European ORLADEYO business to Neopharmed on October 1, 2025.
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The following table summarizes our research and development expenses for the six months ended June 30, 2026 and 2025:
Six Months Ended June 30,
2026 2025
Navenibart $ 27,069 $ —
BCX17725 7,147 5,772
Berotralstat 3,407 5,663
Avoralstat 1,244 5,784
STAR-0310 774 —
Research, discovery and preclinical programs 7,411 6,604
Compensation and related personnel costs 38,481 24,246
Stock-based compensation 12,292 17,855
Other non-program specific and indirect costs 14,463 13,289
European ORLADEYO business (excluding stock-based compensation) — 1,443
Total research and development expenses $ 112,288 $ 80,656
The increase in research and development expenses for the six months ended June 30, 2026 compared to the six months ended June 30, 2025 was primarily driven by the following:
•$27.1 million increase in navenibart and $0.8 million increase in STAR-0310 due to the acquisition of the Phase 3 and Phase 1a product candidates, respectively, in connection with the Merger; and
•$14.2 million increase in compensation and related personnel costs primarily due to expenses incurred in connection with the Merger, including the portion of the Astria stock option payout attributable to post-combination service, totaling $10.7 million, and $4.0 million of Astria related severance costs.
These increases were partially offset by the following:
•$5.6 million decrease in stock-based compensation expense primarily due to research and development related turnover and prior period acceleration of expense pursuant to the BioCryst Pharmaceuticals, Inc. Equity Award Retirement Policy, partially offset by a modification to extend the post-termination exercise period of certain vested stock option awards at the time of retirement for certain individuals to the original expiration date, resulting in $5.4 million of incremental expense in 2026;
•$4.5 million decrease in avoralstat due to the discontinuation of the program in the first quarter of 2026;
•$2.3 million decrease in berotralstat primarily attributed to a decrease in manufacturing and other costs to support FDA approval in pediatric patients in 2025; and
•$1.4 million decrease in research and development expenses associated with our European ORLADEYO business (excluding stock-based compensation) due to the sale of our European ORLADEYO business to Neopharmed on October 1, 2025.
Selling, general, and administrative expenses
Selling, general and administrative expenses represent costs incurred to commercialize our products and support our operations. Sales and marketing expenses include personnel-related costs, activities supporting the commercialization and distribution of marketed products, market research, marketing, medical affairs, market access, and advertising costs. General and administrative expenses include personnel-related costs and other expenses associated with quality, finance,
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human resources, information technology, legal, compliance, and other administrative functions, including transaction-related costs.
The following table summarizes our selling, general, and administrative expenses for the three months ended June 30, 2026 and 2025:
Three Months Ended June 30,
2026 2025
Sales and marketing (excluding stock-based compensation) $ 33,947 $ 33,424
General and administrative (excluding stock-based compensation) 20,810 27,128
European ORLADEYO business (excluding stock-based compensation) — 14,854
Stock-based compensation 9,219 11,977
Total selling, general, and administrative expenses $ 63,976 $ 87,383
The decrease in general and administrative expenses (excluding stock-based compensation) for the three months ended June 30, 2026 compared to the three months ended June 30, 2025 was primarily driven by $5.9 million of transaction-related costs incurred in 2025 associated with the sale of our European ORLADEYO business to Neopharmed on October 1, 2025, partially offset by a $2.4 million increase in employee, facility, and other costs associated with Astria.
Expenses associated with our European ORLADEYO business (excluding stock-based compensation) were $14.9 million for the three months ended June 30, 2025. There were no corresponding expenses for the three months ended June 30, 2026 due to the sale of our European ORLADEYO business to Neopharmed on October 1, 2025.
The decrease in stock-based compensation expense for the three months ended June 30, 2026 compared to the three months ended June 30, 2025 was primarily attributed to employee turnover and prior period acceleration of expense pursuant to the BioCryst Pharmaceuticals, Inc. Equity Award Retirement Policy, partially offset by an increase in awards granted.
The following table summarizes our selling, general, and administrative expenses for the six months ended June 30, 2026 and 2025:
Six Months Ended June 30,
2026 2025
Sales and marketing (excluding stock-based compensation) $ 76,900 $ 71,901
General and administrative (excluding stock-based compensation) 63,203 46,670
European ORLADEYO business (excluding stock-based compensation) — 26,464
Stock-based compensation 18,427 24,817
Total selling, general, and administrative expenses $ 158,530 $ 169,852
The increase in sales and marketing expenses (excluding stock-based compensation) for the six months ended June 30, 2026 compared to the six months ended June 30, 2025 was primarily driven by the following:
•$3.7 million of expense associated with the portion of the Astria stock option payout attributable to post-combination service in connection with the Merger; and
•$1.7 million of expense associated with Astria related severance costs in connection with the Merger.
The increase in general and administrative expenses (excluding stock-based compensation) for the six months ended June 30, 2026 compared to the six months ended June 30, 2025 was primarily driven by the following:
•$14.7 million of expense associated with the portion of the Astria stock option payout attributable to post-combination service in connection with the Merger;
•$6.3 million of expense associated with Astria related severance costs in connection with the Merger; and
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•$4.9 million increase due to incremental employee, facility, and other costs associated with Astria.
These increases were partially offset by $6.4 million of transaction-related costs incurred in 2025 associated with the sale of our European ORLADEYO business to Neopharmed on October 1, 2025.
Expenses associated with our European ORLADEYO business (excluding stock-based compensation) were $26.5 million for the six months ended June 30, 2025. There were no corresponding expenses for the six months ended June 30, 2026 due to the sale of our European ORLADEYO business to Neopharmed on October 1, 2025.
The decrease in stock-based compensation expense for the six months ended June 30, 2026 compared to the six months ended June 30, 2025 was primarily attributed to employee turnover and prior period acceleration of expense pursuant to the BioCryst Pharmaceuticals, Inc. Equity Award Retirement Policy, partially offset by an increase in awards granted.
Other income (expense)
The following tables summarize our other income (expense) for the three and six months ended June 30, 2026 and 2025, respectively:
Three Months Ended June 30,
2026 2025
Interest income $ 2,434 $ 2,516
Interest expense (21,712) (21,582)
Foreign currency gains (losses), net 27 (63)
Loss on extinguishment of debt — (4,171)
Other income 260 —
Total other expense, net $ (18,991) $ (23,300)
Six Months Ended June 30,
2026 2025
Interest income $ 4,690 $ 5,540
Interest expense (41,491) (45,076)
Foreign currency losses (198) (62)
Loss on extinguishment of debt — (4,171)
Other expense (1,202) —
Total other expense, net $ (38,201) $ (43,769)
Interest expense was primarily comprised of non-cash interest expense due to the amortization of interest associated with our royalty financing obligations and interest expense associated with the borrowings under the Blackstone Loan Agreement (as defined below) for the three and six months ended June 30, 2026 and Pharmakon Loan Agreement (as defined in “Note 8—Debt” in the Notes to the Condensed Consolidated Financial Statements in Part I, Item 1 of this report) for the three and six months ended June 30, 2025. The decrease in interest expense for the six months ended June 30, 2026 was primarily due to a lower effective interest rate under the Blackstone Loan Agreement.
In April 2025, we made a $75.0 million partial prepayment on the outstanding principal amount under the Pharmakon term loan resulting in a one-time loss on extinguishment of debt of $4.2 million for the three and six months ended June 30, 2025. We paid off the remainder of the outstanding principal balance of $248.7 million as of June 30, 2025 in two separate prepayments in the third and fourth quarters of 2025.
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Liquidity and Capital Resources
Sources of Liquidity
As of June 30, 2026, our principal sources of liquidity consisted of cash, cash equivalents and available-for-sale investments totaling $352.6 million, cash generated from ORLADEYO sales, available borrowings under the Blackstone Loan Agreement (as defined below), proceeds from licensing and collaborative agreements, and royalty financing arrangements. In addition to the above, we have previously received funding from other sources, including public offerings and private placements of equity securities, cash from research and development agreements, including U.S. Government contracts, government grants, research grants, and interest income on our investments.
On January 23, 2026, we entered into a Loan Agreement (the “Blackstone Loan Agreement”) with Blackstone Alternative Credit Advisors LP and Blackstone Life Sciences Advisors L.L.C., (together, “Blackstone”), as the Blackstone representatives thereunder, the guarantors from time to time party thereto, the lenders from time to time party thereto, and Wilmington Trust, National Association, as agent, pursuant to which the lenders funded term loans in the aggregate principal amount of $400.0 million (the “Term Loans”). We used the proceeds from the Term Loans to pay the cash portion of the consideration required to consummate the Merger. The maturity date of the Term Loans under the Blackstone Loan Agreement is January 23, 2031. Subject to the mutual agreement between us, Blackstone and the lenders, we may request additional term loans up to an aggregate principal amount not exceeding $150.0 million. Our obligations under the Blackstone Loan Agreement are secured by a security interest in, subject to certain exceptions, substantially all of our and our subsidiaries’ assets.
The Blackstone Loan Agreement also contains representations and warranties and affirmative and negative covenants customary for financings of this type (including a minimum liquidity covenant), as well as customary events of default. Certain of the customary negative covenants limit our ability and certain of our 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 certain 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 Blackstone Loan Agreement, or an occurrence of any other event of default, could permit the lenders under the Blackstone Loan Agreement to declare the borrowings thereunder, together with accrued interest and fees, and any applicable yield protection premium, to be immediately due and payable. See “Note 8—Debt” in the Notes to the Condensed Consolidated Financial Statements in Part I, Item 1 of this report for additional information about the Blackstone Loan Agreement.
In 2020 and 2021, we entered into the Royalty Purchase Agreements (as defined in “Note 7—Royalty Financing Obligations” in the Notes to the Condensed Consolidated Financial Statements in Part I, Item 1 of this report) with RPI 2019 Intermediate Finance Trust (“RPI”) and OCM IP Healthcare Holdings Limited, an affiliate of OMERS Capital Markets (“OMERS”). Under the Royalty Purchase Agreements, RPI and OMERS are 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 we sell ORLADEYO directly or through distributors. In addition, RPI and OMERS are entitled to receive a tiered revenue share on amounts generally received by us on account of ORLADEYO sublicense revenue or net sales by licensees outside of the Key Territories. Our required payments to OMERS commenced with the calendar quarter beginning October 1, 2023. No royalty payments are due on direct sales over $550.0 million. See “Note 7—Royalty Financing Obligations” in the Notes to the Condensed Consolidated Financial Statements in Part I, Item 1 of this report for additional information about these financing transactions.
Cash Flows
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The following table summarizes our cash flows for each period presented:
Six Months Ended June 30,
2026 2025
Net cash provided by (used in):
Operating activities $ 42,551 $ 13,785
Investing activities (378,796) 56,222
Financing activities 401,399 (73,157)
Effect of exchange rates on cash, cash equivalents and restricted cash (107) 1,810
Increase (decrease) in cash, cash equivalents and restricted cash, including cash classified within current assets held for sale 65,047 (1,340)
Less: net increase in cash and cash equivalents classified within current assets held for sale — (15,058)
Net increase (decrease) in cash, cash equivalents, and restricted cash $ 65,047 $ (16,398)
Operating Activities
During the six months ended June 30, 2026, we incurred a net loss of $643.4 million, which was primarily driven by the non-cash acquired in-process research and development charge of $697.8 million related to the Merger. After adjusting for this, other non-cash cash items totaling $62.7 million, and operating assets and liabilities used of $74.5 million, net cash provided by operating activities totaled $42.6 million.
During the six months ended June 30, 2025, we generated net income of $5.1 million. After adjusting for non-cash items totaling $68.4 million, and operating assets and liabilities used of $59.8 million, net cash provided by operating activities totaled $13.8 million.
Investing Activities
During the six months ended June 30, 2026, net cash used in investing activities of $378.8 million primarily related to cash paid for the acquisition of Astria, net of cash acquired, of $489.5 million and $135.6 million of purchases of investment securities, partially offset by $246.9 million of sales and maturities of investment securities.
During the six months ended June 30, 2025, net cash provided by investing activities of $56.2 million primarily related to sales and maturities of investment securities of $134.6 million, partially offset by purchases of investment securities of $78.1 million.
Financing Activities
During the six months ended June 30, 2026, net cash provided by financing activities of $401.4 million primarily consisted of net proceeds from the Term Loans under the Blackstone Loan Agreement of $395.0 million and common stock issued under stock-based compensation plans of $25.7 million, partially offset by principal payments on royalty financing obligations of $13.8 million and withholding taxes paid on stock-based awards of $4.9 million.
During the six months ended June 30, 2025, net cash used in financing activities of $73.2 million primarily consisted of repayment of Pharmakon term loan principal and related prepayment premium and fees totaling $71.8 million and principal payments on royalty financing obligations of $4.0 million, partially offset by net proceeds from common stock issued under stock-based compensation plans of $5.8 million.
Plan of Operation and Future Funding Requirements
We intend to contain costs and cash flow requirements by closely managing our third-party costs and headcount, leasing our facilities, and contracting with other parties to conduct certain research and development projects. We may incur additional expenses, potentially resulting in significant losses, as we continue to pursue our research and development activities, commercialize ORLADEYO, and engage in strategic business development. We may incur additional expenses related to the filing, prosecution, maintenance, defense, and enforcement of patent and other intellectual property claims and additional regulatory costs as our clinical programs advance through later stages of development or as regulatory exclusivity for our products expires. The objective of our investment policy is to ensure the safety and preservation of invested funds, as well as to maintain liquidity sufficient to meet cash flow requirements. We place our excess cash with high credit quality financial institutions. We invest in marketable debt securities that may consist of U.S. Treasury
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obligations, U.S. government agency securities, money market funds, certificates of deposit, and corporate notes and bonds in order to limit the amount of our credit exposure. We have not realized any significant losses on our investments.
Our expenses, revenues and cash utilization rate could vary significantly depending on many factors, some of which will consume significant capital resources, including:
•the progress and results of our current and proposed clinical trials for our product candidates;
•the progress made in the development, manufacturing, and regulatory approval process of our product candidates;
•the success of our commercialization efforts for, and market acceptance of, our products;
•our business development activities;
•the amount of funding or assistance, if any, we receive from new partnerships with third parties for the development and/or commercialization of our products and product candidates;
•the development progress of any collaborative agreements for our product candidates; and
•the amount and timing of funding we receive, if any, from U.S. Government contracts.
Based on our expectations for revenue and operating expenses, we believe our financial resources will be sufficient to fund our operations for at least the next 12 months. Our liquidity needs will be largely determined by the success of operations in regard to the successful commercialization of our products and the future progression of our product candidates. In addition to financing our needs through our existing capital resources and interest earned on that capital, as well as revenues from product sales, we periodically evaluate other opportunities to fund future operations, including: (1) out-licensing rights to certain of our products or product candidates, pursuant to which we would receive cash milestone payments; (2) obtaining additional product candidate regulatory approvals, which would generate revenue, milestone payments and cash flow; (3) reducing spending on one or more research and development programs, including by discontinuing development; (4) restructuring operations to change our overhead structure; (5) securing U.S. Government funding of our programs, including obtaining procurement contracts; and/or (6) pursuing royalty or loan financing. We may also, 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. Our future liquidity needs, and our ability to address those needs, will largely be determined by the success of our products and product candidates; the success of our business development efforts; the timing, scope, and magnitude of our research and development and commercial expenses; and key regulatory and operational developments.
Our long-term capital requirements and the adequacy of our available funds will depend upon many factors, including:
•sustained market acceptance of approved products and successful commercialization of such products by either us or our partners;
•the progress and magnitude of our research and development programs;
•the success of our external innovation strategy;
•changes in existing collaborative relationships;
•our ability to establish additional collaborative relationships if and when needed;
•the extent to which our partners will share in the costs associated with the development of our programs or run the development programs themselves;
•our ability to negotiate favorable development and marketing strategic alliances for certain products and product candidates;
•any decision to build or expand our commercial capabilities;
•the scope and results of preclinical studies and clinical trials to develop product candidates;
•our ability to engage sites and enroll subjects in our clinical trials;
•the scope of manufacturing of our products to support our commercial operations and of our product candidates to support our preclinical research and clinical trials;
•the scope of manufacturing of our drug substance and product candidates required for future new drug application filings;
•competitive and technological advances;
•the time and costs involved in obtaining regulatory approvals;
•post-approval commitments for any products that receive regulatory approval;
•our business development activities; and
•the costs involved in all aspects of intellectual property strategy and protection, including the costs involved in preparing, filing, prosecuting, maintaining, defending, and enforcing patent claims.
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We may, in the future, be required to raise additional capital to complete the development and commercialization of our products and product candidates, and we may seek to raise capital in the future, including to take advantage of favorable opportunities in the capital markets. Additional funding may not be available when needed or in the form or on terms acceptable to us. Our future working capital requirements, including the need for additional working capital, will largely be determined by the advancement of our portfolio of product candidates and the commercialization of ORLADEYO. More specifically, our working capital requirements will be dependent on the number, magnitude, scope and timing of our development programs; regulatory approval of our product candidates; the cost, timing and outcome of regulatory reviews, regulatory investigations, and changes in regulatory requirements; the costs of obtaining patent protection for our product candidates; the timing and terms of business development activities; the rate of technological advances relevant to our operations; the efficiency of manufacturing processes developed on our behalf by third parties; the timing, scope and magnitude of commercial spending; and the level of required administrative support for our daily operations. See “Risk Factors—Risks Relating to Our Business—Financial and Liquidity Risks” in Part II, Item 1A of this report for further discussion of the risks related to obtaining additional capital.
Critical Accounting Estimates
The preparation of these consolidated financial statements in accordance with U.S. GAAP requires us to make estimates and judgments that affect the reported amounts of assets, liabilities, revenues, expenses, and related disclosures for the periods presented. Some of these estimates can be subjective and complex with a significant level of estimation uncertainty, and, consequently, actual results may differ from these estimates. The judgments and assumptions used by management are based on historical experience and information available to us at the time that we make these estimates and judgments. To the extent there are material differences between these estimates and actual results, our consolidated financial statements will be affected. Although we believe that our judgments and estimates are appropriate, actual results may differ from these estimates.
While our significant accounting policies are more fully described in “Note 1—Significant Accounting Policies and Concentrations of Risk” in the Notes to the Condensed Consolidated Financial Statements in Part I, Item 1 of this report, we believe the following accounting policies to be most critical to the judgments and estimates used in the preparation of our Condensed Consolidated Financial Statements.
Revenue Recognition
The application of Accounting Standards Codification (“ASC”) Topic 606, Revenue from Contracts with Customers, substantially impacts our reported results, particularly product sales, net, which requires certain estimates in determining the transaction price.
Net revenue from sales of ORLADEYO is recorded at net selling price (transaction price), which includes reserves for variable consideration such as (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, representing our best estimates of the amount of consideration to which we are entitled based on the terms of the applicable contracts and statutory requirements, are based on the amounts earned or to be claimed on the related sales and are classified as reductions of accounts receivable if no payments are required of us or a current liability if a payment is required of us. Actual amounts of consideration may differ from our estimates. If actual results vary from estimates, these estimates are adjusted, which would affect net product revenue and earnings in the period such variances become known.
The most subjective of these estimates are government and managed care rebates. We contract with group purchasing organizations associated with managed care organizations and participate in certain government programs or, collectively, third-party payors, so that ORLADEYO will be eligible for purchase by, or partial or full reimbursement from, such third-party payors. We estimate the rebates we will provide to third-party payors and deduct these estimated amounts from total gross product revenues at the time the revenues are recognized, resulting in a reduction of product revenue and the establishment of a current liability. We estimate the rebates that we will provide to third-party payors based upon (i) our contracts with these third-party payors, (ii) the contractually mandated discounts applicable to the programs, and (iii) product distribution information obtained from our specialty pharmacy regarding payor mix.
Research and Development Expenses and Related Accruals
We estimate research and development expenses based on the review of contracts and purchase orders, discussions with internal personnel to identify services that have been performed on our behalf, discussions with service providers, and assumptions regarding actual work completed and the associated costs incurred when we have not yet been invoiced or
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otherwise notified of actual cost. We consider facts and circumstances known to us at the time, which may include assumptions regarding expected patient enrollment, site activation and estimated project duration. In expensing service fees, we estimate the time period over which services will be performed and the level of effort expended in each period. Examples of estimated accrued research and development 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 our raw materials, drug substance, drug products, and product candidates, and (iv) professional fees.
The financial terms of our agreements may result in uneven payment flows, as payments may depend on factors such as the successful enrollment of patients and the completion of milestones. We accrue for expenses when we determine an obligation has been incurred, regardless of the timing of the invoice. We periodically confirm the accuracy of our estimates with the service providers and adjust if necessary. If we do not identify costs that we have begun to incur or if we underestimate or overestimate the level of these costs, our actual expenses could differ from our estimates.
Royalty Financing Obligations
Under the royalty financing obligations, RPI and OMERS are entitled to receive sales-based royalties on net product sales of ORLADEYO. Interest expense is accrued using the effective interest rate method over the estimated period each of the related liabilities will be paid. This requires us to estimate the total amount of future royalty payments to be generated from product sales over the life of the agreements. We impute interest on the carrying values of each of the royalty financing obligations and record interest expense using an imputed effective interest rate. We reassess the expected royalty payments each reporting period and account for any changes through adjustments to the effective interest rates on a prospective basis. The assumptions used in determining the expected repayment terms of the debt and amortization periods of the issuance costs requires that we 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. Significant changes in forecasted net sales could materially impact each of the liability balances, interest expense and the time periods for repayment.
Asset Acquisitions and Acquired In-Process Research and Development
Accounting for asset acquisitions requires significant judgment, including the application of the screen test in accordance with ASC Topic 805, Business Combinations, and the valuation of acquired in‑process research and development (“IPR&D”). Determining whether substantially all of the fair value of the gross assets acquired is concentrated in a single identifiable asset requires estimates of relative fair values. During 2026, we acquired significant IPR&D related to the navenibart development program in connection with the Merger. The determination of the fair value of acquired IPR&D represents a critical accounting estimate because it requires significant management judgment and the use of unobservable inputs. Key assumptions include development timelines, probabilities of technical and regulatory success, expected future revenues, market conditions, and discount rates. In certain circumstances, we may conclude that an acquired IPR&D asset has no measurable fair value at the acquisition date based on market participant considerations. Changes in these assumptions or judgments could materially affect our accounting conclusions and amounts recognized in our condensed consolidated financial statements. See “Note 2—Acquisition of Astria Therapeutics, Inc.” in the Notes to the Condensed Consolidated Financial Statements in Part I, Item 1 of this report for additional information on the Merger.
Income Taxes
The liability method is used in our accounting for income taxes. Significant management judgment is required in determining any valuation allowance recorded against net deferred tax assets and uncertain tax positions. We have recorded a valuation allowance against substantially all potential tax assets, due to uncertainties in our 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 future earnings in each of the jurisdictions in which we operate and the period over which our deferred tax assets will be recoverable.
We account for uncertain tax positions in accordance with U.S. GAAP. Uncertain tax positions are recorded based upon certain recognition and measurement criteria. We re-evaluate uncertain tax positions and consider various factors, including, but not limited to, changes in tax law and the measurement of tax positions taken or expected to be taken in tax returns. We adjust the amount of the liability to reflect any subsequent changes in the relevant facts and circumstances surrounding the uncertain tax positions.
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Recent Accounting Pronouncements
“Note 1—Significant Accounting Policies and Concentrations of Risk” in the Notes to the Condensed Consolidated Financial Statements included in Part I, Item 1 of this report discusses accounting pronouncements recently issued or proposed but not yet required to be adopted.