Mirum Pharmaceuticals, Inc.

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 hasfour approved medicines: LIVMARLI® (maralixibat) (“Livmarli”), Cholbam® (cholic acid) capsules (“Cholbam”), CTEXLI® (chenodiol) tablets (“Ctexli”), and ATEBRIOZ® (zilurgisertib) (“Atebrioz”).

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. and certain other countries 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”.

In September 2026, the FDA approved zilurgisertib, an oral small molecule ALK2 inhibitor, to reduce the volume of total new heterotopic ossification in adult and pediatric patients aged 12 years and older with fibrodysplasia ossificans progressiva (“FOP”). Zilurgisertib is commercialized in the U.S. under the brand name Atebrioz.

The Company’s development pipeline consists of the clinical-stage product candidates volixibat, brelovitug and MRM-3379, and indication expansion opportunities for Livmarli and Atebrioz.

The Company commenced significant operations in November 2018. The Company views its operations and manages its business asone 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 September 30, 2026, the Company had an accumulated deficit of $1.6 billion and unrestricted cash, cash equivalents and investments of $500.5 million. The Company’s 4.00% convertible senior subordinated notes due 2029 (the “2029 Notes”) are convertible at the option of the holders during the fourth quarter of 2026. If holders of the 2029 Notes elect to convert, the Company may elect to settle such conversions in cash, common stock or a combination of the two. The Company’s 0.00% convertible senior subordinated notes due 2032 (the “2032 Notes”) are not convertible at the option of the holders during the fourth quarter of 2026. The Company believes that its unrestricted cash, cash equivalents and investments of $500.5 million as of September 30, 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.

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 and nine months ended September 30, 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 nine months ended September 30, 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 nine months ended September 30, 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):

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 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), net. To date, the Company hasnot 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 September 30, 2026 and December 31, 2025, the Company didnot have any customer who individually accounted for 10% or more of accounts receivable. For the three and nine months ended September 30, 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 wasno allowance for credit losses as of September 30, 2026. Bad debt expense wasnot material for the nine months ended September 30, 2026. No bad debt expense was recognized for the three months ended September 30, 2026, and three and nine months ended September 30, 2025.

Convertible Notes

The Company evaluates all conversion, repurchase and redemption features contained in a debt instrument to determine if there are any embedded features that require bifurcation as a derivative. The Company accounts for its convertible notes (refer to Note 9) as a long-term liability equal to the proceeds received from issuance, including any embedded conversion features, net of the unamortized debt discount and offering costs in the accompanying consolidated balance sheets. The debt issuance and offering costs are amortized over the contractual term of the convertible notes, using the effective interest method, as interest expense in the accompanying condensed consolidated statements of operations.

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:

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 claim 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 Pharmaceutical Company Limited (“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 September 30, 2026 and December 31, 2025, the Company did not have any contract assets and recorded a liability of $5.9 million and $3.6 million, respectively, 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):

The following table sets forth Product sales, net by geographic area based on the ship-to location (in thousands):

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 $0.2 million gain and $1.8 million loss for the three and nine months ended September 30, 2026, respectively, and a gain of $0.6 million and $1.9 million for the three and nine months ended September 30, 2025, respectively. Realized foreign exchange gains and losses amounted to a $0.1 million loss and $0.8 million gain for the three and nine months ended September 30, 2026, respectively, and a loss of $0.2 million and $1.0 million for the three and nine months ended September 30, 2025, respectively.

Net (Loss) Income Per Share

Basic net (loss) income 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) income per share for the three and nine months ended September 30, 2026 as they are issuable for immaterial cash consideration. Diluted net (loss) income 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) income 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) income per share for the three and nine months ended September 30, 2026 and 2025 (in thousands, except share and per share amounts):

The following outstanding potential dilutive shares have been excluded from the calculation of diluted net (loss) income per share for each period presented due to their anti-dilutive effect:

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. 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 statements 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 statements of operations unless there is an alternative future use. Regulatory approval and commercial milestones are recorded as intangible assets when they are paid or payable.

Recently Adopted Accounting Pronouncements

In November 2024, the Financial Accounting Standards Board (“FASB”) issued ASU 2024-04, Debt—Debt with Conversion and Other Options (Subtopic 470-20): Induced Conversions of Convertible Debt Instruments (“ASU 2024-04”) which clarifies the requirements for determining whether certain settlements of convertible debt instruments should be accounted for as induced conversions. Transactions within the scope of ASU 2024-04 require the fair value of consideration transferred in excess of the consideration issuable under the original conversion terms of the converted debt to be recognized as an expense in the period of conversion, with the remaining consideration recognized as a reduction to equity. The guidance is effective for annual reporting periods beginning after December 15, 2025 and interim reporting periods within those annual reporting periods. The Company adopted this guidance as of January 1, 2026, and the impact of the adoption has been reflected in the financial statements and related disclosures.

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.

In September 2025, the FASB issued ASU 2025-07, Derivatives and Hedging (Topic 815) and Revenue from Contracts with Customers (Topic 606): Derivatives Scope Refinements and Scope Clarification for Share-Based Noncash Consideration from a Customer in a Revenue Contract (“ASU 2025-07”). ASU 2025-07 expands an existing scope exception under Topic 815 to exclude non-exchange-traded contracts where the underlying is based on the operations or activities specific to one of the parties to the contract. The Company early adopted the standard as of January 1, 2026 on a prospective basis. 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 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”), 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):

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 September 30, 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.

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(1). 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):

(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 sheet as of September 30, 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):

As of September 30, 2026, the remaining contractual maturities of available-for-sale debt securities were as follows (in thousands):

During the three and nine months ended September 30, 2026 and 2025, there have beenno material 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 didnot recognize any credit losses on these securities. The unrealized losses on these securities were primarily attributable to the current interest rate environment rather than deterioration in the credit quality of the issuers. The Company has the intent and ability to hold such securities until maturity.

5. Balance Sheet Components

Inventory

Inventory consists of the following (in thousands):

Intangible Assets, Net

The components of the Company’s intangible assets were as follows (in thousands, except for weighted-average remaining amortization period):

Amortization expense was $6.8 million and $20.3 million for the three and nine months ended September 30, 2026, respectively, and $6.0 million and $17.9 million for the three and nine months ended September 30, 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 September 30, 2026 (in thousands):

Accrued Expenses

Accrued expenses consist of the following (in thousands):

Other liabilities

Other liabilities consist of the following (in thousands):

6. Asset Acquisitions

License Agreement with third party

In September 2026, the Company entered into a license agreement under which the Company obtained the right to develop and commercialize a clinical-stage small molecule compound outside of China. The Company made an upfront payment of $13.5 million. In addition, the Company is obligated to pay development and regulatory milestones of up to $32.5 million, commercial milestones of up to $525.0 million and royalties ranging from mid-single digit to low-double digit percentage range.

The Company accounted for the transaction as an asset acquisition as the set of acquired assets did not constitute a business and substantially all the fair value of the gross assets acquired was concentrated in a group of similar identifiable assets. The Company determined that the intellectual property assets acquired did not have an alternative future use and were not deemed to be commercially viable. The Company recorded a $13.5 million charge, representing the acquired IPR&D asset to acquired in-process research and development expense in the unaudited condensed consolidated statements of operations for the three and nine months ended September 30, 2026.

License Agreement with Incyte Corporation

In April 2026, the Company entered into a license agreement with Incyte Corporation, under which the Company obtained the right to develop and commercialize zilurgisertib worldwide. Zilurgisertib is an oral small molecule ALK2 inhibitor in development for the treatment of FOP. 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, including $25.0 million upon FDA approval, and commercial milestones of up to $15.0 million.

The Company accounted for the transaction as an asset acquisition as the set of acquired assets did not constitute a business and substantially all the fair value of the gross assets acquired was concentrated in a group of similar identifiable assets, namely, zilurgisertib. The Company determined that the intellectual property assets acquired did not have an alternative future use and were not deemed to be commercially viable. The Company recorded a $16.4 million charge, representing the acquired IPR&D asset and the related transaction costs to acquired in-process research and development expense in the unaudited condensed consolidated statements of operations for the nine months ended September 30, 2026.

As of September 30, 2026, the Company accrued $25.0 million for the achievement of a regulatory milestone associated with FDA approval of Atebrioz, which was recognized as an intangible asset in the accompanying unaudited condensed consolidated balance sheet as of September 30, 2026 and paid in October 2026.

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 transferable 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 nine months ended September 30, 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):

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):

Allocation of the consideration transferred to the net assets acquired was as follows (in thousands):

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 September 30, 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 September 30, 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. Through September 30, 2026, the Company paid or accrued $35.0 million 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.

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. Under the terms of the Shire Agreement, Shire granted the Company an exclusive, royalty bearing worldwide license to develop and commercialize itstwo 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 September 30, 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 andten years after the first commercial sale of a licensed product in such country. During the nine months ended September 30, 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 nine months ended September 30, 2025, the Company achieved a $5.0 million development milestone related to maralixibat, which was recognized in acquired in-process 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 wereno volixibat development and regulatory milestones achieved during the three and nine months ended September 30, 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 andten 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 September 30, 2026, no milestones have been achieved.

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 nine months ended September 30, 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:

Rent expense was $0.8 million and $2.4 million for the three and nine months ended September 30, 2026, respectively, and $0.6 million and $1.8 million for the three and nine months ended September 30, 2025, respectively. Variable lease payments for operating expenses for the three and nine months ended September 30, 2026 and September 30, 2025 werenot material.

9. Convertible Notes

As of September 30, 2026, the Company had outstanding fixed-rate convertible notes with varying maturities for an aggregate principal amount of $749.0 million (collectively the “Notes”). The Notes are senior subordinated convertible obligations, and interest, where applicable, is payable in arrears, semi-annually. Except as described below, the 2029 Notes are described in Note 10 of the “Notes to Consolidated Financial Statements” in the Annual Report.

The Notes consisted of the following (in thousands):

(1)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.

For the fourth quarter of 2024, for each of the quarters of 2025 and for the first, second and third quarters of 2026, the last reported sale price of the Company’s common stock exceeded 130% of the conversion price of the 2029 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 periods ended March 31, 2026, June 30, 2026 and September 30, 2026, the 2029 Notes were, and for the quarterly period ending December 31, 2026, the 2029 Notes are, convertible at the option of the holders of the 2029 Notes.

The Company amortizes issuance costs related to the issuance of the Notes to interest expense over the term of the Notes using the effective interest method. As of September 30, 2026, the remaining amortization period of the debt issuance costs was approximately 2.6 years and 5.7 years, respectively for the 2029 Notes and the 2032 Notes, and the effective interest rates were 4.6% and 0.4%, respectively for the 2029 Notes and the 2032 Notes.

The following table sets forth interest expense recognized related to the Notes (in thousands):

2032 Notes

In May 2026, the Company issued $690.0 million aggregate principal amount of its 2032 Notes in a private placement to qualified institutional buyers pursuant to Rule 144A under the Securities Act of 1933, as amended. The 2032 Notes were issued pursuant to an Indenture (the “2032 Notes Indenture”), dated May 15, 2026, between the Company and U.S. Bank Trust Company, National Association, as trustee.

The Company received net proceeds of approximately $672.0 million after deducting initial purchasers’ discounts and commissions and offering expenses.

The 2032 Notes are the Company’s senior, unsecured obligations and are (i) senior in right of payment to any of the Company’s future indebtedness that is expressly subordinated to the 2032 Notes in right of payment; (ii) equal in right of payment to any of the Company’s indebtedness that is not so subordinated, including the 2029 Notes; (iii) effectively subordinated to any of the Company’s future secured indebtedness, to the extent of the value of the collateral securing such indebtedness; and (iv) structurally subordinated to all indebtedness and other liabilities, including trade payables, and, to the extent the Company is not a holder thereof, preferred equity, if any, of the Company’s subsidiaries.

The 2032 Notes do not bear interest and will mature on June 1, 2032, unless earlier converted by the noteholders, redeemed by the Company, or repurchased by the Company. Noteholders may convert all or a portion of their 2032 Notes only under the following circumstances:

(i)during any calendar quarter, and only during such calendar quarter, commencing after the calendar quarter ending on September 30, 2026, if the last reported sale price per share of the Company’s common stock exceeds 130% of the conversion price for each of at least 20 trading days, whether or not consecutive, during the 30 consecutive trading days ending on, and including, the last trading day of the immediately preceding calendar quarter;

(ii)during thefive consecutive business days immediately after any 10 consecutive trading day period if the trading price per $1,000 principal amount of 2032 Notes for each trading day of such period was less than 98% of the product of the last reported sale price per share of the Company’s common stock on such trading day and the conversion rate on such trading day;

(iii)upon the occurrence of certain corporate events or distributions on the Company’s common stock, as described in the 2032 Notes Indenture;

(iv)if the Company calls such 2032 Notes for redemption; and

(v)at any time from March 1, 2032 until the close of business on the second scheduled trading day immediately before the maturity date.

The Company may settle conversions of the 2032 Notes by paying or delivering, as applicable, cash, shares of the Company’s common stock or a combination of cash and shares of the Company’s common stock, at the Company’s election. The initial conversion rate is 7.1971 shares of common stock per $1,000 principal amount of 2032 Notes, which represents an initial conversion price of approximately $138.94 per share of common stock. The conversion rate and conversion price are subject to customary adjustments upon the occurrence of certain events. In addition, if certain corporate events that constitute a “Make-Whole Fundamental Change” (as defined in the 2032 Notes Indenture) occur, or if the Company calls any 2032 Notes for redemption, then the conversion rate will, in certain circumstances, be increased for a specified period of time. In no event will the conversion rate be increased to an amount that exceeds 9.3562 shares of common stock per $1,000 principal amount of 2032 Notes, subject to adjustment as provided in the 2032 Notes.

The Company may not redeem the 2032 Notes at its option at any time before June 6, 2029. The 2032 Notes are redeemable, in whole or in part, subject to the partial redemption limitation described below, at the Company’s option at any time, and from time to time, on or after June 6, 2029 and, in the case of any partial redemption, on or before the 30th scheduled trading day immediately before the maturity date, at a cash redemption price equal to the principal amount of the 2032 Notes to be redeemed, plus accrued and unpaid interest, if any, to, but excluding, the redemption date, but only if (i) the 2032 Notes are freely tradable as of the date the Company sends the related redemption notice and all accrued and unpaid additional interest, if any, has been paid in full as of the first interest payment date occurring on or before the date the Company sends such notice; and (ii) the last reported sale price per share of the Company’s common stock exceeds 130% of the conversion price on each of at least 20 trading days, whether or not consecutive, during the 30 consecutive trading days ending on, and including, the trading day immediately before the date the Company sends the related redemption notice and on the trading day immediately before the date the Company sends such notice. In addition, calling any 2032 Notes for redemption will constitute a Make-Whole Fundamental Change with respect to that 2032 Notes, in which case the conversion rate applicable to the conversion of that 2032 Notes will be increased in certain circumstances if it is converted after it is called for redemption. The Company may not elect to redeem less than all of the outstanding 2032 Notes unless at least $75.0 million aggregate principal amount of 2032 Notes are outstanding and not called for redemption as of the time the Company sends the related redemption notice.

If a “Fundamental Change”, as defined in the 2032 Notes, occurs, then, subject to a limited exception for certain cash mergers, noteholders may require the Company to repurchase their 2032 Notes at a cash repurchase price equal to the principal amount of the 2032 Notes to be repurchased, plus accrued and unpaid interest, if any, to, but excluding, the fundamental change repurchase date. The definition of Fundamental Change includes certain business combination transactions involving the Company and certain de-listing events with respect to the Company’s common stock.

The 2032 Notes Indenture contains customary events of default with respect to the 2032 Notes and provides that upon certain events of default occurring and continuing, the trustee may, and the trustee at the request of holders of at least

25% in principal amount of the 2032 Notes shall, declare all principal and accrued and unpaid interest, if any, of the 2032 Notes to be due and payable. In case of certain events of bankruptcy, insolvency or reorganization involving the Company or a significant subsidiary, all of the principal of and accrued and unpaid interest, if any, on the 2032 Notes will automatically become due and payable.

2029 Notes

Induced conversions of the 2029 Notes

In May 2026, the Company entered into privately negotiated exchange and repurchase transactions with certain noteholders of its 2029 Notes. Pursuant to these transactions, the Company paid $474.7 million in cash, including accrued and unpaid interest of $0.3 million, and issued 3,220,529 shares of the Company’s common stock, with such common stock having an aggregate fair value of $329.1 million, in exchange for $237.2 million aggregate principal amount of the 2029 Notes. The transactions were completed concurrently with the issuance of the Company’s 2032 Notes. The carrying amount of the 2029 Notes subject to the transactions, including $4.3 million of unamortized debt issuance costs, was transferred to equity with no gain or loss recognized. The exchange and repurchase transactions resulted in a decrease in additional paid-in capital of $558.9 million.

In September 2026, the Company entered into privately negotiated repurchase transactions with certain noteholders of its 2029 Notes. Pursuant to these transactions, the Company paid $62.0 million in cash, including accrued and unpaid interest of $0.3 million, in exchange for $20.0 million aggregate principal amount of the 2029 Notes. The carrying amount of the 2029 Notes subject to the transactions, including $0.3 million of unamortized debt issuance costs, was transferred to equity with no gain or loss recognized. The repurchase transaction resulted in a decrease in additional paid-in capital of $42.0 million.

As part of each of the May and September 2026 transactions, the Company extended limited-time inducement offers that provided noteholders with consideration, the fair value of which exceeded the value that would have been received under the original conversion terms of the 2029 Notes, resulting in incremental value to the noteholders. The fair value of the total consideration transferred in the May transactions was determined based on the quoted market price of the Company’s common stock on May 12, 2026, the date the May inducement offers were accepted.

The transactions were accounted for as induced conversions. The Company recognized debt conversion inducement expense of $0.1 million and $11.7 million during the three and nine months ended September 30, 2026, respectively, measured as the fair value of the incremental consideration transferred to noteholders in excess of the consideration issuable under the original conversion terms of the 2029 Notes. Debt conversion inducement expense is included in other income (expense), net in the Company’s unaudited condensed consolidated statements of operations.

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 September 30, 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 rules. This automatic shelf registration statement will remain in effect for up to three years from the date it became effective. As of September 30, 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 September 30, 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:

11. Stock-Based Compensation

Stock Options

The following table summarizes stock option activity during the nine months ended September 30, 2026 (in thousands, except share and per share data):

The weighted-average grant date fair value per share of stock options granted during the nine months ended September 30, 2026 and 2025 was $58.34 and $31.86 per share, respectively. The total intrinsic value of options exercised during the nine months ended September 30, 2026 and 2025 was $97.5 million and $94.1 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 September 30, 2026, the total unrecognized stock-based compensation related to unvested stock option awards granted was $89.9 million, which the Company expects to recognize over a weighted-average period of approximately 2.7 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 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 iszero 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:

Restricted Stock Units

The following table summarizes the activity under the Company’s restricted stock units for the nine months ended September 30, 2026:

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 September 30, 2026, the total unrecognized stock-based compensation related to restricted stock unit awards granted was $99.8 million, which the Company expects to recognize over a weighted-average period of approximately 2.2 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 vest only if certain specified sales-based criteria are achieved and the employees continue service with the Company. As of September 30, 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 achieved. PSUs generally vest over athree-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 nine months ended September 30, 2026:

As of September 30, 2026, the total unrecognized stock-based compensation related to performance stock units granted was $29.6 million, which the Company expects to recognize over a weighted-average period of approximately 1.8 years.

Compensation Expense

Total stock-based compensation is reflected in the accompanying unaudited condensed consolidated statements of operations as follows (in thousands):

Stock-based compensation for the nine months ended September 30, 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 asone 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.

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):

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.

Legal Proceedings

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 (as used in this paragraph, 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 oral solutions (the “LIVMARLI Patents”). The LIVMARLI Patent Litigations were initiated, in accordance with the procedures set out in the Hatch-Waxman Amendments of the Federal Food, Drug, and Cosmetic Act (the “Hatch-Waxman Act”), following the submission by Defendants of abbreviated new drug applications (“ANDAs”) directed to generic versions of LIVMARLI oral solutions. Defendants’ ANDAs seek approval to market generic versions of LIVMARLI oral solutions prior to the expiration of one or more of the LIVMARLI Patents and allege that one or more of 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. Based on the Company initiating the litigation, 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 oral solutions 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. On April 22, 2026, the Company had listed newly issued U.S. Patent No. 12,599,602 (the “’602 patent”) in the Orange Book, and on May 18, 2026, the Company, together with its co-plaintiffs filed amended complaints against Sandoz, Hetero, Biophore and Zydus asserting the ’602 patent. On June 1, 2026, Biophore asserted a counterclaim seeking declaratory judgment of non-infringement and invalidity with respect to the ’602 patent. On June 17, 2026, Sandoz asserted a counterclaim seeking declaratory judgment of non-infringement and invalidity with respect to the ’602 patent. The Company and its co-plaintiffs have denied all asserted declaratory judgment claims. On April 16, 2026, the Court ordered that thefour pending cases against Sandoz, Hetero, Biophore and Zydus be consolidated for pretrial purposes. The Court issued a scheduling order on May 19, 2026. Discovery is ongoing.

On September 14, 2026, the Company, along with Satiogen Pharmaceuticals, Inc. and Shire Human Genetic Therapies, Inc. as co-plaintiffs, filed a complaint against Zenara Pharma Private Limited and Biophore India Pharmaceuticals Private Limited (as used in this paragraph, together, “Zenara”) in the U.S. District Court for the District of Delaware (the “LIVMARLI Tablet Litigation”), alleging infringement of certain Orange Book listed patents covering LIVMARLI tablets (the “LIVMARLI Tablets Patents”). The LIVMARLI Tablet Litigation was initiated, in accordance with the procedures set out in the Hatch-Waxman Act, following the submission by Zenara of an ANDA directed to generic versions of LIVMARLI tablets. Zenara’s ANDA seeks approval to market generic versions of LIVMARLI tablets prior to the expiration of one or more of the LIVMARLI Tablet Patents and alleges that one or more of the LIVMARLI Tablet 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 Zenara’s ANDA 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. Based on the Company initiating the litigation, Zenara is subject to a 30-month stay of final regulatory approval through March 29, 2029, preventing Zenara from marketing generic versions of LIVMARLI tablets during that time. On September 28, 2026, Zenara asserted counterclaims against the Company and its co-plaintiffs seeking declaratory judgments of non-infringement and invalidity with respect to the LIVMARLI Tablet Patents. The Company and its co-plaintiffs have denied all asserted declaratory judgment claims. On October 26, 2026, the parties filed a proposed scheduling order.

On October 30, 2026, the Court ordered that the LIVMARLI Patent Litigations and the LIVMARLI Tablet Litigation be consolidated for pretrial purposes (together, the “Consolidated LIVMARLI Litigations”). A Markman hearing is set in the Consolidated LIVMARLI Litigations for July 20, 2027. Trial in the Consolidated LIVMARLI Litigations is set for September 18, 2028.

The Company cannot make any predictions about the final outcome of these matters or the timing thereof.