Financial Analysis

Product revenue, net, was as follows: Three Months Ended June 30, Six Months Ended June 30, (In thousands) 2026 2025 2026 2025 Product revenue, net United States $ 40,917 $ 12,151 $ 59,768 $ 20,877 Rest of world 3,828 304 5,723 304 Total product revenue, net $ 44,745 $ 12,455 $ 65,491 $ 21,181

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Share-Based Payments 2023 Stock Incentive Plan and Inducement Grants In June 2023, our stockholders approved the 2023 Stock Incentive Plan, or the 2023 Plan. The 2023 Plan provides for the grant of incentive stock options, nonstatutory stock options, stock appreciation rights, restricted stock awards, restricted stock units, or RSUs, performance-based stock units, or PSUs, and other stock-based awards to employees, advisors, consultants and non-employee directors. Following the adoption of the 2023 Plan, we ceased granting equity awards under the 2013 Stock Incentive Plan, or the 2013 Plan. Any outstanding equity awards that were previously granted under the 2013 Plan continue to be governed by their terms. Following adoption of the 2013 Plan, we ceased granting equity awards under the 2007 Stock Incentive Plan, or the 2007 Plan. There are no outstanding equity awards under the 2007 Plan. In connection with the start of employment of our Chief Executive Officer and Chief Financial Officer in 2022, our Chief Commercial Officer in 2023, and our Chief Corporate Development and Strategy Officer in March 2025, our Board of Directors granted each of them equity awards in the form of stock options, RSUs and PSUs, which awards were made outside our equity incentive plans as inducements material to their respective entry into employment with us in accordance with Nasdaq Listing Rule 5635(c)(4). As of June 30, 2026, the maximum number of shares reserved under the 2013 Plan, the 2023 Pl …

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Fair Value Measurements We record cash equivalents and marketable securities at fair value. ASC 820, Fair Value Measurements and Disclosures , establishes a fair value hierarchy for those instruments measured at fair value that distinguishes between assumptions based on market data (observable inputs) and our own assumptions (unobservable inputs). The hierarchy consists of three levels: Level 1 Unadjusted quoted prices in active markets for identical assets or liabilities. Level 2 Quoted prices for similar assets and liabilities in active markets, quoted prices in markets that are not active, or inputs which are observable, directly or indirectly, for substantially the full term of the asset or liability. Level 3 Unobservable inputs that reflect our own assumptions about the assumptions market participants would use in pricing the asset or liability in which there is little, if any, market activity for the asset or liability at the measurement date. The following table summarizes our cash equivalents and marketable securities measured at fair value and by level on a recurring basis as of June 30, 2026: (In thousands) Level 1 Level 2 Level 3 Total Cash equivalents $ 30,098 $ 8,643 $ $ 38,741 Total cash equivalents 30,098 8,643 38,741 Marketable securities: U.S. Treasuries 251,421 251,421 Government securities 145,777 145,777 Corporate debt securities 467,985 467,985 Total marketable securities 865,183 865,183 Total cash equivalents and marketable securities $ 30,098 $ 873,826 …

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Leases Our building leases are comprised of office and laboratory space under non-cancelable operating leases. These lease agreements have remaining lease terms of approximately two years and contain various clauses for renewal at our option. The renewal options were not included in the calculation of the operating lease assets and the operating lease liabilities as the renewal options are not reasonably certain of being exercised. The lease agreements do not contain residual value guarantees. The components of lease expense and other information related to leases were as follows: Three Months Ended June 30, Six Months Ended June 30, (In thousands) 2026 2025 2026 2025 Operating lease costs $ 3,806 $ 3,806 $ 7,613 $ 7,613 Cash paid for amounts included in the measurement of operating lease liabilities 5,058 4,911 10,070 9,748 We have not entered into any material short-term leases or financing leases as of June 30, 2026. In arriving at the operating lease liabilities as of June 30, 2026 and December 31, 2025, we applied the weighted-average incremental borrowing rate of 5.7% for both periods over a weighted-average remaining lease term of 1.7 and 2.2 years, respectively. As of June 30, 2026, undiscounted minimum rental commitments under non-cancelable leases were as follows: (In thousands) Remaining 2026 $ 8,441 2027 20,755 2028 3,479 Undiscounted minimum rental commitments 32,675 Interest (1,504) Operating lease liabilities $ 31,171 We provided our landlord a security deposit …

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Recent Accounting Pronouncements In November 2024, the Financial Accounting Standards Board issued Accounting Standards Update, or ASU, 2024-03, Income Statement Reporting Comprehensive Income Expense Disaggregation Disclosures (Subtopic 220-40): Disaggregation of Income Statement Expenses , to improve disclosures around an entitys expenses. Upon adoption, companies will be required to disclose in the notes to the financial statements a disaggregation of certain expense categories included within the expense captions on the face of the income statement. The standard is effective for annual periods beginning after December 15, 2026 and interim periods beginning after December 15, 2027, with early adoption permitted, and can be applied either prospectively or retrospectively. We plan to adopt the standard in our 2027 annual period and are currently assessing its effect on our financial statement disclosures. …

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Product Revenue We generate product revenue from sales of PYRUKYND and AQVESME in the United States to a limited number of specialty distributors and specialty pharmacy providers, and from sales of PYRUKYND to Avanzanite and NewBridge outside of the United States, or collectively, the Customers. These Customers subsequently resell PYRUKYND and AQVESME to pharmacies or dispense PYRUKYND and AQVESME directly to patients. In addition to distribution agreements with Customers, we enter into arrangements with healthcare providers and payors that provide for government-mandated and/or privately-negotiated rebates, chargebacks and discounts with respect to the purchase of PYRUKYND and AQVESME. The performance obligation related to the sale of PYRUKYND and AQVESME is satisfied and revenue is recognized when the Customer obtains control of the product, which occurs at a point in time, typically upon delivery to the Customer. Product revenue, net, was as follows: Three Months Ended June 30, Six Months Ended June 30, (In thousands) 2026 2025 2026 2025 Product revenue, net United States $ 40,917 $ 12,151 $ 59,768 $ 20,877 Rest of world 3,828 304 5,723 304 Total product revenue, net $ 44,745 $ 12,455 $ 65,491 $ 21,181 Reserves for Variable Consideration Revenues from product sales are recorded at the net sales price, or transaction price, which includes estimates of variable consideration for which reserves are established and result from contractual adjustments, government rebates, retur …

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Segment Information Operating segments are identified as components of an enterprise about which separate discrete financial information is available for evaluation by the chief operating decision maker, or CODM, or decision-making group in making decisions on how to allocate resources and assess performance. Our CODM is our CEO. Our CEO views our operations and manages our business as one operating segment, which derives its revenues from the development and commercialization of therapies for patients with rare diseases. Our CEO manages and allocates resources to the operations of our company on a total company basis by assessing the overall level of resources available and how to best deploy these resources across functions and research and development projects that are in line with our long-term company-wide strategic goals. In making these decisions, our CEO uses consolidated financial information for purposes of evaluating performance, forecasting future period financial results, allocating resources and setting incentive targets. The CODM performs this assessment based on our consolidated net (loss) income. Through this analysis, the CODM assesses performance by comparing actual consolidated net (loss) income versus the budget, and then decides how to allocate resources to invest in our research and development programs. The measure of segment assets is reported on the condensed consolidated balance sheets as total assets. The following table contains additional informa …

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Summary of Significant Accounting Policies There have been no material changes to the significant accounting policies previously disclosed in our Annual Report on Form 10-K for the year ended December 31, 2025. Recent Accounting Pronouncements In November 2024, the Financial Accounting Standards Board issued Accounting Standards Update, or ASU, 2024-03, Income Statement Reporting Comprehensive Income Expense Disaggregation Disclosures (Subtopic 220-40): Disaggregation of Income Statement Expenses , to improve disclosures around an entitys expenses. Upon adoption, companies will be required to disclose in the notes to the financial statements a disaggregation of certain expense categories included within the expense captions on the face of the income statement. The standard is effective for annual periods beginning after December 15, 2026 and interim periods beginning after December 15, 2027, with early adoption permitted, and can be applied either prospectively or retrospectively. We plan to adopt the standard in our 2027 annual period and are currently assessing its effect on our financial statement disclosures. …

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