Mirum Pharmaceuticals, Inc. 10-Q

Item 1. Financial Statements. Mirum Pharmaceuticals, Inc. Condensed Consolidated Balance Sheets (Unaudited) (In thousands, except share and per share data) March 31, 2026 December 31, 2025 (Note 2) Assets Current assets: Cash and cash equivalents $ 324,907 $ 296,683 Short-term investments 61,352 86,644 Accounts receivable 133,113 123,330 Inventory 25,518 24,887 Prepaid expenses and other current assets 24,546 18,140 Total current assets 569,436 549,684 Restricted cash 1,733 1,482 Long-term investments 34,353 8,105 Property and equipment, net 2,412 1,862 Operating lease right-of-use assets 12,647 8,741 Intangible assets, net 254,154 260,921 Other assets 16,181 12,018 Total assets $ 890,916 $ 842,813 Liabilities and Stockholders’ Equity Current liabilities: Accounts payable $ 59,399 $ 9,614 Holdback liabilities, current 25,800 — Accrued expenses and other current liabilities 187,625 196,185 Total current liabilities 272,824 205,799 Operating lease liabilities, noncurrent 11,175 7,516 Convertible notes payable, net, noncurrent 310,251 309,797 Holdback liabilities, noncurrent 48,257 — Other liabilities 5,915 5,011 Total liabilities 648,422 528,123 Commitments and contingencies Stockholders’ equity: Preferred stock, $ 0.0001 par value; 10,000,000 shares authorized, and no shares issued and outstanding as of March 31, 2026 and December 31, 2025 — — Common stock, $ 0.0001 par value; 200,000,000 shares authorized; 60,926,165 and 51,896,391 shares issued and outstanding as of March 31, 2026 and December 31, 2025, respectively 6 5 Additional paid-in capital 1,700,215 981,878 Accumulated deficit ( 1,457,699 ) ( 667,544 ) Accumulated other comprehensive (loss) income ( 28 ) 351 Total stockholders’ equity 242,494 314,690 Total liabilities and stockholders’ equity $ 890,916 $ 842,813 The accompanying notes are an integral part of these unaudited condensed consolidated financial statements. 1 Table of Contents Mirum Pharmaceuticals, Inc. Condensed Consolidated Statements of Operations (Unaudited) (In thousands, except share and per share data) Three Months Ended March 31, 2026 2025 Revenue: Product sales, net $ 159,882 $ 111,585 Operating expenses: Cost of sales 28,805 23,018 Research and development 97,910 41,044 Acquired in-process research and development 726,302 5,000 Selling, general and administrative 96,330 57,706 Total operating expenses 949,347 126,768 Loss from operations ( 789,465 ) ( 15,183 ) Other income (expense): Interest income 3,401 3,023 Interest expense ( 3,616 ) ( 3,596 ) Other income, net — 2,108 Net loss before provision for income taxes ( 789,680 ) ( 13,648 ) Provision for income taxes 475 1,029 Net loss $ ( 790,155 ) $ ( 14,677 ) Net loss per share, basic and diluted $ ( 13.43 ) $ ( 0.30 ) Weighted-average shares of common stock used to compute net loss per share, basic and diluted 58,851,021 48,889,058 The accompanying notes are an integral part of these unaudited condensed consolidated financial statements. 2 Table of Contents Mirum Pharmaceuticals, Inc. Condensed Consolidated Statements of Comprehensive Loss (Unaudited) (In thousands) Three Months Ended March 31, 2026 2025 Net loss $ ( 790,155 ) $ ( 14,677 ) Other comprehensive (loss) income: Unrealized loss on available-for-sale investments ( 227 ) ( 23 ) Cumulative translation adjustments ( 152 ) 138 Comprehensive loss $ ( 790,534 ) $ ( 14,562 ) The accompanying notes are an integral part of these unaudited condensed consolidated financial statements. 3 Table of Contents Mirum Pharmaceuticals, Inc. Condensed Consolidated Statements of Stockholders’ Equity (Unaudited) (In thousands, except share amounts) Common Stock Additional Paid-In Capital Accumulated Deficit Accumulated Other Comprehensive Income (Loss) Total Stockholders’ Equity Shares Amount Balance as of December 31, 2025 51,896,391 $ 5 $ 981,878 $ ( 667,544 ) $ 351 $ 314,690 Issuance of common stock and pre-funded warrants in private placement, net of issuance costs of $ 8,600 3,385,149 — 259,948 — — 259,948 Issuance of common stock in connection with asset acquisition 4,404,190 1 415,270 — — 415,271 Stock-based compensation in connection with asset acquisition 112,872 — 10,643 — — 10,643 Issuance of common stock in connection with equity award plans 1,127,563 — 10,131 — — 10,131 Stock-based compensation — — 22,345 — — 22,345 Net loss — — — ( 790,155 ) — ( 790,155 ) Other comprehensive loss — — — — ( 379 ) ( 379 ) Balance as of March 31, 2026 60,926,165 $ 6 $ 1,700,215 $ ( 1,457,699 ) $ ( 28 ) $ 242,494 Common Stock Additional Paid-In Capital Accumulated Deficit Accumulated Other Comprehensive Income (Loss) Total Stockholders’ Equity Shares Amount Balance as of December 31, 2024 48,338,096 $ 5 $ 870,189 $ ( 644,181 ) $ ( 373 ) $ 225,640 Issuance of common stock in connection with equity award plans 1,112,939 — 6,359 — — 6,359 Conversion of convertible notes, net 188 — 6 — — 6 Stock-based compensation — — 15,813 — — 15,813 Net loss — — — ( 14,677 ) — ( 14,677 ) Other comprehensive income — — — — 115 115 Balance as of March 31, 2025 49,451,223 $ 5 $ 892,367 $ ( 658,858 ) $ ( 258 ) $ 233,256 The accompanying notes are an integral part of these unaudited condensed consolidated financial statements. 4 Table of Contents Mirum Pharmaceuticals, Inc. Condensed Consolidated Statements of Cash Flows (Unaudited) (In thousands) Three Months Ended March 31, 2026 2025 Operating activities Net loss $ ( 790,155 ) $ ( 14,677 ) Reconciliation of net loss to net cash used in operating activities: Stock-based compensation 36,202 15,803 Depreciation and amortization 6,874 6,052 Inventory reserves and firm commitment losses — 2,349 Amortization of debt discount and offering costs 454 433 Unrealized foreign exchange loss (gain) 1,098 ( 658 ) Non-cash lease expense 550 390 Fair value of common stock issued in connection with asset acquisition 415,271 — Change in fair value of holdback liabilities ( 998 ) — Other ( 278 ) ( 356 ) Change in operating assets and liabilities: Accounts receivable ( 12,886 ) ( 16,833 ) Prepaid and other current assets ( 6,630 ) ( 3,029 ) Inventory ( 1,920 ) ( 2,672 ) Other assets ( 4,796 ) ( 92 ) Accounts payable, accrued expenses and other liabilities 57,244 11,737 Holdback liabilities 71,790 — Operating lease liabilities ( 583 ) ( 408 ) Net cash used in operating activities ( 228,763 ) ( 1,961 ) Investing activities Purchase of investments ( 34,797 ) ( 33,526 ) Proceeds from maturities of investments 33,892 17,433 Purchase of property and equipment ( 664 ) ( 41 ) Payments made for additions to intangible assets ( 10,000 ) — Net cash used in investing activities ( 11,569 ) ( 16,134 ) Financing activities Proceeds from issuance of common stock and pre-funded warrants in private placement, net of issuance costs of $ 8,600 259,948 — Proceeds from issuance of common stock pursuant to equity plans 10,131 6,359 Net cash provided by financing activities 270,079 6,359 Effect of exchange rate on cash, cash equivalents and restricted cash ( 1,272 ) 1,055 Net increase (decrease) in cash, cash equivalents and restricted cash 28,475 ( 10,681 ) Cash, cash equivalents and restricted cash at beginning of period 298,165 222,928 Cash, cash equivalents and restricted cash at end of period $ 326,640 $ 212,247 Supplemental disclosure of cash flow information: Operating cash flows paid for operating lease $ 810 $ 583 Cash paid for income taxes $ 220 $ 162 Net operating liabilities, excluding cash acquired, assumed in connection with asset acquisition $ 9,561 $ — Non-cash operating, investing and financing activities: Right-of-use assets obtained in exchange for lease liabilities $ 4,472 $ — Decrease in ROU assets and lease liabilities due to lease modification $ — $ 649 Stock-based compensation capitalized to inventory $ 173 $ 212 Conversion of convertible notes, net into shares of common stock $ — $ 6 The accompanying notes are an integral part of these unaudited condensed consolidated financial statements. 5 Table of Contents Mirum Pharmaceuticals, Inc. Notes to Unaudited Condensed Consolidated Financial Statements (Unaudited) 1. Organization and Description of Business Mirum Pharmaceuticals, Inc. (the “Company”) was incorporated in the State of Delaware on May 2, 2018 , and is headquartered in Foster City, California. The Company is a biopharmaceutical company dedicated to transforming the treatment of rare diseases. The Company has three approved medicines: LIVMARLI® (maralixibat) (“Livmarli”), Cholbam® (cholic acid) capsules (“Cholbam”), and CTEXLI® (chenodiol) tablets (“Ctexli”). Livmarli is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (“ALGS”) in the United States (“U.S.”), the European Union (“EU”) and various other countries around the world and for cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (“PFIC”) in the U.S., Canada and Japan and for the treatment of PFIC in the EU. Cholbam is U.S. Food and Drug Administration (“FDA”) approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms of liver disease. In February 2025, the Company received FDA approval for chenodiol tablets for the treatment of cerebrotendinous xanthomatosis in adults, which is commercialized under the brand name Ctexli. Prior to the approval of Ctexli, chenodiol tablets were commercialized under the brand name Chenodal under a medical necessity recognition from the FDA. Cholbam and Ctexli, collectively, are referred to as the “Bile Acid Medicines”. The Company’s development pipeline consists of the clinical-stage product candidate volixibat, MRM-3379 and indication expansion opportunities for Livmarli. On January 23, 2026, the Company acquired Bluejay Therapeutics, Inc. (“Bluejay”). With the completion of the acquisition, the Company added worldwide rights to brelovitug, a late-stage, fully human monoclonal antibody for chronic hepatitis delta virus, a rare and severe liver disease. The Company commenced significant operations in November 2018. The Company views its operations and manages its business as one operating segment and has determined its operating segment on the same basis that it uses to evaluate its performance internally. Liquidity The Company has a limited operating history, has incurred significant operating losses since its inception, and the revenue and income potential of the Company’s business and market are unproven. As of March 31, 2026, the Company had an accumulated deficit of $ 1.5 billion and unrestricted cash, cash equivalents and investments of $ 420.6 million. The Company’s convertible notes are convertible at the option of the holders during the second quarter of 2026. If holders of the convertible notes elect to convert, the Company may elect to settle such conversions in cash, common stock or a combination of the two. The Company believes that its unrestricted cash, cash equivalents and investments of $ 420.6 million as of March 31, 2026, provide sufficient capital resources to continue its operations for at least twelve months from the issuance date of the accompanying unaudited condensed consolidated financial statements. 2. Summary of Significant Accounting Policies Basis of Presentation The accompanying unaudited condensed consolidated financial statements have been prepared in accordance with U.S. generally accepted accounting principles (“GAAP”) for interim financial information and pursuant to Form 10-Q and Article 10 of Regulation S-X of the Securities and Exchange Commission (“SEC”). Accordingly, the accompanying unaudited condensed consolidated financial statements do not include all of the information and notes required by GAAP for complete financial statements. The unaudited interim financial statements reflect all adjustments which, in the opinion of management, are necessary for a fair statement of the results for the periods presented. All such adjustments are of a normal and recurring nature. The unaudited condensed consolidated balance sheet as of December 31, 2025 has been derived from the audited consolidated financial statements at that date but does not include all information and footnotes required by GAAP for complete financial statements. The operating results presented in these unaudited condensed consolidated financial statements are not necessarily indicative of the results that may be expected for any future periods. 6 Table of Contents The accompanying unaudited condensed consolidated financial statements include the accounts of the Company and its wholly owned subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. These unaudited condensed consolidated financial statements should be read in conjunction with the audited consolidated financial statements and the notes thereto in the Company’s Annual Report on Form 10-K (“Annual Report”) for the fiscal year ended December 31, 2025, as filed with the SEC on February 25, 2026. Use of Estimates The preparation of consolidated financial statements in accordance with GAAP requires management to make estimates and assumptions that impact the reported amounts. These estimates and assumptions are based upon historical experience, knowledge of current events and various other factors 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 and the recording of revenue and expenses that are not readily apparent from other sources. Actual results could differ materially from those estimates. The Company’s unaudited condensed consolidated financial statements as of and for the three months ended March 31, 2026 reflect the Company’s estimates of the impact of the geopolitical and macroeconomic environment, including the impact of inflation, tariffs and trade tensions, and foreign exchange rate fluctuations. The duration and the scope of these conditions cannot be predicted; therefore, the extent to which these conditions will directly or indirectly impact the Company’s business, results of operations and financial condition, is uncertain. The Company is not aware of any specific event or circumstance that would require an update to its estimates, judgments and assumptions or a revision of the carrying value of the Company’s assets or liabilities as of the date of this filing. Reclassifications Amounts related to pre-commercialization milestone payments in connection with in-process research and development projects acquired with no alternative future use in a transaction accounted for as an asset acquisition in the statement of operations for the three months ended March 31, 2025 have been reclassified to conform to the current presentation. Significant Accounting Policies There have been no significant changes to the Company’s accounting policies during the three months ended March 31, 2026, as compared to the significant accounting policies described in Note 2 of the “Notes to Consolidated Financial Statements” in the Company’s audited consolidated financial statements included in the Annual Report. Cash, Cash Equivalents and Restricted Cash The Company considers all highly liquid investments that are readily convertible into cash without penalty and with original maturities of three months or less at the date of purchase to be cash equivalents. The carrying amounts reported in the unaudited condensed consolidated balance sheets for cash and cash equivalents are valued at cost, which approximate their fair value. The following table provides a reconciliation of cash, cash equivalents and restricted cash reported within the unaudited condensed consolidated balance sheets that together reflect the same amounts shown in the unaudited condensed consolidated statements of cash flows (in thousands): As of March 31, 2026 2025 Cash and cash equivalents $ 324,907 $ 211,822 Restricted cash 1,733 425 Total cash, cash equivalents, and restricted cash $ 326,640 $ 212,247 Investments The Company classifies all investments in securities as available-for-sale. Management determines the appropriate classification of its investments in securities at the time of purchase. Investments with original maturities beyond three months at the date of purchase and which mature at, or less than twelve months from the balance sheet date, are classified as a current asset. Investments are recorded at fair value, with unrealized gains and losses reported as accumulated other comprehensive income (loss) until realized, with the exception of any declines in fair value below the cost basis that are a result of a credit loss, which, if any, are reported in other income (expense), net in the current period through an allowance 7 Table of Contents for credit losses. Each reporting period, the Company evaluates whether declines in fair values of its available-for-sale securities below their cost basis are a result of credit loss or other factors and whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. This evaluation consists of several qualitative and quantitative factors regarding the severity and duration of the unrealized loss, the creditworthiness of the security issuers, as well as the Company’s ability and intent to hold the available-for-sale security until a forecasted recovery occurs. Additionally, the Company assesses whether it has plans to sell the security or it is more likely than not it will be required to sell any available-for-sale securities before recovery of its amortized cost basis. The cost of debt securities is adjusted for amortization of premiums and accretion of discounts to maturity. Such amortization and accretion, as well as interest and dividends, are included in interest income. Realized gains and losses from the sale of available-for-sale securities, if any, are determined on a specific identification basis and are also included in other income (expense). To date, the Company has not identified any declines in fair value of its investments related to credit loss. Concentrations of Credit Risk and Off-Balance Sheet Risk Financial instruments that potentially subject the Company to a concentration of credit risk consist of cash and cash equivalents, accounts receivable and investments. The Company limits the amount of credit exposure by investing cash that is not required for immediate operating needs in money market funds, government obligations, corporate debt securities and/or commercial paper. Additionally, the Company has established guidelines regarding diversification of its investments and their maturities, which are designed to maintain principal and maximize liquidity. To date, the Company has not experienced any losses associated with these credit risks and continues to believe that this exposure is not significant. The Company relies on a single third-party logistics provider (“3PL”) and a single specialty pharmacy for all of the Company’s sales of its approved medicines in each of the U.S. and Canada as well as a single 3PL outside of North America. The Company sources materials and services through several vendors. Certain materials are sourced from a single vendor. The loss of certain vendors could result in a temporary disruption of the Company’s commercialization efforts. As of March 31, 2026 and December 31, 2025, the Company did not have any customer who individually accounted for 10 % or more of accounts receivable. For the three months ended March 31, 2026 and 2025, the Company did not have revenue attributable to any one customer in excess of 10 % of sales. Accounts Receivable The Company has accounts receivable amounts due from product sales. The Company may also have accounts receivable amounts due from license agreements for milestones achieved, but not yet paid. Amounts payable to the Company are recorded as accounts receivable when the Company’s right to consideration is unconditional. The Company estimates the allowance for credit losses using the current expected credit loss model. Under this model, the allowance for credit losses reflects the Company’s estimate of lifetime expected credit losses. The Company evaluates the collectability of the cash flows based on the risk of loss over the contractual life, even when that risk is remote, based on judgments about the creditworthiness of its customers, historical experience and other relevant information that is available to the Company. There was no allowance for credit losses as of March 31, 2026. There was no bad debt expense for the three months ended March 31, 2026 and 2025. Product Sales, Net Revenues from product sales are recorded at the net sales price, or the transaction price, which may include fixed or variable consideration for discounts, government rebates, co-pay assistance, returns and other allowances that are offered within contracts with a customer relating to the sale of the Company’s approved medicines. Estimates of variable consideration are calculated using the actual product sales each reporting period and the nature of the variable consideration related to those sales. Overall, these estimates reflect the Company’s best estimate of the amount of consideration to which the Company expects to be entitled based on the terms of the contract. The amount of variable consideration that is included in the transaction price may be constrained and is included in product sales, net only to the extent that it is considered probable that a significant reversal in the amount of the cumulative revenue recognized will not occur when the uncertainty associated with the variable consideration is subsequently resolved. Estimates are reviewed and updated as additional information becomes known. Actual amounts of consideration ultimately received may differ materially from estimates. If actual results vary from estimates, the Company will adjust these estimates, which would affect product sales, net and earnings in the period such variances are adjusted. Significant categories of sales deductions are as follows: 8 Table of Contents Government Rebates : The Company records rebates payable under Medicaid and other government programs as a reduction of revenue at the time product revenues are generated. The Company’s rebate calculations may require estimates, including estimates of customer and payor mix and revenue projections, to determine which sales will be subject to rebates and the amount of such rebates. The Company updates its estimates and assumptions on a quarterly basis and records any necessary adjustments to revenue in the period identified. Government rebate payments vary in timing depending on the nature, and are generally quarterly or annually. Other Incentives : Other incentives include a branded co-pay assistance program for eligible patients with commercial insurance in the U.S. The branded co-pay assistance program assists commercially insured patients who have coverage for the Company’s approved medicines and is intended to reduce each participating patient’s portion of the financial responsibility of the purchase price up to a specified dollar amount of assistance. The calculation of the accrual for co-pay assistance is based upon an identification of claims and the cost per claims associated with product that has been recognized as revenue. The Company records amounts paid under the brand specific co-pay assistance program for each patient as a reduction of revenue from product sales. Product Returns : The Company records revenue for product sales, net of estimated product returns. Customers have limited return rights related only to the product’s damage or defect identified upon delivery of the product. The Company estimates the amount of product sales that may be returned and records the estimate as a reduction of revenue and a liability in the period the related product revenue is recognized. The Company relies on a specialty pharmacy for all of its sales of its approved medicines in each of the U.S. and Canada. Outside of North America, the Company’s approved medicines are sold to its authorized distributors, licensed partners or directly to government purchasers or hospitals, which act as end users. For revenues from distributors and the Company’s licensed partner in Japan, Takeda, the Company records net product sales at the time control of the product is transferred, based on the estimated variable consideration. The transaction price, which may include fixed or variable consideration may be subject to constraint and is included in the product sales price only to the extent that it is probable that a significant reversal of the amount of the cumulative revenue recognized will not occur in a future period. The Company recognizes its best estimate of the consideration expected to be received when control of the inventory is transferred. Such estimates may be complex and include estimates as to if and when the distributors and licensed partner’s sales in the market will occur. Estimates are reviewed and updated as additional information, including in-market sales information of the Company’s authorized distributors and licensed partners, becomes known. Actual amounts may ultimately differ from the Company’s estimates. If actual results vary, the Company adjusts these estimates, which could have an effect on earnings in the period of adjustment. The Company is entitled to payment in connection with the supply of product under standard industry payment terms. Actual consideration amounts are determined and settled generally quarterly or annually. If the consideration received or receivable exceeds the Company’s estimates of product sales, the Company recognizes a liability. If the estimated product sales exceed the payment received or receivable, the Company records a receivable or contract asset related to the amounts not yet collected, depending on the circumstances. As of March 31, 2026 and December 31, 2025, the Company did not have any contract assets and recorded a liability of $ 3.6 million related to consideration received in excess of the Company’s estimated net product sales. Liabilities associated with sales deductions are included in accrued expenses and other current liabilities or in other liabilities on the accompanying unaudited condensed consolidated balance sheets depending on contractual settlement timelines. The following table represents total revenues and disaggregates Product sales, net by approved medicine (in thousands): Three Months Ended March 31, 2026 2025 Product sales, net: Livmarli $ 113,804 $ 73,224 Bile Acid Medicines 46,078 38,361 Total product sales, net $ 159,882 $ 111,585 9 Table of Contents The following table sets forth Product sales, net by geographic area based on the ship-to location (in thousands): Three Months Ended March 31, 2026 2025 United States $ 127,927 $ 86,922 Rest of the world 31,955 24,663 Total product sales, net $ 159,882 $ 111,585 Acquired In-Process Research and Development Acquired in-process research and development expenses are recorded when incurred and reflect costs of externally-developed in-process research and development (“IPR&D”) projects, acquired directly in a transaction other than a business combination, that do not have an alternative future use, including upfront and pre-commercialization milestone payments related to various in-licensing arrangements and the costs of rights to IPR&D projects. Foreign Currency The unaudited condensed consolidated financial statements are presented in U.S. dollars. The functional currency for most of the Company’s foreign subsidiaries is their local currency. Balance sheet accounts of international subsidiaries are translated at the current exchange rates as of the end of each accounting period. Income statement items are translated at average exchange rates for the period. The resulting translation adjustments are recorded as a separate component of stockholders’ equity. Foreign currency transaction gains and losses are included in other income (expense), net in the unaudited condensed consolidated statements of operations. Transaction gains and losses result primarily from fluctuations in exchange rates when intercompany receivables and payables are denominated in currencies other than the functional currency of the Company’s subsidiary that recorded the transaction. Unrealized foreign exchange gains and losses amounted to a $ 1.1 million loss for the three months ended March 31, 2026 and a $ 0.7 million gain for the three months ended March 31, 2025. Realized foreign exchange losses were not material for the three months ended March 31, 2026 and 2025. Net Loss Per Share Basic net loss per share is computed by dividing net loss by the weighted-average shares of common stock outstanding for the period, without consideration for potentially dilutive securities. The pre-funded warrants issued in January 2026 (refer to Note 10) were included in the calculation of basic weighted-average shares of common stock used to compute net loss per share for the three months ended March 31, 2026 as they are issuable for immaterial cash consideration. Diluted net loss per share is computed by dividing the net loss by the weighted-average shares of common stock and potentially dilutive securities outstanding for the period determined using the treasury-stock and if-converted methods. Diluted net loss per share excludes the potential impact of the Company’s common stock subject to repurchase, common stock options, restricted stock units, contingently issuable employee stock purchase plan shares, common stock issuable upon conversion of convertible notes and contingently issuable shares for holdback liabilities to the extent their effect would be anti-dilutive. The following table sets forth the computation of basic and diluted net loss per share for the three months ended March 31, 2026 and 2025 (in thousands, except share and per share amounts): Three Months Ended March 31, 2026 2025 Numerator: Net loss $ ( 790,155 ) $ ( 14,677 ) Denominator: Weighted-average shares of common stock outstanding 58,445,732 48,889,058 Add: weighted average of common stock to be issued upon exercise of pre-funded warrants 405,289 — Weighted average shares of common stock used to compute net loss per share, basic and diluted 58,851,021 48,889,058 Net loss per share, basic and diluted $ ( 13.43 ) $ ( 0.30 ) 10 Table of Contents The following outstanding potential dilutive shares have been excluded from the calculation of diluted net income (loss) per share for the periods presented due to their anti-dilutive effect: Three Months Ended March 31, 2026 2025 Options to purchase common stock and restricted stock units 11,659,300 12,712,908 Common stock issuable upon conversion of convertible notes 9,963,490 9,964,058 Indemnity holdback 522,375 — Employee stock purchase plan contingently issuable 41,537 51,465 Total 22,186,702 22,728,431 Acquisitions, including Intangible Assets and Contingent Consideration Acquisitions of businesses are accounted for using the acquisition method of accounting. The Company allocates the purchase price of acquired businesses to the tangible and intangible assets acquired and liabilities assumed based on their estimated fair values on the acquisition date. The purchase price allocation process requires management to make significant estimates and assumptions, especially at the acquisition date with respect to intangible assets and acquired IPR&D assets. Any excess of the purchase price over the estimated fair values of the net assets acquired is recognized as goodwill. Intangible assets are measured at their fair values as of the acquisition date or, in the case of commercial milestone payments, the date they become due. The evaluation of intangible assets includes assessing the amortization period for which the asset is expected to contribute to the future cash flows of the Company. Intangible assets with finite useful lives are amortized over their estimated useful lives on a straight-line basis. The Company tests its finite lived intangible assets for impairment annually or if events or changes in circumstances indicate that it is more likely than not that the asset is impaired. If it is determined that the asset is impaired, the carrying value is written down to its estimated fair value, with the related impairment charge recognized in the consolidated statements of operations in the period in which the impairment occurs. In connection with certain acquisitions, the Company may be required to pay future consideration that is contingent upon the achievement of specified development, regulatory approval or sales-based milestone events. Contingent consideration resulting from a business combination is recorded at its fair value on the acquisition date. Each reporting period thereafter, these obligations are revalued and increases or decreases in their fair value are recorded in the statement of operations until such time that the payment is made. If it is determined that the net assets acquired do not meet the definition of a business combination under the acquisition method of accounting or if substantially all of the fair value of the gross assets acquired is concentrated in a group of similar identifiable assets, the transaction is accounted for as an asset acquisition using the cost accumulation method. In an asset acquisition, no goodwill is recognized and contingent consideration generally is not recognized at the acquisition date. Upfront payments allocated to IPR&D projects at the acquisition date and subsequent pre-commercialization milestone payments are expensed as incurred in the statement of operations unless there is an alternative future use. Recently Adopted Accounting Pronouncements In July 2025, the FASB issued ASU 2025-05, Financial Instruments - Credit Losses (Topic 326): Measurement of Credit Losses for Accounts Receivable and Contract Assets (“ASU 2025-05”). The new guidance provides a practical expedient related to the estimation of expected credit losses for current accounts receivable and current contract assets that arise from transactions accounted for under FASB Accounting Standards Codification 606. The Company adopted the provisions of the amendments as of January 1, 2026, and elected the practical expedient provided to all companies. The adoption of this new accounting standard did not have a material impact on the Company’s consolidated financial statements. Recent Accounting Pronouncements Not Yet Adopted From time to time, new accounting pronouncements are issued by the Financial Accounting Standards Board (“FASB”) or other standard setting bodies and adopted by the Company as of the specified effective date. Unless otherwise discussed, the Company believes that the impact of recently issued standards that are not yet effective will not have a material impact on the accompanying unaudited condensed consolidated financial statements and disclosures. In November 2024, the FASB issued ASU 2024-03, Income Statement - Reporting Comprehensive Income - Expense Disaggregation Disclosures (Subtopic 220-40): Disaggregation of Income Statement Expenses (“ASU 2024-03”), 11 Table of Contents which requires disclosure of disaggregated information about specific categories underlying certain income statement expense line items in the footnotes to the financial statements for both annual and interim periods. Subsequently in January 2025, the FASB issued ASU 2025-01, Income Statement - Reporting Comprehensive Income - Expense Disaggregation Disclosures (Subtopic 220-40): Clarifying the Effective Date (“ASU 2025-01”) to clarify the effective date of ASU 2024-03. ASU 2024-03 is effective for fiscal years beginning after December 15, 2026, and interim periods within annual reporting periods beginning after December 15, 2027. Early adoption is permitted. The Company is currently assessing the impact that adopting this new accounting standard will have on its consolidated financial statements. In September 2025, the FASB issued ASU 2025-06, Intangibles - Goodwill and Other - Internal-Use Software (Subtopic 350-40): Targeted Improvements to the Accounting for Internal-Use Software (“ASU 2025-06”), to modernize the accounting guidance for the costs to develop software for internal use by aligning it with current development practices, especially agile and iterative methods. The guidance removes references to development stages and clarifies when capitalization of software costs should occur. The new guidance also clarifies disclosure requirements for all capitalized internal-use software costs. ASU 2025-06 is effective for annual reporting periods beginning after December 15, 2027, and interim reporting periods within those annual reporting periods. Early adoption is permitted as of the beginning of an annual reporting period. The Company is currently assessing the impact that adopting this new accounting standard will have on its consolidated financial statements. 3. Fair Value Measurements Financial assets and liabilities subject to fair value measurements on a recurring basis and the level of inputs used in such measurements by major security type are presented in the following table (in thousands): March 31, 2026 Level 1 Level 2 Level 3 Total Financial assets: Money market funds $ 261,459 $ — $ — $ 261,459 Corporate debt securities — 75,751 — 75,751 U.S. government bonds — 9,987 — 9,987 Agency bonds — 8,972 — 8,972 Commercial paper — 995 — 995 Total financial assets $ 261,459 $ 95,705 $ — $ 357,164 Financial liabilities: Indemnification equity holdback — — 48,257 48,257 Total financial liabilities $ — $ — $ 48,257 $ 48,257 December 31, 2025 Level 1 Level 2 Level 3 Total Financial assets: Money market funds $ 254,376 $ — $ — $ 254,376 Corporate debt securities — 72,277 — 72,277 U.S. government bonds — 17,016 — 17,016 Commercial paper — 5,456 — 5,456 Total financial assets $ 254,376 $ 94,749 $ — $ 349,125 The carrying amounts of certain financial instruments such as cash and cash equivalents, restricted cash, accounts receivable, prepaid expenses and other current assets, accounts payable and accrued expenses as of March 31, 2026 and December 31, 2025 approximate their related fair values due to their short-term nature. Money market funds are highly liquid investments and are actively traded. The pricing information on these investment instruments is readily available and can be independently validated as of the measurement date. This approach results in the classification of these securities as Level 1 of the fair value hierarchy. Certain financial instruments classified within Level 2 of the fair value hierarchy include the types of instruments that trade in markets that are not considered to be active, but are valued based on quoted market prices, broker or dealer quotations, or alternative pricing sources with reasonable levels of price transparency. The Company reviews trading activity and pricing for these investments as of each measurement date. 12 Table of Contents Holdback Liabilities In January 2026, in connection with the acquisition of Bluejay (refer to Note 6 for further information), the Company recorded at fair value liabilities related to the Company’s common stock issuable upon satisfaction of certain indemnification obligations. The Company assesses the fair value of the liabilities each reporting period until resolution of the related contingency with changes in fair value recorded in other income (expense), net in the accompanying unaudited condensed consolidated statements of operations. The following table provides a summary of the changes in the estimated fair value, which are classified as Level 3 of the fair value hierarchy (in thousands): Indemnification Equity Holdback (1) Balance at January 1, 2026 $ — Initial recognition 49,255 Change in fair value ( 998 ) Balance at March 31, 2026 $ 48,257 __________________ (1) Fair value estimated based on the value of the Company’s common stock in addition to the probability of indemnification adjustments being incurred. The Indemnification Equity Holdback liability is included in Holdback liabilities, noncurrent in the accompanying unaudited condensed consolidated balance sheets as of March 31, 2026. 4. Financial Instruments The fair value and amortized cost of cash equivalents and available-for-sale investments by major security type are presented in the following table (in thousands): March 31, 2026 Amortized Cost Unrealized Gain Unrealized Loss Estimated Fair Value Cash equivalents and investments: Money market funds $ 261,459 $ — $ — $ 261,459 Corporate debt securities 75,820 24 ( 93 ) 75,751 U.S. government bonds 9,976 11 — 9,987 Agency bonds 9,000 — ( 28 ) 8,972 Commercial paper 995 — — 995 Total cash equivalents and investments $ 357,250 $ 35 $ ( 121 ) $ 357,164 Classified as: Cash equivalents $ 261,459 Short-term investments 61,352 Long-term investments 34,353 Total cash equivalents and investments $ 357,164 13 Table of Contents December 31, 2025 Amortized Cost Unrealized Gain Unrealized Loss Estimated Fair Value Cash equivalents and investments: Money market funds $ 254,376 $ — $ — $ 254,376 Corporate debt securities 72,170 107 — 72,277 U.S. government bonds 16,984 32 — 17,016 Commercial paper 5,455 1 — 5,456 Total cash equivalents and investments $ 348,985 $ 140 $ — $ 349,125 Classified as: Cash equivalents $ 254,376 Short-term investments 86,644 Long-term investments 8,105 Total cash equivalents and investments $ 349,125 As of March 31, 2026, the remaining contractual maturities of available-for-sale debt securities were as follows (in thousands): Estimated Fair Value Due within one year $ 61,352 One to two years 34,353 Total $ 95,705 During the three months ended March 31, 2026 and 2025, there have been no significant realized gains or losses on available-for-sale investments, no investments have been in a continuous unrealized loss position for more than 12 months, and the Company did not recognize any credit losses on these securities. 5. Balance Sheet Components Inventory Inventory consists of the following (in thousands): March 31, 2026 December 31, 2025 Raw materials $ 2,612 $ 2,898 Work in progress 19,488 18,473 Finished goods 3,418 3,516 Total inventory $ 25,518 $ 24,887 Intangible Assets, Net The components of the Company’s intangible assets were as follows (in thousands, except for weighted-average remaining amortization period): March 31, 2026 Gross Carrying Value Accumulated Amortization Net Carrying Amount Weighted-Average Remaining Amortization Period (Years) Commercial milestones $ 94,000 $ ( 13,594 ) $ 80,406 11.3 Developed technology 226,620 ( 53,007 ) 173,613 9.5 Assembled workforce 970 ( 835 ) 135 0.4 Total intangible assets $ 321,590 $ ( 67,436 ) $ 254,154 10.0 14 Table of Contents December 31, 2025 Gross Carrying Value Accumulated Amortization Net Carrying Amount Weighted-Average Remaining Amortization Period (Years) Commercial milestones $ 94,000 $ ( 11,660 ) $ 82,340 11.5 Developed technology 226,620 ( 48,255 ) 178,365 9.7 Assembled workforce 970 ( 754 ) 216 0.7 Total intangible assets $ 321,590 $ ( 60,669 ) $ 260,921 10.3 Amortization expense was $ 6.8 million and $ 6.0 million for the three months ended March 31, 2026 and 2025, respectively. Amortization expense was included in cost of sales in the accompanying unaudited condensed consolidated statements of operations. The following table summarizes the estimated future amortization expense associated with the Company’s intangible assets as of March 31, 2026 (in thousands): Amount 2026 (remaining nine months) $ 20,191 2027 26,742 2028 26,742 2029 26,742 2030 26,742 Thereafter 126,995 $ 254,154 Accrued Expenses Accrued expenses consist of the following (in thousands): March 31, 2026 December 31, 2025 Accrued sales deductions and other $ 60,689 $ 51,375 Accrued compensation and related benefits 35,432 47,276 Accrued royalties payable 15,221 16,208 Accrued professional service fees 22,578 17,292 Accrued clinical trials 25,692 13,959 Accrued contract manufacturing and non-clinical costs 18,283 8,721 Accrued interest 5,270 2,108 Accrued loss on firm purchase commitments 2,107 2,107 Operating lease liabilities, current 2,353 2,139 Accrued milestone payment — 35,000 Total accrued expenses and other current liabilities $ 187,625 $ 196,185 Other liabilities Other liabilities consist of the following (in thousands): March 31, 2026 December 31, 2025 Noncurrent accrued sales deductions $ 4,238 $ 3,586 Noncurrent accrued loss on firm purchase commitments 1,425 1,425 Other 252 — Total other liabilities $ 5,915 $ 5,011 15 Table of Contents 6. Asset Acquisitions Asset Purchase Agreement with Bluejay Therapeutics, Inc. On January 23, 2026 (the “Closing Date”), the Company completed the acquisition of Bluejay (the “Bluejay Acquisition”). The aggregate merger consideration transferred and transferrable related to the Bluejay Acquisition was comprised of (i) upfront equity consideration of 4,517,062 shares of the Company’s common stock, after deduction to satisfy applicable taxes, (ii) upfront cash consideration of $ 241.2 million, net of cash acquired, (iii) equity consideration holdback of up to 522,375 shares of the Company’s common stock and cash consideration holdback of up to $ 24.8 million, both payable to Bluejay security holders 12 months after the Closing Date, subject to reduction for potential indemnification and other obligations of Bluejay and, in certain cases, to satisfy applicable taxes, and (iv) up to $ 1.0 million of cash consideration holdback payable to Bluejay security holders upon finalization of working capital adjustments, which are estimated to be paid approximately four months from the closing of the acquisition. Additionally, the Company is obligated to pay up to an aggregate amount of $ 200.0 million upon the achievement of certain commercial milestones. The Company accounted for the Bluejay Acquisition as an asset acquisition, as substantially all of the fair value of the gross assets acquired was concentrated in a single IPR&D asset. The net assets acquired were measured and recognized as an allocation of the transaction price based on their relative fair values as of the transaction date. The acquired IPR&D and related assembled workforce were determined to have no alternative future use and, accordingly, the cost of the acquisition of those assets were recorded as acquired in-process research and development expense as of the acquisition date. The Company estimated the fair value of the IPR&D asset using the discounted cash flow method. This approach incorporates estimates and assumptions related to the forecasted results including, but not limited to, estimates and assumptions regarding revenues, expenses, and discount rate to estimate future cash flows. During the three months ended March 31, 2026, the Company recorded a $ 726.3 million charge, representing an acquired IPR&D asset with no alternative future use, to acquired in-process research and development, as well as stock-based compensation of $ 34.7 million related to unvested Bluejay employee stock awards which were accelerated in contemplation of the Bluejay Acquisition, with $ 19.3 million recorded in selling, general and administrative expenses and $ 15.4 million in research and development expenses in the unaudited condensed consolidated statements of operations. In connection with the Bluejay acquisition, the Company recorded $ 44.8 million of net assets acquired. The fair value of the consideration of approximately $ 771.1 million, inclusive of the transaction costs incurred in connection with the Bluejay Acquisition, is summarized as follows (in thousands): Equity consideration $ 415,271 Equity holdback - indemnity 47,112 Cash consideration, including cash paid in lieu of equity to satisfy taxes 274,814 Cash holdback - indemnity 23,721 Cash holdback - working capital 957 Transaction costs 9,201 Total purchase consideration $ 771,076 The cash consideration of $ 274.8 million in the table above does not include the net effect of the cash and cash equivalents of $ 54.3 million acquired as part of the transaction. The above noted stock-based compensation is comprised as follows (in thousands): Equity consideration $ 10,643 Cash consideration, including cash paid in lieu of equity to satisfy taxes 20,742 Equity holdback - indemnity 2,143 Cash holdback - indemnity and working capital 1,122 Total $ 34,650 16 Table of Contents Allocation of the consideration transferred to the net assets acquired was as follows (in thousands): Assets acquired: IPR&D $ 724,006 Assembled workforce 2,296 Cash and cash equivalents 54,335 Prepaid expenses and other current assets 4,258 Operating lease right-of-use asset 1,113 Other non-current assets 3,888 Total assets acquired $ 789,896 Liabilities assumed: Accounts payable 1,589 Accrued expenses and other current liabilities 16,616 Operating lease liabilities, noncurrent 615 Total liabilities assumed $ 18,820 Net assets acquired $ 771,076 The allocated fair value of the acquired IPR&D and assembled workforce is a permanently non-deductible item for tax purposes. Entities affiliated with Frazier Life Sciences, which are associated with a member of the Company’s board of directors, were Bluejay security holders. Novartis License Through the Bluejay Acquisition, the Company was assigned a license agreement with Novartis Pharma AG (“Novartis”) pursuant to which the Company obtained a worldwide license to develop and commercialize brelovitug from Novartis in June 2021. Under the terms of the license agreement, the Company is obligated to pay up to $ 4.0 million in remaining development milestones and up to $ 27.0 million in commercial milestones. The Company is also obligated to pay commercial royalties of mid-single digit percent of sales to Novartis. In addition, the license agreement contained a $ 4.0 million business milestone payment in the event of a change of control which was payable upon the closing of the Bluejay Acquisition in January 2026. The accrued milestone was included in liabilities assumed at the closing of the Bluejay Acquisition. Through March 31, 2026, the Company paid the $ 4.0 million change of control payment and no further milestones have been achieved. License Agreement with Enthorin Therapeutics, LLC In October 2024, the Company entered into a license agreement with Enthorin Therapeutics, LLC and Dart Neuroscience LLC (“Enthorin”), under which the Company acquired the worldwide rights to develop, manufacture and commercialize a compound designated as ENT-3379, renamed MRM-3379, in exchange for an upfront payment of $ 7.5 million and up to an additional $ 217.5 million upon the achievement of regulatory and sales-based milestones as well as mid-single digit percent royalties on any future sales of MRM-3379. Through March 31, 2026, no milestones have been achieved. Asset Purchase Agreement with Travere Therapeutics, Inc. On August 31, 2023, the Company completed the acquisition of assets of Travere Therapeutics, Inc. (“Travere”). In accordance with the terms and conditions of the Asset Purchase Agreement entered into with Travere, the Company purchased from Travere substantially all of the assets related to its business of development, manufacturing (including synthesis, formulation, finishing or packaging) and commercialization of the Bile Acid Medicines. The Company paid $ 210.4 million upon closing of the transaction, and up to an additional $ 235.0 million is payable upon the achievement of certain milestones based on specified amounts of annual net sales of the Bile Acid Medicines. As of March 31, 2026, the Company recorded $ 25.0 million in accounts payable for the achievement of product sales milestones associated with this agreement. The Company is obligated to pay tiered royalties, based on licensing agreements acquired with the Bile Acid Medicines, with rates ranging from high single digit to mid-teens based on net sales of the Bile Acid Medicines. 17 Table of Contents Assignment and License Agreement with Shire International GmbH (Takeda) In November 2018, the Company entered into an Assignment and License Agreement (the “Shire Agreement”) with Shire International GmbH (“Shire”), which was subsequently acquired by Takeda Pharmaceutical Company Limited (“Takeda”). Under the terms of the Shire Agreement, Shire granted the Company an exclusive, royalty bearing worldwide license to develop and commercialize its two product candidates, Livmarli and volixibat. As part of the Shire Agreement, the Company was assigned license agreements held by Shire with Satiogen Pharmaceuticals, Inc. (“Satiogen”), Pfizer Inc. (“Pfizer”) and Sanofi-Aventis Deutschland GmbH (“Sanofi”). The Company has the right to sublicense under the Shire Agreement and additionally has the right to sublicense under the Satiogen, Pfizer and Sanofi licenses subject to the terms of those license agreements. The Company is obligated to pay Shire up to an aggregate of $ 109.5 million upon the achievement of certain clinical development and regulatory milestones for Livmarli in certain indications and an additional $ 25.0 million upon regulatory approval of Livmarli for each and every other indication. In addition, the Company is required to pay up to an aggregate of $ 30.0 million upon the achievement of certain clinical development and regulatory milestones for volixibat solely for the first indication sought. Upon commercialization, the Company is obligated to pay Shire commercial milestones on total licensed products up to an aggregate of $ 30.0 million. Through March 31, 2026 under this agreement, the Company paid or accrued $ 101.5 million for the achievement of various clinical development, regulatory and commercial milestones. The Company is also obligated to pay tiered royalties with rates ranging from low double-digits to mid-teens based upon annual worldwide net sales for all licensed products; however, these royalties are reduced in part by royalties due under the Satiogen and Sanofi licenses, as discussed below, related to Livmarli and volixibat, as applicable. The Company’s royalty obligations will continue on a licensed product-by-licensed product and country-by-country basis until the later to occur of the expiration of the last valid claim in a licensed patent covering the applicable licensed product in such country, expiration of any regulatory exclusivity for the licensed product in a country and ten years after the first commercial sale of a licensed product in such country. During the three months ended March 31, 2025, the Company achieved the $ 10.0 million regulatory milestone associated with the approval of Livmarli for the treatment of cholestatic pruritus in patients with PFIC five years of age and older (now twelve months of age and older) by the FDA and the $ 10.0 million regulatory milestone associated with the approval of Livmarli for the treatment of cholestatic pruritus in patients with PFIC three months and older by the European Medicines Agency, both of which were capitalized as intangible assets. During the three months ended March 31, 2025, the Company achieved a $ 5.0 million development milestone related to maralixibat, which was recognized in acquired research and development expense in the accompanying unaudited condensed consolidated statements of operations. In addition, as of December 31, 2025, the Company accrued $ 10.0 million for the achievement of a commercial milestone associated with product sales, which was recognized as an intangible asset. There were no volixibat development and regulatory milestones achieved during the three months ended March 31, 2026 and 2025. Pfizer License Through the Shire Agreement, the Company was assigned a license agreement with Pfizer pursuant to which the Company obtained an exclusive, worldwide license to certain Pfizer know-how with a right to sublicense. Upon commercialization of any product utilizing the licensed product, the Company is required to pay to Pfizer a low single-digit royalty on net sales of product sold by the Company, its affiliates or sublicensees. The Company’s royalty obligations continue on a licensed product-by-licensed product basis until the eighth anniversary of the first commercial sale of such licensed product anywhere in the world. Sanofi License Through the Shire Agreement, the Company was assigned a license agreement with Sanofi pursuant to which the Company obtained an exclusive, worldwide license to certain patents and know-how with the right to sublicense to a third party subject to certain financial considerations. The Company is obligated to pay up to an aggregate of $ 36.0 million upon the achievement of certain regulatory, commercialization and product sales milestones. Additionally, upon commercialization, the Company is required to pay tiered royalties in the mid to high single-digit range based upon net sales of licensed products sold by the Company and sublicensees in a calendar year, subject to adjustments in certain circumstances. The Company’s royalty obligations continue on a licensed product-by-licensed product and country-by-country basis until the later to occur of the expiration of the last valid claim in a licensed patent covering the applicable licensed product in such country and ten years after the first commercial sale of a licensed product in such country. Royalty obligations under the Sanofi license are creditable against the royalty obligations to Shire under the Shire Agreement. The Company has not paid milestone payments pursuant to this agreement for the periods presented. Through March 31, 2026, no milestones have been achieved. 18 Table of Contents 7. License Agreements Licensing Agreement with Takeda In September 2021, the Company entered into an exclusive licensing agreement with Takeda for the development and commercialization of Livmarli in Japan for ALGS, PFIC, and BA. Further, in October 2024, the parties entered into a commercial supply agreement. Under the terms of the agreements, Takeda is responsible for development and commercialization of Livmarli for licensed indications in Japan, while the Company is responsible for commercial supply to Takeda. In accordance with the agreements, in exchange for commercial inventory supply, the Company is eligible to receive a percentage of Takeda’s net sales, which range from high double digits declining to mid double digits over the first four years from commercial launch and thereafter remains at mid double digits. The Company records net product sales at the time control of the product is transferred, based on the estimated variable consideration. 8. Leases The Company has operating leases for office spaces in various global locations, including its headquarters in Foster City, California, the lease for which was entered into in January 2024 with an initial lease term of approximately five years (the “Initial Lease”). In January 2026, the Company amended the Initial Lease (the “Amended Agreement”) to expand its headquarters by approximately 19,400 square feet (the “Expanded Space”) with a lease term of approximately five years from the commencement date. The Amended Agreement also extends the lease term of the Initial Lease to be coterminous with the Expanded Space. The extension of the lease term of the Initial Lease resulted in the addition of $ 3.4 million of right-of-use assets and lease liabilities during the three months ended March 31, 2026. The addition of the Expanded Space will result in approximately $ 5.8 million of incremental base rent payments over the lease term at the time of lease commencement. The following tables contain a summary of other information and the undiscounted future minimum payments pertaining to the Company’s operating leases that had commenced as of the end of the periods presented: March 31, 2026 Weighted-average incremental borrowing rate 7.5 % Weighted-average remaining lease term (in years) 5.1 years Undiscounted Rent Payments as of March 31, 2026 (in thousands) 2026 (remaining nine months) $ 2,437 2027 3,449 2028 2,883 2029 2,950 2030 2,723 Thereafter 1,821 Total undiscounted lease payments 16,263 Less: imputed interest ( 2,735 ) Total lease liability $ 13,528 Rent expense was $ 0.8 million and $ 0.6 million for the three months ended March 31, 2026 and 2025, respectively. Variable lease payments for operating expenses for the three months ended March 31, 2026 and 2025 were not material. 19 Table of Contents 9. Convertible Notes Except as described below, the Company’s convertible notes are described in Note 10 of the “Notes to Consolidated Financial Statements” in the Annual Report. The convertible notes consisted of the following (in thousands): March 31, 2026 December 31, 2025 Principal amount $ 316,226 $ 316,226 Unamortized debt discount and issuance costs ( 5,975 ) ( 6,429 ) Net carrying amount $ 310,251 $ 309,797 For the fourth quarter of 2024, for each of the quarters of 2025 and for the first quarter of 2026, the last reported sale price of the Company’s common stock exceeded 130 % of the conversion price of the 4.00 % Convertible Senior Notes due 2029 (the “Notes”) for more than 20 trading days during the 30 consecutive trading days ended on each respective quarter end. As a result, for the quarterly period ended March 31, 2026, the Notes were, and for the quarterly period ending June 30, 2026, the Notes are, convertible at the option of the holders of the Notes. During the three months ended March 31, 2026 and through the date of this filing, the amount of the principal balance of the Notes that has been converted was not material. The Company incurred $ 10.9 million of transaction costs related to the issuance of the Notes, which are being amortized to interest expense over the term of the Notes using the effective interest method. As of March 31, 2026, the remaining amortization period of the debt discount was approximately 3.1 years and the effective interest on the Notes was 4.6 %. The following table sets forth interest expense recognized related to the Notes (in thousands): Three Months Ended March 31, 2026 2025 Coupon interest expense $ 3,162 $ 3,163 Amortization of debt discount and issuance costs 454 433 Total interest expense on convertible notes $ 3,616 $ 3,596 As of March 31, 2026 and December 31, 2025, the estimated fair value of the Notes was $ 946.3 million and $ 818.5 million, respectively. The fair values were determined based on the quoted price of the convertible notes in an inactive market on the last trading day of the reporting period and have been classified as Level 2 in the fair value hierarchy. 10. Stockholders’ Equity Common Stock On January 23, 2026, immediately following the consummation of the Bluejay Acquisition, the Company completed the private placement of 3,385,149 shares of the Company’s common stock (“PIPE Shares”) and pre-funded warrants to purchase 536,412 shares of the Company’s common stock (the “Warrant Shares”) for aggregate net proceeds of approximately $ 259.9 million, after estimated offering expenses of $ 8.6 million. The purchase price per PIPE Share was $ 68.48 and the purchase price per pre-funded warrant was $ 68.4799 (which equals the purchase price of a PIPE Share, less $ 0.0001 , the exercise price of each Warrant Share). Each pre-funded warrant has an exercise price equal to $ 0.0001 per underlying share of common stock. The pre-funded warrants are exercisable at any time after their original issuance and may be exercised at any time until exercised in full. The Company may not effect the exercise of any pre-funded warrants and the selling stockholder that holds pre-funded warrants (together with its affiliates and other attribution parties) may not exercise any portion of a pre-funded warrant to the extent that, immediately after giving effect to such exercise, the holder would own more than 9.99 % of the Company’s outstanding common stock, which percentage may be increased or decreased at the holder’s option (not to exceed 19.99 %) upon 61 days’ notice to the Company, subject to the terms of the pre-funded warrants. As of March 31, 2026, none of the Warrant Shares were exercised. On August 12, 2025, the Company filed an automatic shelf registration statement on Form S-3 with the SEC, which became effective upon filing, pursuant to which the Company registered for sale from time to time in one or more offerings an unlimited amount of any combination of the Company’s common stock, preferred stock, debt securities and warrants, so long as the Company continues to satisfy the requirements of a “well-known seasoned issuer” under SEC 20 Table of Contents rules. This automatic shelf registration statement will remain in effect for up to three years from the date it became effective. As of March 31, 2026, the Company had not issued any securities pursuant to the automatic shelf registration statement. On November 2, 2023, the Company entered into a Sales Agreement (the “2023 Sales Agreement”) with Leerink and Cantor Fitzgerald & Co. (the “Sales Agents”), pursuant to which the Company may, from time to time, sell up to an aggregate amount of $ 200.0 million of its common stock through the Sales Agents in an “at-the-market” offering. The Company is not required to sell shares under the 2023 Sales Agreement. Sales of the Company’s common stock, if any, under the 2023 Sales Agreement may be made in any transactions that are deemed to be “at the market offerings” as defined in Rule 415 under the Securities Act. The Company will pay a given designated Sales Agent a commission of up to 3.0 % of the aggregate gross proceeds of any shares of common stock sold through such Sales Agent pursuant to the 2023 Sales Agreement. As of March 31, 2026, the Company had not issued any securities pursuant to the 2023 Sales Agreement. Common Stock Reserved for Issuance Common stock reserved for issuance is as follows: As of March 31, 2026 Stock options, restricted stock units and performance stock units issued and outstanding 11,659,300 Reserved for future stock awards or option grants 4,261,036 Reserved for employee stock purchase plan 2,277,982 Common stock issuable upon conversion of convertible notes 9,963,490 Common stock issuable upon exercise of Warrant Shares 536,412 Common stock held back in connection with asset acquisition 522,375 29,220,595 11. Stock-Based Compensation Stock Options The following table summarizes stock option activity during the three months ended March 31, 2026 (in thousands, except share and per share data): Number of Awards Weighted- Average Exercise Price Weighted- Average Remaining Contractual Life (in Years) Aggregate Intrinsic Value Outstanding as of December 31, 2025 8,958,491 $ 25.41 6.5 $ 479,984 Granted 826,063 $ 100.67 Exercised ( 419,111 ) $ 24.17 Canceled and forfeited ( 26,053 ) $ 46.18 Outstanding as of March 31, 2026 9,339,390 $ 32.07 6.6 $ 570,172 Vested and exercisable as of March 31, 2026 5,960,516 $ 19.43 5.5 $ 434,830 The weighted-average grant date fair value per share of stock options granted during the three months ended March 31, 2026 and 2025 was $ 58.76 and $ 31.97 per share, respectively. The total intrinsic value of options exercised during the three months ended March 31, 2026 and 2025 was $ 27.8 million and $ 20.0 million, respectively. Intrinsic value is calculated as the difference between the exercise price of the underlying options and the fair value of the common stock for the options that had exercise prices that were lower than the per share fair value of the common stock on the date of exercise. As of March 31, 2026, the total unrecognized stock-based compensation related to unvested stock option awards granted was $ 108.4 million, which the Company expects to recognize over a weighted-average period of approximately 3.0 years. The fair value of each employee and non-employee stock option grant is estimated on the date of grant using the Black-Scholes option-pricing model. Due to the Company’s limited operating history and a lack of company specific 21 Table of Contents historical and implied volatility data, the expected stock price volatility was based upon the weighting of the Company’s historical volatility and the historical volatility of a peer group of publicly traded companies. The historical volatility data was computed using the daily closing prices for the Company’s and its peer companies’ shares during the equivalent period of the calculated expected term of the stock-based awards. Due to the lack of historical exercise history, the expected term of the Company’s stock options for employees has been determined utilizing the “simplified” method for awards. The risk-free interest rate is determined by reference to the U.S. Treasury yield curve in effect at the time of grant of the award for time periods approximately equal to the expected term of the award. Expected dividend yield is zero based on the fact that the Company has never paid cash dividends and does not expect to pay any cash dividends in the foreseeable future. The following assumptions were used to estimate the fair value of stock option awards granted during the following periods: Three Months Ended March 31, 2026 2025 Expected term (in years) 5.50 - 6.08 6.08 Expected volatility 53.29 %- 67.69 % 70.70 %- 71.31 % Risk-free interest rate 3.68 %- 4.13 % 4.05 %- 4.65 % Expected dividend yield — — Restricted Stock Units The following table summarizes the activity under the Company’s restricted stock units for the three months ended March 31, 2026: Number of Awards Weighted-Average Grant Date Fair Value per Award Unvested and outstanding as of December 31, 2025 1,415,919 $ 39.08 Granted 884,742 $ 100.64 Vested ( 454,173 ) $ 33.53 Cancelled/Forfeited ( 14,076 ) $ 53.74 Unvested and outstanding as of March 31, 2026 1,832,412 $ 70.07 The fair value of restricted stock unit awards granted to employees and nonemployees is equal to the closing market price of the Company’s common stock on the grant date. As of March 31, 2026, the total unrecognized stock-based compensation related to restricted stock unit awards granted was $ 113.7 million, which the Company expects to recognize over a weighted-average period of approximately 2.6 years. Performance Stock Units The fair value of performance stock units (“PSUs”) granted to employees is equal to the closing market price of the Company’s common stock on the grant date. PSUs are subject to vest only if certain specified sales-based criteria are achieved and the employees’ continued service with the Company. As of March 31, 2026, certain specified sales-based criteria were deemed probable of achievement or already achieved. Stock-based compensation for PSUs is recognized over the service period beginning in the period the Company determines it is probable that the performance criteria will be 22 Table of Contents achieved. PSUs generally vest over a three-year service period. The number of shares earned is adjusted based on the specified sales-based criteria achievement. The following table summarizes the activity under the Company’s performance stock units for the three months ended March 31, 2026: Number of Awards Weighted-Average Grant Date Fair Value per Award Unvested and outstanding as of December 31, 2025 353,743 $ 34.91 Granted 388,034 $ 73.31 Vested ( 254,279 ) $ 25.75 Cancelled/Forfeited — $ — Unvested and outstanding as of March 31, 2026 487,498 $ 70.25 As of March 31, 2026, the total unrecognized stock-based compensation related to performance stock units granted was $ 7.1 million, which the Company expects to recognize over a weighted-average period of approximately 1.3 years. The unrecognized stock-based compensation related to performance stock units granted during the three months ended March 31, 2026 excludes compensation related to awards for which the specified sales-based criteria was not deemed probable of achievement as of March 31, 2026. Compensation Expense Total stock-based compensation is reflected in the accompanying unaudited condensed consolidated statements of operations as follows (in thousands): Three Months Ended March 31, 2026 2025 Selling, general and administrative $ 32,937 $ 10,243 Research and development 24,189 5,229 Cost of sales 348 331 Total $ 57,474 $ 15,803 Stock-based compensation for the three months ended March 31, 2026 includes $ 19.3 million and $ 15.4 million in selling, general and administrative and research and development, respectively, related to unvested Bluejay employee stock awards which were accelerated in contemplation of the Bluejay Acquisition (refer to Note 6). This amount was recognized as stock-based compensation expense of the post-combination entity immediately after closing of the Bluejay Acquisition, as the related consideration paid and payable is not subject to any future service period. 12. Segment Reporting The Company’s chief operating decision maker (“CODM”), the Chief Executive Officer, manages the Company’s operations and business as one operating segment and allocates resources to operations of the Company on an entity-wide basis. The CODM assesses performance of the Company and determines resource allocation primarily based on net product sales and income (loss) from operations on a consolidated basis. The CODM uses income (loss) from operations to monitor budget versus actual results and considers any adjustments and actions required for good fiscal management. 23 Table of Contents The Company’s CODM is regularly provided with entity-wide expense categories similar to those found in the consolidated statements of operations, as well as the following (in thousands): Three Months Ended March 31, 2026 2025 Revenue: Product sales, net $ 159,882 $ 111,585 Less: Cost of sales (excluding intangible amortization) 22,120 17,124 General and administrative expenses (excluding stock-based compensation) 19,175 11,681 Commercialization and Medical Affairs expenses (excluding stock-based compensation) 44,218 35,782 Research and development expenses (excluding stock-based compensation) 73,721 35,815 Acquired IPR&D 726,302 5,000 Stock-based compensation 57,126 15,472 Intangible amortization 6,685 5,894 Loss from operations $ ( 789,465 ) $ ( 15,183 ) 13. Commitments and Contingencies Certain of the Company’s contractual arrangements with contract manufacturing organizations require binding forecasts or commitments to purchase minimum amounts for the manufacture of drug product supply, which may be material to the Company's unaudited condensed consolidated financial statements. The Company is subject to potential liabilities under government regulations and various claims and legal actions that are pending or may be asserted from time-to-time. These matters arise in the ordinary course and conduct of the Company’s business and may include, for example, commercial, intellectual property, and employment matters. The Company intends to defend itself vigorously in such matters and when warranted, take legal action against others. Furthermore, the Company regularly assesses contingencies to determine the degree of probability and range of possible loss for potential accrual in its unaudited condensed consolidated financial statements. An estimated loss contingency is accrued in the Company’s unaudited condensed consolidated financial statements if it is probable that a liability has been incurred and the amount of the loss can be reasonably estimated. The Company does not accrue amounts for liabilities that it does not believe are probable. Litigation is inherently unpredictable, and unfavorable resolutions could occur. As a result, assessing contingencies is highly subjective and requires judgment about future events. During the periods presented, the Company has not recorded any accrual for loss contingencies associated with government regulations, claims or legal actions, determined that an unfavorable outcome is probable or reasonably possible, or determined that the amount or range of any possible loss is reasonably estimable. On December 19, 2025, the Company, along with Satiogen Pharmaceuticals, Inc. and Shire Human Genetic Therapies, Inc. as co-plaintiffs, filed four complaints against Sandoz Inc. (“Sandoz”); Annora Pharma Private Limited, Hetero Labs Limited, and Hetero USA Inc. (together, “Hetero”); Zenara Pharma Private Limited and Biophore India Pharmaceuticals Private Limited (together, “Biophore”); and Zydus Lifesciences Global FZE, Zydus Lifesciences Limited, and Zydus Pharmaceuticals (USA) Inc. (together, “Zydus”) (all collectively, “Defendants”) in the U.S. District Court for the District of Delaware (the “LIVMARLI Patent Litigations”), alleging infringement of certain Orange Book listed patents covering LIVMARLI (the “LIVMARLI Patents”). The LIVMARLI Patent Litigations were initiated following the submission by Defendants, in accordance with the procedures set out in the Hatch-Waxman Act, of ANDAs directed to generic versions of LIVMARLI. Defendants’ ANDAs seek approval to market generic versions of LIVMARLI prior to the expiration of the LIVMARLI Patents and allege that the LIVMARLI Patents are invalid, unenforceable, and/or not infringed. The Company is seeking, among other relief, an order that the effective date of any FDA approval of Defendants’ ANDAs be no earlier than the expiration of the asserted patents listed in the Orange Book, and such further and other relief as the court may deem appropriate. The Defendants are subject to a 30-month stay of final regulatory approval through March 29, 2029, preventing them from marketing generic versions of LIVMARLI during that time. On February 20, 2026, Sandoz asserted counterclaims against the Company and its co-plaintiffs seeking declaratory judgments of non-infringement and invalidity with respect to certain LIVMARLI Patents. On March 30, 2026, Biophore asserted counterclaims against the Company and its co-plaintiffs seeking declaratory judgments of non-infringement and invalidity with respect to certain LIVMARLI Patents. The Company and its co-plaintiffs have denied all asserted declaratory judgment claims. On April 16, 2026, the Court ordered that the four pending cases against Sandoz, Hetero, Biophore and 24 Table of Contents Zydus be consolidated for pretrial purposes. Trial in the LIVMARLI Patent Litigations has not yet been scheduled. The Company cannot make any predictions about the final outcome of these matters or the timing thereof. 14. Subsequent Event In April 2026, the Company entered into an agreement with Incyte Corporation (“Incyte”) whereby the Company obtained the right to commercialize zilurgisertib upon FDA approval. Zilurgisertib is an oral small molecule ALK2 inhibitor in development for the treatment of fibrodysplasia ossificans progressiva. The Company made an upfront payment of $ 16.0 million in April 2026. In addition, the Company is obligated to pay royalties in the mid-to-high single digit percentage range and additional regulatory milestones of up to $ 48.0 million and commercial milestones of up to $ 15.0 million. 25 Table of Contents