Volixibat

Portfolio receipts : • Achieved over $50 million of cash receipts, including $33.6 million in royalties and $16.9 million milestones, in full year 2025 • Total receipts increased 9% with royalties up 68% versus full year 2024 Business development : Added 22 assets to portfolio, including five programs in Phase 2 or Phase 3 development Stock buyback program : Repurchased and retired 648,048 shares for an aggregate of $16.0 million Company acquisitions : Completed seven acquisitions, accumulating $11.7 million of non-dilutive capital 1 , economic interests of approximately 25% in up to $1.1 billion of milestones and low to mid-single digit royalties from eight partnered programs Key 2026 pipeline events : • Phase 2b data from volixibat in PSC in Q2 and Phase 3 data from ersodetug in tumor HI in 2H • Potential for EMA decisions on OJEMDA™ and MIPLYFFA™ marketing authorization applications • Regulatory updates related to ersodetug in congenital HI and seralutinib in PAH Webcast at 8:00 am Eastern Time today EMERYVILLE, Calif., March 18, 2026 (GLOBE NEWSWIRE) -- XOMA Royalty Corporation (NASDAQ: XOMA), the biotech royalty aggregator, reported its 2025 fourth quarter and full year financial results and highlighted recent actions that have the potential to deliver additional shareholder value. “We continue to search for innovative ways to drive enhanced optionality in the XOMA portfolio, with the addition of 22 assets and two platform technologies over the past year,” stated Owen Hughes, Chief Executive Officer of XOMA Royalty. “With multiple commercial assets delivering growing royalty receipts, we achieved positive cash flow from operations and were able to return $16 million of capital through a share buyback in 2025. Looking ahead, with 14 programs in registrational studies, we anticipate a number of catalysts over the ensuing years, including several regulatory updates and late-stage clinical readouts in 2026, which, if positive, will further diversify our commercial royalty streams and drive growing free cash flow in 2027 and beyond.” Portfolio Updates Day One OJEMDA New Drug Application filing in Japan triggered $2 million milestone in 4Q25 OJEMDA FY 2026 revenue guidance of $225 – $250 million 2 In February 2026, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a positive opinion recommending the conditional marketing authorization of OJEMDA 3 In March 2026, Day One and Servier announced that they have entered into a definitive agreement for Servier to acquire Day One for $21.50 per share in cash, representing a total equity value of approximately $2.5 billion 4 Zevra Therapeutics A Marketing Authorization Application for the evaluation of arimoclomol (MIPLYFFA) for the treatment of NPC is under review by the EMA 5 Rezolute In December 2025, Rezolute announced that the Phase 3 clinical study of ersodetug for the treatment of congenital hyperinsulinism (“HI”) demonstrated reductions from baseline in hypoglycemia events by self-monitored blood glucose at both ersodetug dose levels, but the reductions were not statistically significant compared to placebo, due to a pronounced study effect 6 Rezolute will meet with FDA under its Breakthrough Therapy Designation in the first quarter of 2026 to determine next steps for the program 6 Rezolute anticipates topline results of upLIFT, a Phase 3, single-arm, open-label study in participants with tumor HI, in the second half of 2026 6 Gossamer Bio & Chiesi In February 2026, Gossamer Bio announced topline results from the Phase 3 PROSERA clinical trial evaluating seralutinib for the treatment of PAH 7 Seralutinib demonstrated a placebo-adjusted improvement in Six-Minute Walk Distance (6MWD) of +13.3 meters at Week 24 (p = 0.0320), missing the prespecified alpha threshold of 0.025 7 Gossamer plans to meet with the U.S. FDA to discuss the path forward 7 Volixibat Volixibat VISTAS study in primary sclerosing cholangitis (PSC) topline data expected in Q2 2026 8 Volixibat VANTAGE study in primary biliary cholangitis (PBC) expected to complete enrollment in H2 2026 8 Business Development Activity Takeda Strategic Royalty Share Transaction In December 2025, XOMA amended its collaboration with Takeda XOMA will receive low to mid-single-digit royalties and up to $852.6 million in potential milestones across nine development-stage assets, including osavampator, which is being evaluated in Phase 3 studies for major depressive disorder; volixibat, which is being evaluated in PSC and PBC; OHB-607, which Oak Hill Bio Ltd and its partner are developing for the prevention of bronchopulmonary dysplasia in extremely premature infants; REC-4881, which is in Phase 2 development for familial adenomatous polyposis; and five early-stage Oak Hill Bio assets Prior to amending the collaboration, XOMA held a mid-single digit royalty and $16.25 million in potential milestones associated with mezagitamab Following the transaction, XOMA will retain a low single-digit royalty entitlement on mezagitamab and up to $13.0 million in milestones Company Acquisitions Completed or served as the structuring agent in the acquisition of seven companies since the beginning of 2025 Accumulated non-dilutive capital of $11.7 million, net of transaction expenses Obtained economic interests of approximately 25% in up to $1.1 billion of potential milestone payments and low to mid-single-digit royalties from eight partnered assets Eligible for 25-70% of proceeds related to any future out license or sale of legacy assets or platform technology from these companies, including the ctLNP delivery platform from Generation Bio Fourth Quarter and Full-Year 2025 Financial Results In the fourth quarter of 2025, XOMA Royalty received $3.2 million in cash receipts from royalties and commercial payments and $3.3 million in milestone payments and paid $1.4 million in dividends on the XOMA Royalty Perpetual Preferred stocks. For the full year of 2025, XOMA Royalty received $50.5 million in cash receipts, including $33.6 million in royalties and commercial payments and $16.9 million in milestone payments and fees. During 2025, XOMA Royalty deployed $25.0 million to acquire additional assets for its royalty and milestone portfolio, repurchased 648,048 shares of its common stock for a cost of $16.0 million, and paid $5.5 million in dividends on the XOMA Royalty Perpetual Preferred stocks. Income and Revenue : Income and revenues for the three months ended December 31, 2025 and 2024, were $13.8 million and $8.7 million, respectively. Income and revenues for the years ended December 31, 2025 and 2024, were $52.1 million and $28.5 million, respectively. The increase in both periods was primarily driven by increased income related to VABYSMO® (faricimab-svoa) and OJEMDA™ (tovorafenib) and milestone payments received from Rezolute and Takeda. General and Administrative (G&A) Expenses : G&A expenses for the three months ended December 31, 2025 and 2024, were $10.4 million and $7.0 million, respectively. G&A expenses for the years ended December 31, 2025 and 2024, were $36.1 million and $34.5 million, respectively. The increase of $1.6 million in 2025 was primarily due to an increase in business development and deal-related costs of $3.7 million and an increase in lease costs of $1.0 million primarily related to the HilleVax acquisition partially offset by $3.6 million in costs related to exit packages for Kinnate senior leadership in 2024. G&A expenses for the year ended December 31, 2025, also include an increase of approximately $1.1 million associated with ongoing litigation initiated by XOMA Royalty against Janssen Biotech, Inc., asserting claims for breach of contract and unjust enrichment arising from Janssen's unauthorized use of XOMA’s intellectual property in the commercialization of TREMFYA (guselkumab). XOMA Royalty expects to continue to incur legal fees and other professional service costs associated with pursuing this litigation. Litigation is inherently uncertain, and there can be no assurance regarding the outcome of the matter or the timing or amount of any potential recovery. XOMA Royalty’s G&A expenses for the three months ended December 31, 2025 and 2024, included non-cash stock-based compensation expenses of $3.9 million and $2.2 million, respectively, and $9.3 million and $10.3 million for the full years of 2025 and 2024, respectively. Interest Expense : Interest expense for the three months ended December 31, 2025 and 2024, was $3.0 million and $3.4 million, respectively. Interest expense for the twelve months ended December 31, 2025 and 2024, were $13.0 million and $13.8 million, respectively. Interest expense relates to the Blue Owl Loan established in December 2023. Net Income (Loss) : XOMA Royalty reported net income of $6.1 million and $31.7 million for the three months and year ended December 31, 2025, as compared to net losses of $4.0 million and $13.8 million in the corresponding periods of 2024. Cash Position : On December 31, 2025, XOMA Royalty had cash and cash equivalents of $133.7 million, including $50.8 million in restricted cash. The restricted cash balance included $42.3 million related to the assumed HilleVax lease and $2.2 million related to the Blue Owl Loan. Cash and cash equivalents of $106.4 million as of December 31, 2024, included $4.8 million in restricted cash related to the Blue Owl Loan. Webcast The Company will host a webcast on March 18, 2026, at 8:00 am Eastern Time to discuss the results and provide a business update. The webcast will be accessible on the “News & Events” page in the Investors section of XOMA Royalty’s website ( ). A replay of the webcast will be available for 30 days following the live event. About XOMA Royalty Corporation XOMA Royalty is a biotechnology royalty aggregator playing a distinctive role in helping biotech companies achieve their goal of improving human health. XOMA Royalty acquires the potential future economics associated with pre-commercial and commercial therapeutic candidates that have been licensed to pharmaceutical or biotechnology companies. When XOMA Royalty acquires the future economics, the seller receives non-dilutive, non-recourse funding they can use to advance their internal drug candidate(s) or for general corporate purposes. The Company has an extensive and growing portfolio of assets (asset defined as the right to receive potential future economics associated with the advancement of an underlying therapeutic candidate). For more information about the Company and its portfolio, please visit or follow XOMA Royalty Corporation on LinkedIn . Forward-Looking Statements/Explanatory Notes Certain statements contained in this press release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, including statements regarding the timing and amount of potential commercial payments to XOMA Royalty, expectations around future royalty cash flows covering XOMA Royalty’s core operating expenses (the inflection point) and other developments related to VABYSMO® (faricimab-svoa), OJEMDA™ (tovorafenib), MIPLYFFA™ (arimoclomol), XACIATO™ (clindamycin phosphate) vaginal gel 2%, IXINITY® [coagulation factor IX (recombinant)], DSUVIA® (sufentanil sublingual tablet), and DARE to PLAY™ Sildenafil Cream and Sildenafil Cream, 3.6%; the potential occurrences and timing of the events listed under “ Key 2026 Pipeline Events”; expectations regarding the inflection point in XOMA Royalty’s business model of breakeven operating cash flows; the anticipated timings of regulatory filings and approvals related to assets in XOMA Royalty’s portfolio; and the potential of XOMA Royalty’s portfolio of partnered programs and licensed technologies generating substantial milestone and royalty proceeds over time. In some cases, you can identify such forward-looking statements by terminology such as “anticipate,” “intend,” “believe,” “estimate,” “plan,” “seek,” “project,” “expect,” “may,” “will”, “would,” “could” or “should,” the negative of these terms or similar expressions. These forward-looking statements are not a guarantee of XOMA Royalty’s performance, and you should not place undue reliance on such statements. These statements are based on assumptions that may not prove accurate, and actual results could differ materially from those anticipated due to certain risks inherent in the biotechnology industry, including those related to the fact that our product candidates subject to out-license agreements are still being developed, and our licensees may require substantial funds to continue development which may not be available; we do not know whether there will be, or will continue to be, a viable market for the products in which we have an ownership or royalty interest; and if the therapeutic product candidates to which we have a royalty interest do not receive regulatory approval, our third-party licensees will not be able to market them. Other potential risks to XOMA Royalty meeting these expectations are described in more detail in XOMA Royalty's most recent filing on Form 10-K and in other filings with the Securities and Exchange Commission. Consider such risks carefully when considering XOMA Royalty's prospects. Any forward-looking statement in this press release represents XOMA Royalty's beliefs and assumptions only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. XOMA Royalty disclaims any obligation to update any forward-looking statement, except as required by applicable law. EXPLANATORY NOTE: Any references to “portfolio” in this press release refer strictly to milestone and/or royalty rights associated with a basket of drug products in development. Any references to “assets” in this press release refer strictly to milestone and/or royalty rights associated with individual drug products in development. As of the date of this press release, the commercial assets in XOMA Royalty’s milestone and royalty portfolio are VABYSMO® (faricimab-svoa), OJEMDA™ (tovorafenib), MIPLYFFA™ (arimoclomol), XACIATO™ (clindamycin phosphate) vaginal gel 2%, IXINITY® [coagulation factor IX (recombinant)], DARE to PLAY™ (Sildenafil Cream), and DSUVIA® (sufentanil sublingual tablet). All other assets in the milestone and royalty portfolio are investigational compounds. Efficacy and safety have not been established. There is no guarantee that any of the investigational compounds will become commercially available. XOMA ROYALTY CORPORATION CONSOLIDATED STATEMENTS OF OPERATIONS (in thousands, except per share amounts) Three Months Ended December 31, Year Ended December 31, 2025 2024 2025 2024 Income and revenues: Income from purchased receivables under the EIR method $ 7,706 $ 5,081 $ 26,745 $ 15,066 Income from purchased receivables under the cost recovery method 4,619 1,291 13,744 3,201 Revenue from contracts with customers 1,100 600 10,350 6,650 Revenue recognized under units-of-revenue method 332 1,742 1,310 3,570 Total income and revenues 13,757 8,714 52,149 28,487 Operating expenses: Research and development 281 864 1,712 2,875 General and administrative 10,410 6,993 36,092 34,478 Credit losses on purchased receivables — 7,904 — 30,904 Amortization of intangible assets 884 206 2,961 206 Total operating expenses 11,575 15,967 40,765 68,463 Income (loss) from operations 2,182 (7,253 ) 11,384 (39,976 ) Other income (expense), net: Gains on acquisitions 3,220 — 21,224 19,316 Change in fair value of embedded derivative related to RPA — — — 8,100 Interest expense (3,027 ) (3,394 ) (13,031 ) (13,840 ) Other income, net 3,782 1,021 12,238 6,921 Net income (loss) before tax 6,157 (9,626 ) 31,815 (19,479 ) Income tax (expense) benefit (54 ) 5,658 (103 ) 5,658 Net income (loss) $ 6,103 $ (3,968 ) $ 31,712 $ (13,821 ) Net income (loss) available to (attributable to) common stockholders, basic $ 3,319 $ (5,336 ) $ 18,516 $ (19,293 ) Basic net income (loss) per share available to (attributable to) common stockholders $ 0.27 $ (0.45 ) $ 1.53 $ (1.65 ) Weighted average shares used in computing basic net income (loss) per share available to (attributable to) common stockholders 12,208 11,868 12,081 11,701 Net income (loss) available to (attributable to) common stockholders, diluted $ 4,679 $ (5,336 ) $ 26,184 $ (19,293 ) Diluted net income (loss) per share available to (attributable to) common stockholders $ 0.26 $ (0.45 ) $ 1.46 $ (1.65 ) Weighted average shares used in computing diluted net income (loss) per share available to (attributable to) common stockholders 18,095 11,868 17,982 11,701 XOMA ROYALTY CORPORATION CONSOLIDATED BALANCE SHEETS (in thousands, except share and per share amounts) December 31, December 31, 2025 2024 ASSETS Current assets: Cash and cash equivalents $ 82,908 $ 101,654 Short-term restricted cash 5,441 1,330 Investment in equity securities 382 3,529 Trade and other receivables, net 4,896 1,839 Short-term royalty and commercial payment receivables under the EIR method 22,780 14,763 Short-term royalty and commercial payment receivables under the cost recovery method - 413 Prepaid expenses and other current assets 852 2,076 Total current assets 117,259 125,604 Long-term restricted cash 45,361 3,432 Property and equipment, net 21 32 Operating lease right-of-use assets 256 319 Long-term royalty and commercial payment receivables under the EIR method 4,433 4,970 Long-term royalty and commercial payment receivables under the cost recovery method 55,888 55,936 Exarafenib milestone asset 3,600 3,214 Investment in warrants 697 — Intangible assets, net 44,756 25,909 Other assets - long term 427 1,861 Total assets $ 272,698 $ 221,277 LIABILITIES AND STOCKHOLDERS’ EQUITY Current liabilities: Accounts payable $ 2,208 $ 1,053 Accrued and other liabilities 9,885 5,752 Contingent consideration under RPAs, AAAs, and CPPAs - 3,000 Operating lease liabilities 2,464 446 Unearned revenue recognized under units-of-revenue method 1,268 1,361 Preferred stock dividend accrual 1,424 1,368 Current portion of long-term debt 12,526 11,394 Contingent value rights liabilities - current portion 5,045 - Total current liabilities 34,820 24,374 Unearned revenue recognized under units-of-revenue method – long-term 3,193 4,410 Exarafenib milestone contingent consideration 3,600 3,214 Long-term operating lease liabilities 20,114 483 Long-term debt 96,451 106,875 Contingent value rights liabilities - long-term 10,457 - Deferred tax liability 103 - Total liabilities 168,738 139,356 Convertible preferred stock, $0.05 par value, 5,003 shares authorized, issued and outstanding as of December 31, 2025 and December 31, 2024 20,019 20,019 Stockholders’ equity: 8.625% Series A cumulative, perpetual preferred stock, $0.05 par value, 984,000 shares authorized, issued and outstanding as of December 31, 2025 and December 31, 2024 49 49 8.375% Series B cumulative, perpetual preferred stock, $0.05 par value, 3,600 shares authorized, 1,760.5 and 1,600 shares issued and outstanding as of December 31, 2025 and December 31, 2024, respectively — — Common stock, $0.0075 par value, 277,333,332 shares authorized, 11,858,955 and 11,952,377 shares issued and outstanding as of December 31, 2025 and December 31, 2024, respectively 89 90 Additional paid-in capital 1,305,200 1,298,747 Accumulated other comprehensive income 53 73 Accumulated deficit (1,221,450 ) (1,237,057 ) Total stockholders’ equity 83,941 61,902 Total liabilities, convertible preferred stock and stockholders’ equity $ 272,698 $ 221,277 XOMA ROYALTY CORPORATION CONSOLIDATED STATEMENTS OF CASH FLOWS (in thousands) Year Ended December 31, 2025 2024 Cash flows from operating activities: Net income (loss) $ 31,712 $ (13,821 ) Adjustments to reconcile net loss to net cash used in operating activities: Income from purchased receivables under the EIR method (5,925 ) (15,066 ) Stock-based compensation expense 9,273 10,312 Gains on acquisitions (21,224 ) (19,316 ) Credit losses on purchased receivables — 30,904 Gain on sale of equity securities (3,663 ) — Income tax expense (benefit) 103 (5,658 ) Common stock contribution to 401(k) 141 118 Amortization of intangible assets 2,961 206 Depreciation 11 10 Accretion of long-term debt discount and debt issuance costs 1,385 1,350 Non-cash lease expense 64 60 Change in fair value of equity securities (90 ) (131 ) Change in fair value of available-for-sale debt securities classified as cash equivalents (20 ) 73 Change in fair value of derivatives (93 ) — CVR liability working capital adjustment (394 ) — Changes in assets and liabilities: Trade and other receivables, net (2,426 ) (835 ) Prepaid expenses and other assets 3,839 302 Accounts payable and accrued liabilities (10,597 ) 1,598 Operating lease liabilities (876 ) (284 ) Unearned revenue recognized under units-of-revenue method (1,310 ) (3,570 ) Net cash provided by (used in) operating activities 2,871 (13,748 ) Cash flows from investing activities: Net cash acquired in Kinnate acquisition — 18,926 Net cash acquired in Turnstone acquisition 3,850 — Net cash and restricted cash acquired in HilleVax acquisition 46,384 — Net cash, cash equivalents, and restricted cash acquired in LAVA acquisition 15,263 — Net cash and cash equivalents acquired in Mural acquisition 4,464 — Payments of consideration under RPAs, AAAs, and CPPAs (8,000 ) (53,000 ) Receipts under RPAs, AAAs, and CPPAs 3,300 29,248 Net payment for IP acquired under the Pulmokine Acquisition — (20,176 ) Payment for BioInvent contract-based intangible asset (20,725 ) — Payment of contingent consideration related to Kinnate IP asset (550 ) — Purchase of property and equipment — (20 ) Purchase of equity securities (99 ) (3,237 ) Sale of equity securities 6,999 — Payment to issue short-term loan to Xeno (5,877 ) — Receipt from short-term loan repayment by Xeno 5,877 — Net cash provided by (used in) investing activities 50,886 (28,259 ) Cash flows from financing activities: Proceeds from issuance of common stock 323 — Proceeds from issuance of preferred stock 4,019 — Payments of preferred and common stock issuance and financing costs (672 ) — Principal payments – debt (10,598 ) (6,902 ) Debt issuance costs and loan fees paid in connection with long-term debt (80 ) (740 ) Payment of preferred stock dividends (5,472 ) (5,472 ) Repurchases of common stock (16,043 ) (13 ) Proceeds from exercise of options and other share-based compensation 5,046 5,214 Taxes paid related to net share settlement of equity awards (2,986 ) (3,214 ) Net cash used in financing activities (26,463 ) (11,127 ) Net increase (decrease) in cash, cash equivalents, and restricted cash 27,294 (53,134 ) Cash, cash equivalents, and restricted cash as of the beginning of the period 106,416 159,550 Cash, cash equivalents, and restricted cash as of the end of the period $ 133,710 $ 106,416 Supplemental Cash Flow Information: Cash paid for interest $ 11,906 $ 9,985 Cash paid for taxes $ 277 $ — Non-cash investing and financing activities: Accrual of contingent value rights liability in the Turnstone acquisition $ 1,110 $ — Accrual of contingent value rights liability in the HilleVax acquisition $ 5,673 $ — Accrual of contingent value rights liability in the LAVA acquisition $ 9,114 $ — Right-of-use assets obtained in exchange for operating lease liabilities in the HilleVax acquisition $ 22,525 $ — Relative fair value basis reduction of right-of-use assets in the HilleVax acquisition $ (22,525 ) $ — Transaction costs in connection with Mural acquisition included in accrued expenses $ 320 $ — Excise tax accrual due to stock repurchases $ 68 $ — Reclassification of equity classified awards to liabilities $ (739 ) $ — Reclassification of deferred issuance cost to equity $ 578 $ — Preferred stock dividend accrual $ 1,424 $ 1,368 Estimated fair value of the Exarafenib milestone asset $ — $ 2,922 Estimated fair value of the Exarafenib milestone contingent consideration $ — $ (2,922 ) Right-of-use assets obtained in exchange for operating lease liabilities in the Kinnate acquisition $ — $ 824 Relative fair value basis reduction of rights-of-use assets in the Kinnate acquisition $ — $ (824 ) Accrual of contingent consideration under the Affitech CPPA $ — $ 3,000 Accrual of contingent consideration under the LadRx AAA $ — $ 1,000 Investor contact: Media contact: Juliane Snowden Kathy Vincent XOMA Royalty Corporation KV Consulting & Management +1-646-438-9754 kathy@kathyvincent.com juliane.snowden@xoma.com 1 This amount includes structuring agent fees associated with Repare Therapeutics and ESSA Pharmaceuticals. 2 3 4 5 6 7 8

- Study designed to support potential label expansion into additional settings of cholestatic pruritus - Topline data expected in Q4 2026 FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today announced completion of enrollment in EXPAND, a Phase 3 randomized, double-blind, placebo-controlled study evaluating LIVMARLI ® (maralixibat) for the treatment of cholestatic pruritus in patients aged 6 months or older with rare cholestatic liver diseases, including biliary atresia. Cholestatic liver diseases are characterized by impaired bile flow, resulting in elevated serum bile acids, severe pruritus, and significantly reduced quality of life. While LIVMARLI is approved in the United States and European Union for the treatment of cholestatic pruritus associated with Alagille syndrome and progressive familial intrahepatic cholestasis (PFIC), patients with other rare cholestatic liver diseases continue to experience debilitating itch and treatment options remain limited. “Completing enrollment in EXPAND marks an important milestone in our efforts to broaden the reach of LIVMARLI to additional patients living with cholestatic pruritus,” said Joanne Quan, M.D., Chief Medical Officer at Mirum Pharmaceuticals. “Itch due to cholestasis profoundly affects the daily lives of patients and their families. We aim to address the unmet medical needs of these patients by leveraging our established IBAT mechanism.” The EXPAND study enrolled patients with cholestatic pruritus associated with a range of rare cholestatic liver diseases. In the 20-week placebo-controlled portion, patients are randomized to receive maralixibat 285 micrograms per kilogram (µg/kg) twice daily or placebo. The primary endpoint is change in pruritus severity from baseline to Week 20. Secondary endpoints include changes in serum bile acids and other markers of cholestatic liver disease. “For the patients and families living with rare liver diseases, cholestatic pruritus isn’t just an itch—it can be relentless, exhausting, and life-altering,” said Dr. Mercedes Martinez, M.D., Professor of Pediatrics and Medicine at CUMC, Columbia University Vagelos College of Physicians and Surgeons. “Yet for many of these conditions, there are still no approved treatment options for pruritus. The EXPAND study marks an important milestone in exploring whether a targeted therapy like LIVMARLI can offer meaningful relief to patients who urgently need better treatment options.” “Families affected by biliary atresia and other rare cholestatic conditions understand the relentless burden of itch,” said Jen Lau, MPH, Co-Founder and Executive Director of BARE, Inc. (Biliary Atresia Research and Education). “The completion of enrollment in EXPAND brings hope that more patients may soon have access to a much-needed treatment option.” Topline data from the EXPAND study are expected in the fourth quarter of 2026. About the EXPAND Phase 3 Study EXPAND is a Phase 3, randomized, double-blind, placebo-controlled study evaluating the safety and efficacy of LIVMARLI ® (maralixibat) in children aged 6 months and older and adults with cholestatic pruritus associated with rare cholestatic liver diseases. These diseases include biliary atresia and other rare conditions for which LIVMARLI is not currently approved. The primary endpoint is change in pruritus severity from baseline to Week 20. Secondary endpoints include changes in serum bile acids and other markers of cholestatic liver disease. About LIVMARLI ® (maralixibat) oral solution and LIVMARLI ® (maralixibat) tablets LIVMARLI® (maralixibat) is an orally administered, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for two pediatric cholestatic liver diseases. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. three months of age and older and in Europe for patients two months of age and older. It is also approved in the U.S. for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) 12 months of age and older and in Europe for the treatment of PFIC in patients three months of age and older. For more information for U.S. residents, please visit LIVMARLI.com . LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for the treatment of ALGS and PFIC. LIVMARLI is currently being evaluated in the Phase 3 EXPAND study in additional settings of cholestatic pruritus. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum’s clinical trials section on the company’s website. IMPORTANT SAFETY INFORMATION Limitation of Use: LIVMARLI is not for use in PFIC type 2 patients who have a severe defect in the bile salt export pump (BSEP) protein. LIVMARLI can cause side effects, including: Liver injury. Changes in certain liver tests are common in patients with ALGS and PFIC but can worsen during treatment. These changes may be a sign of liver injury. In PFIC, this can be serious or may lead to liver transplant or death. Your healthcare provider should do blood tests and physical exams before starting and during treatment to check your liver function. Tell your healthcare provider right away if you get any signs or symptoms of liver problems, including nausea or vomiting, skin or the white part of the eye turns yellow, dark or brown urine, pain on the right side of the stomach (abdomen), bloating in your stomach area, loss of appetite or bleeding or bruising more easily than normal. Stomach and intestinal (gastrointestinal) problems . LIVMARLI can cause stomach and intestinal problems, including diarrhea and stomach pain. Your healthcare provider may advise you to monitor for new or worsening stomach problems including stomach pain, diarrhea, blood in your stool or vomiting. Tell your healthcare provider right away if you have any of these symptoms more often or more severely than normal for you. A condition called Fat Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat is common in patients with ALGS and PFIC but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment and may monitor for bone fractures and bleeding which have been reported as common side effects. US Prescribing Information EU SmPC Canadian Product Monograph About Mirum Pharmaceuticals Mirum Pharmaceuticals (NASDAQ: MIRM) is a leading rare disease company with a global footprint of approved products and a broad pipeline of investigational medicines. Purpose-built to bring forward breakthrough medicines for people with overlooked conditions, Mirum combines deep rare disease expertise with strong connections to patient communities. The company’s commercial portfolio includes LIVMARLI ® (maralixibat) for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), CHOLBAM ® (cholic acid) for bile-acid synthesis disorders, and CTEXLI ® (chenodiol) for cerebrotendinous xanthomatosis (CTX). Mirum’s clinical-stage pipeline includes volixibat, an IBAT inhibitor in late-stage development for primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), brelovitug, a fully human monoclonal antibody in late-stage development for chronic hepatitis delta virus (HDV) and MRM-3379, a PDE4D inhibitor being evaluated for Fragile X syndrome (FXS). Mirum’s success is driven by a team dedicated to advancing high impact medicines through strategic development, disciplined execution and purposeful collaboration across the rare disease ecosystem. Learn more at and follow Mirum on Facebook , LinkedIn , Instagram and X . Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, LIVMARLI ® (maralixibat) efficacy in additional patients living with cholestatic pruritus, Mirum’s leadership in rare disease, expectations regarding LIVMARLI ® (maralixibat) ongoing development, including the potential completion of and successful results from the EXPAND trial, the anticipated timing for data from the EXPAND study, and any potential regulatory submission. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “anticipate,” “expected,” “will,” “could,” “would,” “potential,” “continue,” “plans,” “intended,” “believe,” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the development of acquired product candidates, including the failure of any expected synergies to be realized; risk and uncertainties arising from the integration of an acquired company, its employees and its assets with Mirum’s business; risks associated with evaluating companies and assets for acquisition, including that the perceived benefits of the acquisition are not realized; risks and uncertainties with the development of investigational medicines generally, including the failure of future studies to generate the same or similar data as prior studies and the potential that estimated prevalences are materially inaccurate; the risks and uncertainties associated with Mirum’s business in general, the impact of geopolitical and macroeconomic events, and the other risks described in Mirum’s Annual Report for the year ended December 31, 2024, filed with the Securities and Exchange Commission on February 26, 2025, and subsequent filings with the Securities and Exchange Commission, which are available at . All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. Contacts Investor Contact: Andrew McKibben ir@mirumpharma.com Media Contact: Meredith Kiernan Media@mirumpharma.com