31 unchanged sentences
The communication of the critical audit matter does not alter in any way our opinion on the consolidated financial statements, taken as a whole, and we are not, by communicating the critical audit matter below, providing a separate opinion on the critical audit matter or on the accounts or disclosures to which it relates.
+Added: Inventory Costing
Description of the Matter
−Removed: As described in Notes 2 and 5 to the consolidated financial statements, inventory, which totaled $22.4 million as of December 31, 2024, is comprised of raw materials, work-in-progress and finished goods for Livmarli, Chenodal and Cholbam (“the Products”).
+Added: As described in Notes 2 and 5 to the consolidated financial statements, inventory, which totaled $24.9 million as of December 31, 2025, is comprised of raw materials, work-in-progress and finished goods.
Inventory is valued at the lower of cost or net realizable value, with cost determined on a first-in, first-out basis.
−Removed: Auditing the inventory costing was complex because the nature of the process for inventory costing for the Products involves the use of a variety of inputs such as third-party material costs and allocated internal labor, all of which are tracked in manually-prepared spreadsheets.
−Removed: How We Addressed the Matter in Our Audit To test the completeness and accuracy of the inventory balance, our substantive audit procedures included, among others, agreeing a sample of production costs and purchases used in the spreadsheets to actual costs incurred, agreeing a sample of capitalized labor costs to payroll records, confirming certain quantities of on-hand inventory directly with third-party service providers, and confirming amounts owed to certain third-party service providers.
+Added: Auditing the inventory costing was complex because the nature of the process for such costing involves the use of a variety of inputs such as third-party material costs and allocated internal labor, all of which are tracked in manually-prepared spreadsheets.
+Added: How We Addressed the Matter in Our Audit We obtained an understanding, evaluated the design and tested the operating effectiveness of internal controls over the inventory process.
+Added: For example, we tested controls over management's review of inputs used in determining the costs to be capitalized into inventory.
+Added: To test the completeness and accuracy of the inventory balance, our audit procedures included, among others, agreeing a sample of production costs and purchases used in the spreadsheets to actual costs incurred, agreeing a sample of capitalized labor costs to payroll records, confirming certain quantities of on-hand inventory directly with third-party service providers, and confirming amounts owed to certain third-party service providers.
/s/ Ernst & Young LLP
48 unchanged sentences
Accounts payable $ 9,614 $ 14,618
−Removed: Accrued expenses 110,224 78,544
−Removed: Operating lease liabilities, current 1,709 1,104
+Added: Accrued expenses and other current liabilities 196,185 111,933
Total current liabilities 205,799 126,551
9 unchanged sentences
200,000,000 shares authorized;
−Removed: 48,338,096 and 46,723,143 shares issued and outstanding as of December 31, 2024 and 2023
+Added: 51,896,391 and 48,338,096 shares issued and outstanding as of December 31, 2025 and 2024, respectively
Additional paid-in capital 981,878 870,189
Accumulated deficit ( 667,544 ) ( 644,181 )
−Removed: Accumulated other comprehensive (loss) income ( 373 ) 1,644
+Added: Accumulated other comprehensive income (loss) 351 ( 373 )
Total stockholders’ equity 314,690 225,640
18 unchanged sentences
Interest expense ( 14,389 ) ( 14,311 ) ( 15,105 )
−Removed: Change in fair value of derivative liability — — 906
Loss from termination of revenue interest purchase agreement — — ( 49,076 )
1 unchanged sentence
Loss before provision for income taxes ( 21,427 ) ( 86,912 ) ( 162,424 )
−Removed: Provision for (benefit from) income taxes 1,030 991 ( 6,406 )
+Added: Provision for income taxes 1,936 1,030 991
Net loss $ ( 23,363 ) $ ( 87,942 ) $ ( 163,415 )
−Removed: Net loss per share:
−Removed: Basic $ ( 1.85 ) $ ( 4.00 ) $ ( 4.01 )
−Removed: Diluted $ ( 1.85 ) $ ( 4.00 ) $ ( 4.02 )
−Removed: Weighted-average shares of common stock outstanding
−Removed: Basic 47,522,594 40,885,124 33,839,072
−Removed: Diluted 47,522,594 40,885,124 33,982,493
+Added: Net loss per share, basic and diluted $ ( 0.47 ) $ ( 1.85 ) $ ( 4.00 )
+Added: Weighted-average shares of common stock, basic and diluted 50,198,304 47,522,594 40,885,124
The accompanying notes are an integral part of these consolidated financial statements.
6 unchanged sentences
Other comprehensive income (loss):
−Removed: Unrealized gain (loss) on available-for-sale investments 130 230 ( 194 )
+Added: Unrealized gain on available-for-sale investments 10 130 230
Cumulative translation adjustments 714 ( 2,147 ) 1,631
11 unchanged sentences
Balance as of December 31, 2022 36,956,345 $ 4 $ 535,074 $ ( 392,824 ) $ ( 217 ) $ 142,037
−Removed: Issuance of common stock in connection with asset acquisition 609,305 — 15,585 — — 15,585
−Removed: Issuance of common stock in connection with equity incentive plans 357,934 — 5,438 — — 5,438
−Removed: Issuance of common stock in at-the-market offerings, net of issuance costs of $ 785
−Removed: 1,160,915 — 21,289 — — 21,289
−Removed: Issuance of common stock in connection with public offering, net of issuance costs $ 5,922
−Removed: 4,000,000 1 86,077 — — 86,078
−Removed: Restricted common stock vested in the period 122,464 — — — — —
−Removed: Issuance of common stock in connection with Employee Stock Purchase Plan 123,131 — 1,834 — — 1,834
−Removed: Stock-based compensation — — 27,448 — — 27,448
−Removed: Net loss — — — ( 135,665 ) — ( 135,665 )
−Removed: Other comprehensive loss — — — — ( 182 ) ( 182 )
−Removed: Balance as of December 31, 2022 36,956,345 $ 4 $ 535,074 $ ( 392,824 ) $ ( 217 ) $ 142,037
Issuance of common stock in private placement, net of issuance costs of $ 7,796
15 unchanged sentences
Balance as of December 31, 2024 48,338,096 $ 5 $ 870,189 $ ( 644,181 ) $ ( 373 ) $ 225,640
+Added: Issuance of common stock in connection with equity award plans 3,450,566 — 36,200 — — 36,200
+Added: Issuance of common stock in connection with employee stock purchase plan 106,977 3,942 3,942
+Added: Conversion of convertible notes, net 752 — 24 — — 24
+Added: Stock-based compensation — — 71,523 — — 71,523
+Added: Net loss — — — ( 23,363 ) — ( 23,363 )
+Added: Other comprehensive income — — — — 724 724
+Added: Balance as of December 31, 2025 51,896,391 $ 5 $ 981,878 $ ( 667,544 ) $ 351 $ 314,690
The accompanying notes are an integral part of these consolidated financial statements.
6 unchanged sentences
Net loss $ ( 23,363 ) $ ( 87,942 ) $ ( 163,415 )
−Removed: Reconciliation of net loss to net cash provided by (used in) operating activities:
+Added: Reconciliation of net loss to net cash provided by operating activities:
Stock-based compensation 71,424 48,444 35,023
4 unchanged sentences
Non-cash interest expense related to the revenue interest liability — — 5,060
−Removed: Deferred income taxes associated with an acquisition — — ( 6,580 )
Unrealized foreign exchange (gain) loss ( 1,720 ) ( 1,231 ) 1,764
15 unchanged sentences
Payments made for additions to intangible assets — ( 20,000 ) ( 20,000 )
−Removed: Net cash (used in) provided by investing activities ( 90,125 ) ( 107,200 ) 7,700
+Added: Net cash used in investing activities ( 23,954 ) ( 90,125 ) ( 107,200 )
Financing activities
1 unchanged sentence
Proceeds from issuance of common stock in at-the-market offerings, net of issuance costs — — 14,480
−Removed: Proceeds from issuance of common stock in public offerings, net of issuance costs — — 86,078
Proceeds from issuance of convertible notes, net — — 305,304
4 unchanged sentences
Effect of exchange rate on cash, cash equivalents and restricted cash 3,222 ( 1,297 ) ( 133 )
−Removed: Net (decrease) increase in cash, cash equivalents and restricted cash ( 63,398 ) 158,323 ( 3,337 )
−Removed: Cash, cash equivalents and restricted cash equivalents at beginning of period 286,326 128,003 131,340
+Added: Net increase (decrease) in cash, cash equivalents and restricted cash 75,237 ( 63,398 ) 158,323
+Added: Cash, cash equivalents and restricted cash at beginning of period 222,928 286,326 128,003
Cash, cash equivalents and restricted cash at end of period $ 298,165 $ 222,928 $ 286,326
8 unchanged sentences
Stock-based compensation capitalized to inventory $ 688 $ 1,122 $ 822
+Added: Conversion of convertible notes, net into shares of common stock $ 24 $ — $ —
Issuance of common stock in connection with settlement of Contingent Milestone and Indemnification Holdback liabilities $ — $ — $ 5,188
−Removed: Contingent Milestone and Indemnification Holdback liabilities for common stock issuance in exchange for acquired intangible assets $ — $ — $ 5,431
−Removed: Issuance of common stock in exchange for acquired intangible $ — $ — $ 15,585
−Removed: Deferred tax liability incurred from acquired intangible $ — $ — $ 6,580
The accompanying notes are an integral part of these consolidated financial statements.
5 unchanged sentences
The Company is a biopharmaceutical company dedicated to transforming the treatment of rare diseases.
+Added: The Company commenced significant operations in November 2018.
As of December 31, 2025, the Company had three approved medicines:
−Removed: LIVMARLI® (maralixibat) oral solution (“Livmarli”), CHOLBAM® (cholic acid) capsules (“Cholbam”), and CHENODAL® (chenodiol) tablets (“Chenodal”).
−Removed: Livmarli is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (“ALGS”) in the United States (“U.S.”) and various other countries around the world and for cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (“PFIC”) in the U.S.
−Removed: and for the treatment of PFIC in the European Union (“EU”).
+Added: LIVMARLI® (maralixibat) (“Livmarli”), CHOLBAM® (cholic acid) capsules (“Cholbam”), and CTEXLI® (chenodiol) tablets (“Ctexli”).
+Added: 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.
On August 31, 2023, the Company completed the acquisition of assets of Travere Therapeutics, Inc.
1 unchanged sentence
Cholbam is 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.
−Removed: Chenodal at the time of acquisition was approved for the treatment of radiolucent stones in the gallbladder and had received medical necessity recognition by the FDA for the treatment of cerebrotendinous xanthomatosis (“CTX”).
−Removed: Subsequently, in February 2025, the Company received FDA approval for chenodiol tablets for the treatment of CTX in adults, which will be commercialized under the brand name Ctexli.
+Added: Ctexli at the time of acquisition was approved for the treatment of radiolucent stones in the gallbladder, had received medical necessity recognition by the FDA for the treatment of cerebrotendinous xanthomatosis (“CTX”) and was commercialized under the brand name Chenodal.
+Added: Subsequently, in February 2025, the Company received FDA approval for chenodiol tablets for the treatment of CTX in adults, which is commercialized under the brand name Ctexli.
The Company’s development pipeline consists of the clinical-stage product candidate volixibat, MRM-3379 and indication expansion opportunities for Livmarli.
−Removed: The Company commenced significant operations in November 2018.
+Added: On January 23, 2026, the Company acquired Bluejay Therapeutics, Inc.
+Added: With the completion of the acquisition, the Company adds worldwide rights to brelovitug, a late-stage, fully human monoclonal antibody for chronic hepatitis delta virus (HDV), a rare and severe liver disease.
The Company views its operations and manages its business as one operating segment.
4 unchanged sentences
The Company’s convertible notes are convertible at the option of the holders during the first quarter of 2026.
−Removed: 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 $ 292.8 million as of December 31, 2024 provide sufficient capital resources to continue its operations for at least twelve months from the issuance date of the accompanying consolidated financial statements.
+Added: 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.
+Added: The Company believes that its unrestricted cash, cash equivalents and investments of $ 391.4 million as of December 31, 2025 provide sufficient capital resources to continue its operations for at least twelve months from the issuance date of the accompanying consolidated financial statements.
Summary of Significant Accounting Policies
5 unchanged sentences
Use of Estimates
−Removed: The preparation of consolidated financial statements in accordance with GAAP requires management to make estimates and assumptions that impact the reported amounts of assets, liabilities and expenses and the disclosure of contingent assets and liabilities in the financial statements and accompanying notes.
−Removed: 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
−Removed: and the recording of expenses that are not readily apparent from other sources.
+Added: The preparation of consolidated financial statements in accordance with GAAP requires management to make estimates and assumptions that impact the reported amounts of assets, liabilities, revenues and expenses and the disclosure
+Added: of contingent assets and liabilities in the financial statements and accompanying notes.
+Added: 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 expenses that are not readily apparent from other sources.
Actual results could differ materially from those estimates.
−Removed: The Company’s consolidated financial statements as of and for the year ended December 31, 2024 reflect the Company’s estimates of the impact of the geopolitical and macroeconomic environment, including the impact of inflation, tariffs and trade tensions, bank failures, high interest rates and foreign exchange rate fluctuations.
+Added: The Company’s consolidated financial statements as of and for the year ended December 31, 2025 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;
1 unchanged sentence
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.
−Removed: Reclassifications
−Removed: Amounts related to the cost recognized on sale of acquired inventory in the statement of cash flows for the year ended December 31, 2023 have been reclassified to conform to the current presentation.
Cash, Cash Equivalents and Restricted Cash
20 unchanged sentences
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.
−Removed: 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
−Removed: for credit losses.
−Removed: 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.
+Added: 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.
+Added: Each reporting period, the Company evaluates whether declines in fair values of its available-for-sale
+Added: 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.
27 unchanged sentences
If actual market conditions are less favorable than projected by management, additional write-downs of inventory may be required.
−Removed: Accruals for firm purchase commitments amounted to $ 4.4 million and $ 5.2 million as of December 31, 2024 and December 31, 2023, respectively, of which $ 2.5 million and $ 3.8 million was included in other liabilities on the consolidated balance sheets as of December 31, 2024 and December 31, 2023, respectively.
Property and Equipment
3 unchanged sentences
As of December 31, 2025, property and equipment consisted primarily of leasehold improvements of $ 0.8 million and furniture and equipment of $ 2.1 million.
−Removed: As of December 31, 2023, property and equipment consisted primarily of leasehold improvements of $ 1.3 million
−Removed: and furniture and equipment of $ 0.9 million.
+Added: As of December 31, 2024, property and equipment consisted primarily of leasehold improvements of $ 0.2 million and furniture and equipment of $ 1.6 million.
Accumulated depreciation as of December 31, 2025 and 2024 was $ 1.1 million and $ 0.7 million, respectively.
−Removed: Depreciation expense was $ 0.5 million for the year ended December 31, 2024, and $ 0.3 million for each of the years ended December 31, 2023 and 2022.
+Added: Depreciation expense was $ 0.3 million, $ 0.5 million and $ 0.3 million for the years ended December 31, 2025, 2024 and 2023, respectively.
Impairment of Long-Lived Assets
12 unchanged sentences
The Company has not recorded any impairments to its intangible assets for any of the periods presented.
−Removed: The components of the Company’s intangible assets were as follows (in thousands, except for weighted-average remaining amortization period):
−Removed: December 31, 2024
−Removed: Gross Carrying Value Accumulated Amortization
−Removed: Net Carrying Amount Weighted-Average Remaining Amortization Period (Years)
−Removed: Commercial milestones $ 59,000 $ ( 7,092 ) $ 51,908 12.6
−Removed: Developed technology 226,620 ( 29,248 ) 197,372 10.7
−Removed: Assembled workforce 970 ( 431 ) 539 1.7
−Removed: Total intangible assets $ 286,590 $ ( 36,771 ) $ 249,819 11.1
−Removed: December 31, 2023
−Removed: Gross Carrying Value Accumulated Amortization
−Removed: Net Carrying Amount Weighted-Average Remaining Amortization Period (Years)
−Removed: Commercial milestones $ 39,000 $ ( 3,318 ) $ 35,682 16.2
−Removed: Developed technology 226,620 ( 10,239 ) 216,381 11.7
−Removed: Assembled workforce 970 ( 108 ) 862 2.7
−Removed: Total intangible assets $ 266,590 $ ( 13,665 ) $ 252,925 12.3
−Removed: Amortization expense was $ 23.1 million, $ 10.5 million and $ 2.9 million for the years ended December 31, 2024, 2023 and 2022, respectively, and was included in cost of sales on the accompanying consolidated statements of operations.
−Removed: The following table summarizes the estimated future amortization expense associated with the Company’s intangible assets as of December 31, 2024 (in thousands):
−Removed: 2025 $ 23,898
−Removed: Thereafter 131,405
The Company determines if a contractual arrangement is or contains a lease at inception.
17 unchanged sentences
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.
−Removed: The Company accounts for its convertible notes (refer to Note 10) as a 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.
+Added: The Company accounts for its
+Added: convertible notes (refer to Note 10) as a 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 consolidated statements of operations.
2 unchanged sentences
The effective interest rate was calculated based on the rate that would enable the debt to be repaid in full over the anticipated life of the arrangement.
−Removed: The interest rate on
−Removed: the liability varied during the term of the agreement depending on a number of factors, including the level of actual and forecasted product sales, net.
+Added: The interest rate on the liability varied during the term of the agreement depending on a number of factors, including the level of actual and forecasted product sales, net.
The Company evaluated the interest rate quarterly based on actual product sales, net and forecast product sales, net, utilizing the prospective method.
2 unchanged sentences
The RIPA contained certain features that met the definition of being an embedded derivative requiring bifurcation as a separate compound financial instrument apart from the RIPA.
−Removed: The derivative liability was initially measured at fair value on issuance and was subject to remeasurement at each reporting period with changes in fair value recognized as other income (expense) in the accompanying consolidated statements of operations as the change in fair value of derivative liability.
+Added: The derivative liability was initially measured at fair value on issuance and was subject to remeasurement at each reporting period.
In April 2023, the derivative liability was extinguished in connection with the termination of the RIPA (refer to Note 6).
4 unchanged sentences
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.
−Removed: Estimates of variable consideration are calculated based on the actual product sales each reporting period and the nature of the variable consideration related to those sales.
+Added: 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.
−Removed: Estimates are reviewed and updated quarterly as additional information becomes known.
+Added: Estimates are reviewed and updated as additional information becomes known.
Actual amounts of consideration ultimately received may differ materially from estimates.
11 unchanged sentences
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.
−Removed: The Company records amounts paid under the brand specific co-pay assistance program for each patient as a reduction of revenue from product sales.
+Added: The Company records amounts paid under the brand specific co-pay assistance program
+Added: for each patient as a reduction of revenue from product sales.
To date, actual other incentives have not differed materially from the Company’s estimates.
20 unchanged sentences
License and Collaboration Arrangements
−Removed: The Company enters into collaborative arrangements with partners and analyzes the collaboration arrangements to assess whether they are within the scope of Collaborative Arrangements (Topic 808) ("Topic 808") and determine whether such arrangements involve joint operating activities performed by parties that are both active participants in the activities and exposed to significant risks and rewards dependent on the commercial success of such activities.
+Added: The Company enters into collaborative arrangements with partners and analyzes the collaboration arrangements to assess whether they are within the scope of Collaborative Arrangements (Topic 808) (“Topic 808”) and determines whether such arrangements involve joint operating activities performed by parties that are both active participants in the activities and exposed to significant risks and rewards dependent on the commercial success of such activities.
This assessment is performed throughout the life of the arrangement based on changes in the responsibilities of all parties in the arrangement.
14 unchanged sentences
If it is probable that a significant reversal of cumulative revenue recognized for the contract would not occur, the associated milestone value is included in the transaction price.
−Removed: Milestone payments that are not within the control of the Company or the licensee, such as regulatory approvals, are not generally
−Removed: considered probable of being achieved until those approvals are received.
+Added: Milestone payments that are not within the control of the Company or the licensee, such as regulatory approvals, are not generally considered probable of being achieved until those approvals are received.
Given the high degree of uncertainty around the occurrence of these events, the Company generally determines the milestone and other contingent amounts to be fully constrained until the uncertainty associated with these payments is resolved.
6 unchanged sentences
Accordingly, the Company estimates a stand-alone selling price through maximizing the use of observable inputs such as market data, project cost estimates, and targeted margins.
−Removed: Cost of Product Sales
+Added: Cost of Sales
Prior to receiving approval from the U.S.
1 unchanged sentence
Subsequent to receiving FDA or other foreign regulatory authority approval, when commercialization is considered probable and the future economic benefit is expected to be realized, the Company begins capitalizing inventory costs incurred.
−Removed: Cost of product sales consist of manufacturing costs, transportation and freight, amortization of capitalized intangible assets, royalties and indirect overhead costs associated with the manufacturing and distribution of the Company's approved products.
−Removed: Cost of product sales may also include period costs related to certain manufacturing services and inventory adjustment charges.
+Added: Cost of sales consist of manufacturing costs, transportation and freight, amortization of capitalized intangible assets, royalties and direct and indirect overhead costs associated with the manufacturing and distribution of the Company's approved products.
+Added: Cost of sales may also include period costs related to certain manufacturing services and inventory adjustment charges.
Research and Development Expenses
14 unchanged sentences
The Company accounts for forfeitures as they occur.
−Removed: The Company evaluates the grant date fair value of awards granted for impacts of any potential material non-public information at the time of grant.
Foreign Currency
6 unchanged sentences
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 its subsidiary that recorded the transaction.
−Removed: Unrealized foreign exchange gains and losses amounted to a $ 1.2 million gain for the year ended December 31, 2024, a $ 1.8 million loss for the year ended December 31, 2023, and were insignificant for the year ended December 31, 2022.
−Removed: Realized foreign exchange gains and losses were insignificant for the years ended December 31, 2024, 2023 and 2022.
+Added: Unrealized foreign exchange gains and losses amounted to a $ 1.7 million and a $ 1.2 million gain for the years ended December 31, 2025 and 2024, respectively, and $ 1.8 million loss for the year ended December 31, 2023.
+Added: Realized foreign exchange losses amounted to a $ 1.0 million loss for the year ended December 31, 2025 and were insignificant for the years ended December 31, 2024 and 2023.
Income taxes are recorded using the liability method, under which deferred tax assets and liabilities are determined based on differences between the financial reporting and tax basis of assets and liabilities and are measured using the enacted tax rates and laws that will be in effect when the differences are expected to reverse.
9 unchanged sentences
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 and common stock issuable upon conversion of convertible notes because their effect would be anti-dilutive due to the Company’s net loss.
−Removed: The following table sets forth the computation of basic and diluted earnings per share for the years ended December 31, 2024, 2023 and 2022 (in thousands, except share and per share data):
−Removed: Year Ended December 31,
−Removed: 2024 2023 2022
−Removed: Net loss, basic $ ( 87,942 ) $ ( 163,415 ) $ ( 135,665 )
−Removed: Change in fair value of Holdback Indemnification liability — — ( 214 )
−Removed: Change in fair value of Contingent Milestone liability — — ( 700 )
−Removed: Net loss, diluted $ ( 87,942 ) $ ( 163,415 ) $ ( 136,579 )
−Removed: Weighted-average shares of common stock outstanding, basic 47,522,594 40,885,124 33,839,072
−Removed: Effect of dilutive securities:
−Removed: Weighted-average Holdback-Indemnification shares issuable — — 19,590
−Removed: Weighted-average contingent milestone shares — — 123,831
−Removed: Weighted-average shares of common stock outstanding, diluted 47,522,594 40,885,124 33,982,493
−Removed: Net loss per share, basic $ ( 1.85 ) $ ( 4.00 ) $ ( 4.01 )
−Removed: Net loss per share, diluted $ ( 1.85 ) $ ( 4.00 ) $ ( 4.02 )
The following outstanding potential dilutive shares have been excluded from the calculation of diluted net loss per share for the periods presented due to their anti-dilutive effect:
6 unchanged sentences
Recently Adopted Accounting Pronouncements
−Removed: On January 1, 2024, the Company adopted Accounting Standards Update (“ASU”) No.
−Removed: 2023-07, Segment Reporting (Topic 280):
−Removed: Improvements to Reportable Segment Disclosures (“ASU 2023-07”).
−Removed: This new guidance is designed to improve reportable segment disclosure requirements, primarily through enhanced disclosures about significant segment expenses and is effective for public business entities for annual periods beginning after December 15, 2023, and interim periods beginning after December 15, 2024.
−Removed: The Company adopted ASU 2023-07 in 2024 using a retrospective method for all periods presented.
−Removed: For further information, refer to Note 14 Segment Reporting .
−Removed: Recent Accounting Pronouncements Not Yet Adopted
−Removed: 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.
−Removed: 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 consolidated financial statements and disclosures.
−Removed: In December 2023, the FASB issued ASU No.
+Added: In December 2023, the Financial Accounting Standards Board (“FASB”) issued Accounting Standards Update (“ASU”) No.
+Added: 2023-09, Income Taxes (Topic 740):
Improvements to Income Tax Disclosures (“ASU 2023-09”).
2 unchanged sentences
ASU 2023-09 is effective for fiscal years beginning after December 15, 2024.
−Removed: Early adoption is permitted.
−Removed: The Company is currently assessing the impact that adopting this new accounting standard will have on its consolidated financial statements.
+Added: The Company adopted ASU 2023-09 in 2025 using a prospective method.
+Added: For further information, refer to Note 13 Income Taxes .
+Added: Recent Accounting Pronouncements Not Yet Adopted
+Added: 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.
+Added: 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 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):
−Removed: Disaggregation of Income Statement Expenses (“ASU 2024-03”), which requires disclosure of disaggregated information about specific categories underlying certain income statement
−Removed: expense line items in the footnotes to the financial statements for both annual and interim periods.
+Added: 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.
−Removed: ASU 2024-03 is effective for fiscal years beginning after December 15, 2026, and interim reporting periods beginning after December 15, 2027.
+Added: 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.
+Added: In July 2025, the FASB issued ASU 2025-05, Financial Instruments - Credit Losses (Topic 326):
+Added: Measurement of Credit Losses for Accounts Receivable and Contract Assets (“ASU 2025-05”).
+Added: 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.
+Added: ASU 2025-05 is effective for annual reporting periods beginning after December 15, 2025, and interim reporting periods within those annual reporting periods.
+Added: Early adoption is permitted.
+Added: The Company does not expect that adoption of this new accounting standard will have a material impact on its consolidated financial statements.
+Added: In September 2025, the FASB issued ASU 2025-06, Intangibles - Goodwill and Other - Internal-Use Software (Subtopic 350-40):
+Added: 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.
+Added: The guidance removes references to development stages and clarifies when capitalization of software costs should occur.
+Added: 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.
+Added: Early adoption is permitted as of the beginning of an annual reporting period.
+Added: The Company is currently assessing the impact that adopting this new accounting standard will have on its consolidated financial statements.
Fair Value Measurements
4 unchanged sentences
Money market funds $ 254,376 $ — $ — $ 254,376
−Removed: treasury bills 2,964 — — 2,964
Corporate debt securities — 72,277 — 72,277
government bonds — 17,016 — 17,016
−Removed: Agency bonds — 2,492 — 2,492
+Added: Commercial paper — 5,456 — 5,456
Total financial assets $ 254,376 $ 94,749 $ — $ 349,125
3 unchanged sentences
Money market funds $ 202,965 $ — $ — $ 202,965
+Added: treasury bills $ 2,964 $ — $ — $ 2,964
+Added: Corporate debt securities $ — $ 37,978 $ — $ 37,978
+Added: government bonds $ — $ 26,904 $ — $ 26,904
+Added: Agency bonds $ — $ 2,492 $ — $ 2,492
Total financial assets $ 205,929 $ 67,374 $ — $ 273,303
6 unchanged sentences
The Company reviews trading activity and pricing for these investments as of each measurement date.
−Removed: Derivative Liability
−Removed: The debt pursuant to the RIPA (refer to Note 6 “Revenue Interest Purchase Agreement” for further information) contained an embedded derivative requiring bifurcation as a single compound derivative instrument.
−Removed: The Company estimated the fair value of the derivative liability using a “with-and-without” method.
−Removed: The “with-and-without” methodology involves valuing the whole instrument on an as-is basis and then valuing the instrument without the individual embedded derivative.
−Removed: In 2023, there was no change in the fair value of the derivative liability prior to its extinguishment in connection with the termination of the RIPA in April 2023.
−Removed: Indemnification Holdback and Contingent Milestone Liabilities
−Removed: In May 2022, in connection with the acquisition of Satiogen Pharmaceuticals, Inc.
−Removed: (“Satiogen”) (refer to Note 7 “Asset Acquisitions” for further information), the Company recorded at fair value liabilities related to the Company’s common stock issuable upon satisfaction of certain purchase price adjustments and indemnification obligations and contingent consideration upon achievement of a certain milestone.
−Removed: The fair value of the liabilities was classified within
−Removed: Level 3 of the fair value hierarchy and was estimated based upon the value of the Company’s common stock price, management’s estimate of the probability of indemnification obligations being incurred during the one year following the acquisition date, and management’s estimate of the probability of the milestone being met.
−Removed: The fair value of the liabilities was initially measured on May 20, 2022, the date on which the Company completed the acquisition of Satiogen.
−Removed: The Company assessed the fair value of the liabilities each reporting period until resolution of the related contingency, and changes in fair value were recorded in other income (expense), net in the accompanying consolidated statements of operations.
−Removed: The contingencies were resolved in December 2022 and June 2023 and the Company issued 199,993 and 31,631 shares of common stock, respectively.
−Removed: The change in fair value of the liabilities was insignificant for the years ended December 31, 2023 and 2022.
−Removed: There was no outstanding balance related to the liabilities as of December 31, 2023.
Financial Instruments
6 unchanged sentences
Money market funds $ 254,376 $ — $ — $ 254,376
−Removed: treasury bills 2,957 7 — 2,964
Corporate debt securities 72,170 107 — 72,277
government bonds 16,984 32 — 17,016
−Removed: Agency bonds 2,490 2 — 2,492
+Added: Commercial paper 5,455 1 — 5,456
Total cash equivalents and investments $ 348,985 $ 140 $ — $ 349,125
10 unchanged sentences
Money market funds $ 202,965 $ — $ — $ 202,965
−Removed: Total cash equivalents $ 278,116 $ — $ — $ 278,116
+Added: treasury bills $ 2,957 $ 7 $ — $ 2,964
+Added: Corporate debt securities $ 37,942 $ 50 $ ( 14 ) $ 37,978
+Added: government bonds $ 26,819 $ 90 $ ( 5 ) $ 26,904
+Added: Agency bonds $ 2,490 $ 2 $ — $ 2,492
+Added: Total cash equivalents and investments $ 273,173 $ 149 $ ( 19 ) $ 273,303
Classified as:
Cash equivalents $ 202,965
−Removed: Total cash equivalents $ 278,116
+Added: Short-term investments 57,812
+Added: Long-term investments 12,526
+Added: Total cash equivalents and investments $ 273,303
As of December 31, 2025, the remaining contractual maturities of available-for-sale debt securities were as follows (in thousands):
10 unchanged sentences
Total inventory $ 24,887 $ 22,403
+Added: Intangible Assets, Net
+Added: The components of the Company’s intangible assets were as follows (in thousands, except for weighted-average remaining amortization period):
+Added: December 31, 2025
+Added: Gross Carrying Value Accumulated Amortization
+Added: Net Carrying Amount Weighted-Average Remaining Amortization Period (Years)
+Added: Commercial milestones $ 94,000 $ ( 11,660 ) $ 82,340 11.5
+Added: Developed technology 226,620 ( 48,255 ) 178,365 9.7
+Added: Assembled workforce 970 ( 754 ) 216 0.7
+Added: Total intangible assets $ 321,590 $ ( 60,669 ) $ 260,921 10.3
+Added: December 31, 2024
+Added: Gross Carrying Value Accumulated Amortization
+Added: Net Carrying Amount Weighted-Average Remaining Amortization Period (Years)
+Added: Commercial milestones $ 59,000 $ ( 7,092 ) $ 51,908 12.6
+Added: Developed technology 226,620 ( 29,248 ) 197,372 10.7
+Added: Assembled workforce 970 ( 431 ) 539 1.7
+Added: Total intangible assets $ 286,590 $ ( 36,771 ) $ 249,819 11.1
+Added: Amortization expense was $ 23.9 million, $ 23.1 million and $ 10.5 million for the years ended December 31, 2025, 2024 and 2023, respectively, and was included in cost of sales on the accompanying consolidated statements of operations.
+Added: The following table summarizes the estimated future amortization expense associated with the Company’s intangible assets as of December 31, 2025 (in thousands):
+Added: 2026 $ 26,958
+Added: Thereafter 126,995
Accrued Expenses
2 unchanged sentences
Accrued compensation and related benefits 47,276 32,248
−Removed: Accrued professional service fees 12,545 7,941
−Removed: Accrued contract manufacturing and non-clinical costs 9,453 9,922
Accrued royalties payable 16,208 9,361
+Added: Accrued professional service fees 17,292 12,545
Accrued clinical trials 13,959 7,148
+Added: Accrued contract manufacturing and non-clinical costs 8,721 7,555
+Added: Accrued loss on firm purchase commitments 2,107 1,898
+Added: Operating lease liabilities, current 2,139 1,709
Accrued interest 2,108 2,108
−Removed: Total accrued expenses $ 110,224 $ 78,544
+Added: Accrued milestone payment 35,000 —
+Added: Total accrued expenses and other current liabilities $ 196,185 $ 111,933
+Added: Other liabilities
+Added: Other liabilities consist of the following (in thousands):
+Added: Noncurrent accrued sales deductions and other $ 3,586 $ —
+Added: Noncurrent accrued loss on firm purchase commitments 1,425 2,509
+Added: Total other liabilities $ 5,011 $ 2,509
Revenue Interest Purchase Agreement
In December 2020, the Company entered into the RIPA, as amended in September 2021, with Oberland as agent for the Purchasers, and the Purchasers to obtain financing for the commercialization and further development of Livmarli and other working capital needs.
−Removed: Pursuant to the RIPA, the Company received $ 115.0 million consisting of an upfront payment of $ 50.0 million in December 2020 and $ 65.0 million in April 2021 associated with the acceptance for filing by the FDA of a New Drug Application for Livmarli for the treatment of cholestatic pruritus in patients with ALGS, less certain transaction expenses.
−Removed: Under the RIPA, the Company was entitled to receive an additional $ 35.0 million upon FDA approval of Livmarli, which it elected to forgo.
−Removed: The Company was also entitled to receive up to approximately $ 50.0 million at the option of the Purchasers to finance in-licenses or other acquisitions on or prior to December 31, 2022, which the Company did not request.
−Removed: As consideration for such payments, the Purchasers had the right to receive the Revenue Interests from the Company based on annual product sales, net of Livmarli, in tiered payments (the “Revenue Interest Payments”) based on whether annual product sales, net were (i) less than or equal to $ 350.0 million (“Tier 1”), (ii) exceeding $ 350.0 million and less than or equal to $ 1.1 billion (“Tier 2”), or (iii) exceeding $ 1.1 billion (“Tier 3”).
−Removed: The Revenue Interest Payments were initially 9.75 % (at Tier 1) and 2.0 % (at Tier 2 and Tier 3) of such annual net sales.
+Added: Pursuant to the RIPA, the Company received a total of $ 115.0 million.
+Added: As consideration for such payments, the Purchasers had the right to receive the Revenue Interests from the Company based on annual product sales, net of Livmarli.
Under the RIPA, the Company had an option (the “Call Option”) to terminate the RIPA and repurchase future Revenue Interests at any time upon advance written notice.
If the Call Option was exercised, the required repurchase price is 175.0 % of the Cumulative Purchaser Payments (minus all payments the Company has made to the Purchasers in connection with the Revenue Interests), if such option was exercised prior to the third anniversary of the closing date, and 195.0 % of the Cumulative Purchaser Payments (minus all payments Company has made to the Purchasers in connection with the Revenue Interests) if such option was exercised thereafter.
−Removed: In addition, the RIPA contained various representations and warranties, information rights, non-financial and financial covenants, indemnification obligations and other provisions that are customary for a transaction of this nature.
−Removed: The Purchasers’ obligations to fund the scheduled installments were subject to certain customary conditions as set forth in the RIPA.
−Removed: Concurrently with the RIPA, the Company entered into a Common Stock Purchase Agreement (“CSPA”) with certain affiliates of Oberland, pursuant to which the Company sold an aggregate of 509,164 shares of its common stock for an aggregate purchase price of $ 10.0 million.
−Removed: The $ 50.0 million upfront payment received pursuant to the RIPA and $ 10.0 million received pursuant to the CSPA was allocated between the resulting financial instruments on a relative fair value basis, with $ 49.2 million allocated to the debt under the RIPA and $ 10.8 million allocated to the common stock issued under the CSPA.
−Removed: The Company imputed interest expense associated with the revenue interest liability using the effective interest rate method.
−Removed: The effective interest rate was calculated based on the rate that would enable the debt to be repaid in full over the anticipated life of the arrangement.
−Removed: The interest rate on this liability was variable during the term of the agreement depending on a number of factors, including the level of forecasted product sales, net.
−Removed: The Company evaluated the interest rate quarterly based on each period's product sales, net forecasts utilizing the prospective method.
−Removed: The Company recorded interest expense related to this arrangement of $ 5.1 million and $ 16.0 million for the years ended December 31, 2023 and 2022, respectively.
−Removed: No interest expense related to this arrangement was recorded for the year ended December 31, 2024.
−Removed: The Company incurred $ 0.9 million of issuance costs in connection with the RIPA, which were being amortized to interest expense over the estimated term of the debt.
+Added: The Company recorded interest expense related to this arrangement of $ 5.1 million for the year ended December 31, 2023.
+Added: No interest expense related to this arrangement was recorded for the years ended December 31, 2025 and 2024.
In April 2023, the Company exercised the Call Option and repurchased all future Revenue Interests.
1 unchanged sentence
As a result, the RIPA terminated in accordance with its terms.
−Removed: The following table summarizes the revenue interest liability activity during the year ended December 31, 2023 (in thousands):
−Removed: Revenue Interest Liability Derivative Liability
−Removed: Balance at December 31, 2022 $ 140,351 $ 1,090
−Removed: Interest expense recognized 5,060 —
−Removed: Revenue interest payments ( 2,883 ) —
−Removed: Revenue interest liability settlement ( 192,694 ) —
−Removed: Loss (gain) from termination of revenue interest purchase agreement 50,166 ( 1,090 )
−Removed: Balance at December 31, 2023 $ — $ —
The net loss from termination of the RIPA of $ 49.1 million, comprised of the $ 50.2 million loss related to the settlement of the revenue interest liability and the $ 1.1 million gain on the derecognition of the related derivative liability, was recorded in the accompanying consolidated statements of operations.
−Removed: As of December 31, 2023, there were no outstanding balances related to the revenue interest liability and the derivative liability.
+Added: As of December 31, 2025 and 2024, there were no outstanding balances related to the revenue interest liability and the derivative liability.
Asset Acquisitions
3 unchanged sentences
The Company determined that the intellectual property assets acquired did not have an alternate future use and were not deemed to be commercially viable.
−Removed: the upfront payment of $ 7.5 million was recorded as research and development expense in the year ended December 31, 2024.
+Added: Therefore, the upfront payment of $ 7.5 million was recorded as research and development expense in the year ended December 31, 2024.
+Added: Through December 31, 2025, no milestones have been achieved.
Asset Purchase Agreement with Travere Therapeutics, Inc.
−Removed: On August 31, 2023, the Company completed the Bile Acid Portfolio Acquisition.
+Added: In August 2023, the Company completed the Bile Acid Portfolio Acquisition.
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.
−Removed: The Company assessed the fair value of this contingent consideration in its determination of whether the acquisition of the Bile Acid Portfolio should be accounted for as an asset acquisition.
−Removed: The Company estimated the fair value of the contingent consideration using a Monte Carlo simulation model, which includes the amount and timing of revenue projections subsequent to the acquisition date as a significant assumption.
−Removed: The Company and Travere concurrently entered into a transitional services agreement pursuant to which Travere was obligated to perform certain services for a period of time, not anticipated to last beyond 12 months post-close, with respect to the Company’s use and operation of the assets purchased.
−Removed: The transitional services agreement has since terminated according to its terms.
−Removed: The Company accounted for the transaction as an asset acquisition as substantially all the fair value of the gross assets acquired was concentrated in a group of similar identifiable assets, namely, the developed technology related to the Bile Acid Medicines.
−Removed: The developed technology asset consists of certain processes and at-market contracts related to the manufacture and commercialization of the Bile Acid Medicines, regulatory approvals, and other assets, and are considered a single asset as they are interdependently linked.
−Removed: The Company estimated the fair value of the developed technology asset using the discounted cash flow method.
−Removed: This approach incorporates significant estimates and assumptions related to the forecasted results including, but not limited to, estimates and assumptions regarding revenues, expenses, and discount rates to estimate future cash flows.
−Removed: The Company determined that the developed technology asset acquired was commercially viable and the cost of the acquisition was recorded as an intangible asset in the accompanying consolidated balance sheet as of the acquisition date.
−Removed: The acquired assets are amortizable for tax purposes.
+Added: In connection with the Bile Acid Portfolio Acquisition, the Company recorded $ 198.5 million of developed technology intangible assets on the consolidated balance sheet at the time of the acquisition.
+Added: As of December 31, 2025, the Company accrued $ 25.0 million for the achievement of product sales milestones associated with achievement of certain net product sales, which was recognized as an intangible asset in the accompanying consolidated balance sheet as of December 31, 2025.
+Added: Through December 31, 2025, the Company paid or accrued $ 25.0 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.
7 unchanged sentences
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.
−Removed: Upon commercialization, the Company is obligated to pay Shire product sales milestones on total licensed products up to an aggregate of $ 30.0 million.
−Removed: Through December 31, 2024 under this agreement, the Company paid $ 86.5 million for the achievement of various clinical development, regulatory and commercial milestones.
+Added: Upon commercialization, the Company is obligated to pay Shire commercial milestones on total licensed products up to an aggregate of $ 30.0 million.
+Added: Through December 31, 2025 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;
1 unchanged sentence
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.
−Removed: In January 2023, the Company paid a regulatory milestone of $ 15.0 million associated with approval of Livmarli by the European Commission for the treatment of cholestatic pruritus in patients with ALGS two months of age and older.
−Removed: In December 2023, the Company paid a commercial milestone of $ 5.0 million associated with product sales.
In April 2024, the Company paid a $ 10.0 million 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.
In July 2024, the Company paid a $ 10.0 million milestone associated with the approval of Livmarli for the treatment of PFIC in patients three months and older by the European Medicines Agency.
+Added: In February 2025, the Company achieved a $ 5.0 million development milestone related to maralixibat, which was recognized in research and development expense in the accompanying consolidated statements of operations.
+Added: 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 in the accompanying consolidated balance sheet as of December 31, 2025.
There were no volixibat development and regulatory milestones achieved during the years ended December 31, 2025, 2024 and 2023.
−Removed: Satiogen License
−Removed: Through the Shire Agreement, the Company was assigned a license agreement with Satiogen 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.
−Removed: Pursuant to the terms of the license agreement, the Company was obligated to pay to Satiogen up to an aggregate of $ 10.5 million upon the achievement of certain milestones, of which $ 0.5 million was for initiation of certain development activities, $ 5.0 million for the completion of regulatory approvals and $ 5.0 million for commercialization activities.
−Removed: Additionally, the Company was required to pay a low single-digit royalty on net sales.
−Removed: The Company’s royalty obligations continued on a licensed product-by-licensed product and country-by-country basis until the expiration of the last valid claim in a licensed patent covering the applicable licensed product in such country.
−Removed: Royalty obligations under the Satiogen license were creditable against the royalty obligations to Shire under the Shire Agreement.
−Removed: The Company has not paid milestone payments pursuant to this agreement for the periods presented.
−Removed: In May 2022, the Company completed the merger and acquisition of Satiogen for total consideration of approximately $ 24.2 million.
−Removed: At acquisition, Satiogen’s assets consisted of cash and intangible assets related to developed technology.
−Removed: The purchase consideration consisted of 609,305 shares of the Company’s common stock issued upon the closing of the acquisition and cash consideration of $ 2.6 million, excluding $ 0.2 million of stock option exercise prices deemed to have been paid immediately prior to the acquisition, in respect of an equivalent amount of cash on the books of Satiogen.
−Removed: In 2023, upon the resolution of the related contingencies, the Company issued an additional 231,624 shares of common stock as purchase consideration.
−Removed: Through the transaction, the Company obtained all Satiogen licensing payments and Satiogen-owned intellectual property relating to Livmarli and volixibat.
−Removed: The transaction resulted in a reduction of total licensing royalty obligations for Livmarli and volixibat.
−Removed: 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, the Satiogen intangible assets consisted of intellectual property.
−Removed: The Company evaluated that the intellectual property assets acquired were deemed to be commercially viable and the cost of the acquisition was recorded as an intangible asset.
−Removed: There was no gain or loss recognized from settlement of the preexisting contractual relationship with Satiogen as the pre-existing contract was determined to be at fair value on the date of acquisition.
−Removed: Through the date of settlement in June 2023, there were no significant expenses incurred that were approved for settlement against the Indemnification Holdback.
−Removed: As the number of shares potentially issuable upon the resolution of the Indemnification Holdback and the Contingent Milestone was variable, they were recorded as liabilities at their respective fair values on the date of acquisition using the Company’s common stock price.
−Removed: The fair value of the Indemnification Holdback was additionally determined based on management’s estimate of the probability of indemnification obligations being incurred during the one year following the acquisition date, while the fair value of the Contingent Milestone was additionally determined based upon management’s estimate of the probability of the milestone being met until the contingency was resolved in December 2022.
−Removed: The fair value of the Indemnification Holdback liability and the Contingent Milestone liability were remeasured at each reporting period until settled, with resulting changes in the fair value recorded in other income (expense) in the accompanying consolidated statements of operations.
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.
−Removed: Upon commercialization of any product utilizing the licensed product, the Company will be required to pay to Pfizer a low single-digit royalty on net sales of product sold by the Company, its affiliates or sublicensees.
+Added: 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.
6 unchanged sentences
The Company has not paid milestone payments pursuant to this agreement for the periods presented.
−Removed: As of December 31, 2024, no milestones had been accrued as there were no potential milestones considered probable.
+Added: Through December 31, 2025, no milestones have been achieved.
Collaboration and License Agreements
−Removed: License and Collaboration Agreement with CANbridge
−Removed: In April 2021, the Company entered into an exclusive license and collaboration agreement with CANbridge Pharmaceuticals, Inc.
−Removed: (“CANbridge”), as amended in March 2024.
−Removed: Under the terms of the agreement, CANbridge has obtained the exclusive right to develop and commercialize Livmarli within the Greater China regions (China, Hong Kong, Macau and Taiwan).
−Removed: In connection with the agreement, the Company received an upfront payment of $ 11.0 million, which, upon satisfaction of the performance obligation and receipt by CANbridge of the right to use and benefit from the license in May 2021, was recorded as license revenue.
−Removed: Additionally, the Company is eligible to receive up to $ 5.0 million in research and development funding, and up to $ 109.0 million for the achievement of future regulatory and commercial milestones, with double-digit tiered royalties based on product net sales.
−Removed: Through December 31, 2024, the Company has received research funding of $ 5.0 million and regulatory milestone payments of $ 7.3 million.
−Removed: The Company concluded at inception of the agreement that the transaction price should not include the variable consideration related to unachieved developmental and regulatory milestones as this consideration was considered to be constrained as it is probable that the inclusion of such variable consideration could result in a significant reversal in cumulative revenue.
−Removed: The Company will recognize any consideration related to sales-based payments when the related sales occur, as the Company has determined that these amounts relate predominantly to the license granted and therefore will be recognized at the later of (i) when or as the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied).
−Removed: The Company re-evaluates the transaction price at each reporting period as uncertain events are resolved and other changes in circumstances occur.
−Removed: In January 2022 and June 2023, CANbridge achieved regulatory milestones, triggering milestone payments to the Company of $ 2.0 million and $ 5.0 million, respectively, which were recorded as license revenue in the accompanying consolidated statements of operations for the years ended December 31, 2023 and 2022, respectively.
−Removed: Research and development funding payable by CANbridge to the Company, which is reflected as a reduction of research and development expense in the accompanying consolidated statements of operations for the year ended December 31, 2024, was insignificant.
−Removed: For the years ended December 31, 2023, and 2022 , the Company recorded research and development funding of $ 2.3 million and $ 0.9 million, respectively.
−Removed: As of December 31, 2024 and 2023, such research and development funding included in accounts receivable on the accompanying consolidated balance sheets was insignificant.
−Removed: License and Collaboration Agreement with GC Biopharma
−Removed: In July 2021, the Company entered into an exclusive license and collaboration agreement with GC Biopharma.
−Removed: Under the terms of the agreement, GC Biopharma has obtained the exclusive right to develop and commercialize Livmarli within South Korea for ALGS, PFIC, and biliary atresia (“BA”).
−Removed: In connection with the agreement, the Company received a $ 5.0 million upfront payment, which, upon satisfaction of the performance obligation and receipt by GC Biopharma of the right to use and benefit from the license, was recorded as license revenue.
−Removed: Additionally, the Company is entitled to certain research and development funding and up to $ 23.0 million for the achievement of future regulatory and commercial milestones, with double-digit tiered royalties based on product net sales.
−Removed: At inception of the agreement, the Company concluded that the transaction price should not include the variable consideration related to unachieved developmental and regulatory milestones as this consideration was considered to be constrained as it is probable that the inclusion of such variable consideration could result in a significant reversal in
−Removed: cumulative revenue for this contract when the uncertainty is resolved in the future.
−Removed: The Company will recognize any consideration related to sales-based payments (including milestones and royalties) when the related sales occur, as the Company has determined that these amounts relate predominantly to the license granted and therefore will be recognized on the later to occur of satisfaction of the performance obligation or the occurrence of the related sales.
−Removed: The Company re-evaluates the transaction price at each reporting period as uncertain events are resolved and other changes in circumstances occur.
−Removed: In February 2023, GC Biopharma achieved a regulatory milestone under this agreement triggering a milestone payment to the Company of $ 2.5 million, which upon the release of the constraint was recognized as license revenue in the accompanying consolidated statements of operations for the year ended December 31, 2023.
−Removed: For the years ended December 31, 2024, 2023 and 2022, research and development funding payable by GC Biopharma to the Company was insignificant.
−Removed: As of December 31, 2024 and 2023, such research and development funding recorded as a receivable in accounts receivable on the accompanying consolidated balance sheets was insignificant.
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.
−Removed: Under the terms of the agreement, Takeda will be responsible for regulatory approval and commercialization of Livmarli in Japan.
−Removed: Takeda will also be responsible for development, including conducting clinical studies in cholestatic indications.
−Removed: The Company is responsible for commercial supply to Takeda which was to be negotiated at a later date.
−Removed: Subsequently, in October 2024, the Company entered into a commercial supply agreement with Takeda.
−Removed: In accordance with the agreements, in exchange for the supply of goods, the Company is eligible to receive a percentage of Takeda’s annual 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.
−Removed: The revenues related to the commercial supply will be recognized in product sales, net upon the transfer of control to Takeda.
−Removed: In January 2019, the Company entered into an operating lease agreement for office space which consisted of approximately 5,600 square feet (the “Initial Lease”).
−Removed: The lease term was approximately four years with an option to extend the term for one five -year term, which at the time was not reasonably assured of exercise and therefore, not included in the lease term.
−Removed: Rent payments commenced in August 2019.
−Removed: In November 2019, the Company amended the operating lease agreement (the “Amended Agreement”) to extend the term of the Initial Lease through March 2025.
−Removed: This extension was accounted for as a lease modification.
−Removed: Additionally, pursuant to the Amended Agreement, the Company expanded the office space by 5,555 square feet for a five -year term expiring in March 2025 (the “Expanded Space”).
−Removed: The Company accounted for the Expanded Space as a separate contract as there were material additional rights of use that were not included in the Initial Lease.
−Removed: The Amended Agreement contained a tenant improvement allowance of $ 0.8 million in connection with the expanded space, which has been recorded as leasehold improvements within property and equipment, net on the accompanying consolidated balance sheets with a corresponding reduction of the ROU asset at inception of the lease for the expanded space.
−Removed: In January 2024, the Company entered into an operating lease agreement for approximately 36,300 square feet of office space (the “New Lease”).
−Removed: The lease term is approximately five years .
−Removed: Concurrently with entering into the New Lease, the Company entered into an amendment of its existing Amended Agreement to accelerate the lease expiration date from March 2025 to shortly after the commencement date of the New Lease.
−Removed: The New Lease commenced and the Amended Agreement, as amended, terminated in May 2024.
+Added: Further, in October 2024, the parties entered into a commercial supply agreement.
+Added: 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.
+Added: 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.
+Added: The Company records net product sales at the time control of the product is transferred, based on the estimated variable consideration.
+Added: 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”).
+Added: In January 2026, the Company amended the Initial Lease (the “Amended Agreement”) to expand its headquarters office space by approximately 19,400 square feet (the “Expanded Space”) with a lease term of approximately five years from the commencement date.
+Added: The Amended Agreement extends the lease term of the Initial Lease to be coterminous with the Expanded Space.
+Added: The addition of the expanded space will result in approximately $ 10.6 million of additional base rent compared to the remaining payments of the Initial Lease.
+Added: In March 2025, the Company entered into an operating lease agreement for office space at an international location.
+Added: The lease commenced in the second quarter of 2025 and has a lease term of approximately five years .
+Added: In December 2025, the Company amended the terms of a lease at another international location to include additional space with a five-year lease term expiring January 2031 and a commencement date of December 2025.
+Added: In addition, the amendment extended the lease term of the existing space to the same term.
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:
−Removed: As of December 31,
+Added: December 31, 2025
Weighted-average incremental borrowing rate 7.4 %
−Removed: Weighted-average remaining lease term (in years) 4.7 years 2.1 years
+Added: Weighted-average remaining lease term (in years) 4.0 years
Years Ended December 31, Undiscounted
1 unchanged sentence
(in thousands)
+Added: Thereafter 21
Total undiscounted lease payments 11,122
1 unchanged sentence
Total lease liability $ 9,655
−Removed: Rent expense was $ 1.8 million for the year ended December 31, 2024, and $ 0.8 million for each of the years ended December 31, 2023 and 2022.
+Added: Rent expense was $ 2.4 million, $ 1.8 million and $ 0.8 million for the years ended December 31, 2025, 2024 and 2023, respectively.
Variable lease payments for the years ended December 31, 2025, 2024 and 2023 were insignificant.
18 unchanged sentences
The initial conversion rate for the Notes is 31.5075 shares of common stock per $ 1,000 principal amount of Notes, which represents an initial conversion price of approximately $ 31.74 per share of common stock.
−Removed: The conversion rate and conversion price will be subject to customary adjustments upon the occurrence of certain
+Added: The conversion rate and conversion price will be 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 Indenture) occur, then the conversion rate will, in certain circumstances, be increased for a specified period of time.
1 unchanged sentence
As a result, the Notes are convertible at the option of the holders of the Notes during the first quarter of 2026, the quarter immediately following the quarter when the conditions were met, as stated in the terms of the Notes.
−Removed: Through the date of this filing, none of the principal balance of the Notes has been converted.
The Company may not redeem the Notes at its option at any time before May 5, 2026.
12 unchanged sentences
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.
−Removed: As of December 31, 2024, the remaining amortization period of the debt discount was approximately 4.3 years and the effective interest on the Notes was 4.6 %.
+Added: As of December 31, 2025, the
+Added: remaining amortization period of the debt discount was approximately 3.3 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):
Year Ended December 31,
+Added: 2025 2024 2023
Coupon interest expense $ 12,650 $ 12,650 $ 8,925
4 unchanged sentences
Stockholders’ Equity
−Removed: In August 2020, the SEC declared effective a registration statement on Form S-3 (“2020 Shelf Registration”) covering the sale of up to $ 300.0 million of the Company’s securities.
−Removed: Also, in August 2020, the Company entered into a sales agreement (“2020 Sales Agreement”) with SVB Securities LLC, recently acquired by Leerink Partners LLC (“Leerink”) pursuant to which the Company could elect to issue and sell, from time to time, shares of common stock having an aggregate offering price of up to $ 75.0 million under the 2020 Shelf Registration through Leerink acting as the sales agent and/or principal.
−Removed: During the year ended December 31, 2023, the Company issued and sold 658,206 shares of common stock pursuant to the 2020 Sales Agreement resulting in gross proceeds to the Company of $ 15.0 million.
−Removed: The net proceeds for the year ended December 31, 2023 to the Company after deducting sales commissions to SVB Securities and other issuance expenses were approximately $ 14.5 million.
−Removed: The 2020 Shelf Registration expired in August 2023, and no further sales may be made under the 2020 Sales Agreement.
−Removed: Through its expiration date, the Company issued and sold an aggregate of 2,125,090 shares of common stock pursuant to the 2020 Sales Agreement resulting in aggregate gross proceeds to the Company of $ 43.7 million.
−Removed: In August 2022, the Company completed an underwritten public offering of its common stock pursuant to the 2020 Shelf Registration.
−Removed: The Company issued and sold 3,478,261 shares of common stock at a public offering price of $ 23.00 per share.
−Removed: In addition, the Company granted the underwriters an option, exercisable for 30 days, to purchase up to 521,739 additional shares of its common stock at the public offering price, less the underwriting discounts, commissions and offering expenses, which the underwriters exercised in full.
−Removed: The underwritten public offering, including the underwriters’ exercise of their option, resulted in net proceeds to the Company of $ 86.1 million after deducting underwriting discounts, commissions and offering expenses.
−Removed: On September 9, 2022, the Company filed an automatic shelf registration statement on Form S-3 with the SEC (the “2022 Shelf Registration”), 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.
+Added: In August 2025, the Company filed an automatic shelf registration statement on Form S-3 with the SEC (the “2025 Shelf Registration”), which became effective upon filing, pursuant to which the Company may register 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.
−Removed: As of December 31, 2024, the Company had not issued any securities pursuant to the automatic shelf registration statement.
−Removed: On August 31, 2023, in connection with and immediately prior to the closing of the Bile Acid Portfolio Acquisition, the Company completed the private placement of 8,000,000 shares of the Company’s common stock at a price per share of $ 26.25 , resulting in net proceeds of approximately $ 202.2 million, which the Company used to finance the upfront payment at the closing of the Bile Acid Portfolio Acquisition.
−Removed: On November 2, 2023, the Company entered into a Sales Agreement (the “2023 Sales Agreement”) with Leerink and Cantor Fitzgerald & Co.
+Added: As of December 31, 2025, the Company had not issued any securities pursuant to the 2025 Shelf Registration.
+Added: In November 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 “ATM Offering”).
3 unchanged sentences
As of December 31, 2025, the Company had not issued any securities pursuant to the 2023 Sales Agreement.
+Added: In August 2023, in connection with and immediately prior to the closing of the Bile Acid Portfolio Acquisition, the Company completed the private placement of 8,000,000 shares of the Company’s common stock at a price per share of $ 26.25 , resulting in net proceeds of approximately $ 202.2 million, which the Company used to finance the upfront payment at the closing of the Bile Acid Portfolio Acquisition.
Common Stock Reserved for Issuance
30 unchanged sentences
Vested and exercisable as of December 31, 2025 5,747,724 $ 17.61 5.4 $ 352,815
−Removed: 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.
+Added: Intrinsic value is calculated as the difference between the exercise price of the underlying options and the fair value of the common stock at December 31, 2025 or on the date of exercise, 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.
The weighted-average grant date fair value per share of stock options granted during the years ended December 31, 2025, 2024 and 2023 was $ 32.71 , $ 20.02 and $ 18.25 per share, respectively.
6 unchanged sentences
The risk-free interest rate is determined by reference to the U.S.
−Removed: Treasury yield curve in effect at the time of grant of the award for
−Removed: time periods approximately equal to the expected term of the award.
+Added: 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.
25 unchanged sentences
As of December 31, 2025, certain specified sales-based criteria were deemed probable of achievement or already achieved.
−Removed: 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
+Added: 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 a three-year service period.
1 unchanged sentence
The following table summarizes the activity under the Company’s performance stock units in base units for the year ended December 31, 2025:
−Removed: Awards in base units Weighted-Average Grant Date
+Added: Awards Weighted-Average Grant Date
Fair Value per Award
7 unchanged sentences
In July 2019, the Company’s board of directors and stockholders approved and adopted the 2019 Employee Stock Purchase Plan (“ESPP”).
−Removed: The number of shares of common stock available for issuance under the ESPP will be automatically increased on the first day of each calendar year during the first ten years of the term of the ESPP, beginning with January 1, 2020 and ending with January 1, 2029, by an amount equal to the lesser of (i) 1 % of the outstanding number of shares of common stock on December 31st of the preceding calendar year, (ii) 1,500,000 shares of common stock or (iii) such lesser amount as determined by the Company’s board of directors.
−Removed: The ESPP became effective on July 17, 2019 and generally provides for six-month consecutive offering periods beginning on May 11th and November 11th of each year.
During the year ended December 31, 2025, 106,977 shares were issued under the ESPP.
As of December 31, 2025, the Company had 1,759,019 shares available for future issuance under the ESPP.
−Removed: The stock-based compensation related to the ESPP for the years ended December 31, 2024, 2023 and 2022 was $ 1.0 million, $ 0.9 million and $ 0.7 million, respectively.
−Removed: Restricted Common Stock
−Removed: In November 2018, in connection with the issuance of Series A Preferred Stock, the Company’s founders agreed to modify their outstanding shares of common stock to include vesting provisions that require continued service to the Company in order to vest in those shares.
−Removed: As such, the 562,500 modified shares of common stock became compensatory upon such modification.
−Removed: During the year ended December 31, 2022, 122,464 shares vested.
−Removed: All restricted common stock was fully vested as of December 31, 2022.
Stock-Based Compensation Expense
23 unchanged sentences
Federal — — —
−Removed: State — — ( 1,078 )
−Removed: — — ( 6,581 )
Provision for (benefit from) income taxes $ 1,936 $ 1,030 $ 991
−Removed: The Company acquired Satiogen in a tax-free reorganization, whereby the Company did not receive a step-up in tax basis of the acquired intangible assets, resulting in a deferred tax liability of $ 6.6 million.
−Removed: The deferred tax liability provided an additional source of taxable income to support the realization of the pre-existing deferred tax assets.
−Removed: As a result, a portion of the Company’s valuation allowance was released and a $ 6.6 million tax benefit was recorded for the year ended December 31, 2022.
−Removed: A reconciliation of the federal statutory income tax rate to the Company’s effective income tax rate is as follows:
+Added: Beginning in 2025 annual reporting, the Company adopted ASU 2023-09 prospectively.
+Added: See Note 2 for additional details on the adoption of ASU 2023-09.
+Added: A reconciliation of the U.S.
+Added: federal statutory income tax rate to the
+Added: effective tax rate pursuant to the disclosure requirements of ASU 2023-09 for the year ended December 31, 2025 is as follows (in thousands, except percentages):
Year Ended December 31, 2025
+Added: Loss before provision of income taxes $ ( 21,427 )
+Added: Provision for income taxes at U.S.
+Added: Federal statutory rate ( 4,500 ) 21.00 %
+Added: State income taxes, net of Federal effect (1)
641 ( 2.99 ) %
+Added: Foreign tax effects
+Added: Foreign rate differential ( 285 ) 1.33 %
+Added: Cantonal tax impacts 106 ( 0.49 ) %
+Added: Other ( 42 ) 0.20 %
+Added: Other foreign jurisdictions 695 ( 3.24 ) %
+Added: Effects of cross-border tax laws
+Added: Subpart F 527 ( 2.46 ) %
+Added: Change in valuation allowance 11,778 ( 54.97 ) %
+Added: Nontaxable or nondeductible items
+Added: Stock-based compensation ( 5,167 ) 24.11 %
+Added: Executive compensation 4,189 ( 19.55 ) %
+Added: Intercompany sale 2,943 ( 13.74 ) %
+Added: Transaction costs 440 ( 2.05 ) %
+Added: Other 83 ( 0.39 ) %
+Added: Tax credits ( 12,629 ) 58.94 %
+Added: Changes in unrecognized tax benefits 3,157 ( 14.74 ) %
+Added: Total tax provision $ 1,936 ( 9.04 ) %
+Added: __________________
+Added: (1) State taxes in Florida, Texas and Michigan made up majority (greater than 50%) of the tax effect in this category.
+Added: A reconciliation of the federal statutory income tax rate to the Company’s effective income tax rate for the years ended December 31, 2024 and 2023 is as follows:
+Added: Year Ended December 31,
Federal statutory income tax rate 21.00 % 21.00 %
7 unchanged sentences
Change in valuation allowance ( 29.54 ) ( 24.69 )
−Removed: Total tax (provision) benefit ( 1.18 ) % ( 0.61 ) % 4.51 %
+Added: Total tax provision ( 1.18 ) % ( 0.61 ) %
+Added: The amounts of income taxes paid, net of refunds received by the Company are as follows (in thousands):
+Added: Year Ended December 31, 2025
+Added: State and Local
+Added: Netherlands 256
+Added: Switzerland 142
+Added: Income taxes paid, net of amounts refunded $ 2,157
The significant components of the Company’s deferred taxes are as follows (in thousands):
33 unchanged sentences
Balance at beginning of year $ 12,965 $ 10,454
−Removed: Increase related to prior year tax positions 337 —
+Added: (Decreases)/increases related to prior year tax positions ( 110 ) 337
Increases related to current year tax positions 4,269 2,174
1 unchanged sentence
The Company has considered the amounts and probabilities of the outcomes that can be realized upon ultimate settlement with the tax authorities and determined unrecognized tax benefits primarily related to credits should be established as noted in the summary roll-forward above.
−Removed: The Company does not expect that its uncertain tax positions will materially change in the next twelve months.
The Company’s effective income tax rate would not be impacted if the unrecognized tax benefits were recognized in 2025 and 2024, as the Company is in a full valuation allowance position.
34 unchanged sentences
As a result, assessing contingencies is highly subjective and requires judgment about future events.
−Removed: 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.
+Added: During the periods presented, the Company has not recorded any accruals for loss contingencies.
+Added: On December 19, 2025, the Company, along with Satiogen Pharmaceuticals, Inc.
+Added: and Shire Human Genetic Therapies, Inc.
+Added: as co-plaintiffs, filed four complaints against Sandoz Inc.
+Added: Annora Pharma Private Limited, Hetero Labs Limited, and Hetero USA Inc.
+Added: (together, “Hetero”);
+Added: Zenara Pharma Private Limited and Biophore India Pharmaceuticals Private Limited (together, “Biophore”);
+Added: and Zydus Lifesciences Global FZE, Zydus Lifesciences Limited, and Zydus Pharmaceuticals (USA) Inc.
+Added: (together, “Zydus”) (all collectively, “Defendants”) in the U.S.
+Added: District Court for the District of Delaware (the “LIVMARLI Patent Litigations”), alleging infringement of certain Orange Book listed patents covering LIVMARLI (the “LIVMARLI Patents”).
+Added: 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.
+Added: 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.
+Added: 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.
+Added: 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.
+Added: 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.
+Added: Trial in the LIVMARLI Patent Litigations
+Added: has not yet been scheduled.
+Added: The Company cannot make any predictions about the final outcome of these matters or the timing thereof.
+Added: Subsequent Events
+Added: On January 23, 2026, the Company completed the acquisition of Bluejay.
+Added: As consideration for the transaction, the Company paid to the holders of Bluejay’s securities an aggregate amount of $ 224.2 million in cash, net of cash acquired in the transaction, and 4,673,597 shares of Company common stock, subject in certain cases to deduction to satisfy applicable taxes, and will pay up to an aggregate amount of $ 25.8 million in cash and up to 522,375 shares of Company common stock, subject to certain conditions and deduction to satisfy applicable taxes.
+Added: Additionally, the Company is obligated to pay up to an aggregate amount of $ 200.0 million upon the achievement of certain commercial milestones.
+Added: Entities affiliated with Frazier Life Sciences, which are associated with a member of the Company’s board of directors, were Bluejay security holders.
+Added: Immediately following the completion of the acquisition, the Company completed the private placement of 3,385,149 shares of the Company’s common stock (“PIPE Shares”) and pre-funded warrants (the “Pre-Funded Warrants”) to purchase 536,412 shares of Company common stock (the “Warrant Shares”) for an aggregate gross proceeds of approximately $ 268.5 million.
+Added: 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) .
Changes in and Disagreements With Accountants on Accounting and Financial Disclosure.
Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.