\nMirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today reported financial results for the first quarter 2026 and provided a business update.<\/p>\n
\n\u201c2026 is off to an excellent start, driven by strong commercial momentum, the recent VISTAS and AZURE-1 topline results, disciplined clinical execution, and continued expansion of our pipeline,\u201d said Chris Peetz, Chief Executive Officer of Mirum. \u201cWe also announced today the in-license of zilurgisertib, a once-daily oral ALK2 inhibitor for fibrodysplasia ossificans progressiva from Incyte. Zilurgisertib has completed a pivotal study for FOP and its NDA has been accepted, creating the potential for an additional product launch for our rare genetic disease team later this year. We\u2019re grateful to Incyte for all the work they have conducted on this program and look forward to potentially bringing another high impact medicine to patients.\u201d<\/p>\n
Q1 and Recent Highlights<\/b><\/p>\n
Commercial: Raising Full Year Revenue Guidance to $660 to $680 Million<\/b><\/p>\n Regulatory and Pipeline: Milestones on Track with Expansion of Leadership in Rare Disease<\/b><\/p>\n Business Development: Obtained Exclusive Worldwide Rights to Zilurgisertib<\/b><\/p>\n Corporate & Financial: Strong Balance Sheet and Financial Independence<\/b><\/p>\n Business Update Conference Call<\/b><\/p>\n \nMirum will host a conference call today, May 6 at 1:30 p.m. PT\/4:30 p.m. ET, to provide business updates. Join the call using the following details:<\/p>\n Conference Call Details:<\/b><\/p>\n \nUS\/Toll-Free: + 1 833 461 5787 \nYou may also access the call via webcast by visiting the Investors<\/a> section of Mirum\u2019s corporate website. The archived webcast will be available for replay.<\/p>\n About LIVMARLI\u00ae (maralixibat) oral solution<\/b>and LIVMARLI\u00ae (maralixibat) tablets<\/b><\/p>\n \nLIVMARLI\u00ae (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<\/a>.<\/p>\n \nLIVMARLI 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\u2019s clinical trials section<\/a> on the company\u2019s website.<\/p>\n IMPORTANT SAFETY INFORMATION<\/b><\/p>\n \nLimitation 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.<\/p>\n LIVMARLI can cause side effects, including:<\/b><\/p>\n Liver injury. <\/b>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.<\/p>\n Stomach and intestinal (gastrointestinal) problems<\/b>. 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.<\/p>\n \nA condition called Fat Soluble Vitamin (FSV) <\/b>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.<\/p>\n US Prescribing Information About CHOLBAM\u00ae (cholic acid) capsules<\/b><\/p>\n \nThe FDA approved CHOLBAM\u00ae (cholic acid) capsules in March 2015, the first FDA-approved treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects, and for adjunctive treatment of patients with peroxisome biogenesis disorder-Zellweger spectrum disorder. The effectiveness of CHOLBAM has been demonstrated in clinical trials for bile acid synthesis disorders and the adjunctive treatment of peroxisomal disorders. An estimated 200 to 300 patients are current candidates for therapy.<\/p>\n CHOLBAM (cholic acid) Indication<\/b><\/p>\n \nCHOLBAM is a bile acid indicated for<\/p>\n LIMITATIONS OF USE<\/b><\/p>\n \nThe safety and effectiveness of CHOLBAM on extrahepatic manifestations of bile acid synthesis disorders due to single enzyme defects or peroxisomal disorders, including Zellweger spectrum disorders, have not been established.<\/p>\n IMPORTANT SAFETY INFORMATION<\/b><\/p>\n WARNINGS AND PRECAUTIONS \u2013 Exacerbation of liver impairment<\/b><\/p>\n \nMonitor liver function and discontinue CHOLBAM in patients who develop worsening of liver function while on treatment.<\/p>\n \nConcurrent elevations of serum gamma glutamyltransferase (GGT) and alanine aminotransferase (ALT) may indicate CHOLBAM overdose.<\/p>\n \nDiscontinue treatment with CHOLBAM at any time if there are clinical or laboratory indicators of worsening liver function or cholestasis.<\/p>\n ADVERSE REACTIONS<\/b><\/p>\n \nThe most common adverse reactions (\u22651%) are diarrhea, reflux esophagitis, malaise, jaundice, skin lesion, nausea, abdominal pain, intestinal polyp, urinary tract infection, and peripheral neuropathy. Please see full Prescribing Information<\/a> for additional Important Safety Information.<\/p>\n About CTEXLI\u00ae (chenodiol) tablets<\/b><\/p>\n \nCTEXLI\u00ae<\/b> (chenodiol) tablets is FDA-approved for the treatment of adults with cerebrotendinous xanthomatosis (CTX). Chenodiol is another name for chenodeoxycholic acid (CDCA). CDCA is a naturally occurring bile acid that was originally approved for the treatment of people with radiolucent stones in the gallbladder. CTEXLI was evaluated as part of the Phase 3 RESTORE study, the first and only clinical trial for CTX. CTX is a rare progressive disease that can affect the brain, spinal cord, tendons, eyes and arteries.<\/p>\n IMPORTANT SAFETY INFORMATION<\/b><\/p>\n \nCTEXLI can cause side effects, including:<\/p>\n Liver Injury<\/b>: You will need to undergo laboratory testing before starting and while taking CTEXLI to check your liver function. Changes in certain liver tests may occur during treatment and may be a sign of liver injury. This can be serious. Stop taking CTEXLI immediately and tell your healthcare provider right away if you get any signs or symptoms of liver problems, including, stomach (abdomen) pain, bruising, dark-colored urine, feeling tired (fatigue), bleeding, yellowing of the skin and eyes, nausea, and itching.<\/p>\n Most Common Side Effects<\/b>: Diarrhea, headache, stomach pain, constipation, high blood pressure, muscular weakness, and upper respiratory tract infection.<\/p>\n \nTell your healthcare provider about all the medications that you take, as CTEXLI may interact with other medicines.<\/p>\n US Prescribing Information<\/a><\/p>\n About Volixibat<\/b><\/p>\n \nVolixibat is an investigational oral, minimally absorbed agent designed to selectively inhibit the ileal bile acid transporter (IBAT). Volixibat may offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids, through inhibition of IBAT, thereby reducing bile acids systemically and in the liver. Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (PSC) (VISTAS study) and primary biliary cholangitis (PBC) (VANTAGE study). In 2024, the Phase 2b VANTAGE study of volixibat in PBC met the primary endpoint. No new safety signals were observed, and the most common adverse event was diarrhea with all cases mild to moderate. Volixibat has been granted FDA Breakthrough Therapy designation for the treatment of PBC.<\/p>\n About Brelovitug<\/b><\/p>\n \nBrelovitug is an investigational, highly potent, pan-genotypic, fully human immunoglobulin G1 (IgG1) monoclonal antibody (mAb) that targets the surface antigen (anti-HBsAg) on both the chronic hepatitis delta virus (HDV) and the hepatitis B virus (HBV). Brelovitug is designed to neutralize and remove hepatitis B and hepatitis D virions and deplete HBsAg-containing subviral particles. Brelovitug has FDA Breakthrough Therapy designation for the treatment of chronic HDV infection and PRIME and Orphan designations from the European Medicines Agency.<\/p>\n \nIn the previously reported 778-001 Phase 2 study, brelovitug demonstrated strong antiviral activity in HDV, achieving 100% virologic response, along with improvements in liver enzyme levels and a favorable safety profile at Week 48. Brelovitug is currently being evaluated in the global Phase 3 AZURE clinical program. Mirum owns worldwide rights to brelovitug.<\/p>\n About Zilurgisertib<\/b><\/p>\n \nZilurgisertib is an investigational, oral, small molecule, activin receptor-like kinase 2 (ALK2) inhibitor in development for the treatment of Fibrodysplasia Ossificans Progressiva (FOP). Zilurgisertib is designed to inhibit the ALK2 receptor which is abnormally active in most patients with FOP and leads to bone formation in soft tissues, a process known as heterotopic ossification (HO). FOP is an ultra-rare genetic disease that affects approximately 300 patients in the U.S. and 900 worldwide, with diagnosis typically occurring in early childhood. Zilurgisertib was evaluated in the PROGRESS pivotal Phase 2 study, which formed the basis of a new drug application (NDA). The FDA has accepted the NDA for zilurgisertib in FOP under Priority Review with a Prescription Drug User Fee Act (PDUFA) date of September 26, 2026.<\/p>\n \nMirum licensed zilurgisertib from Incyte for development and commercialization globally.<\/p>\n About MRM-3379<\/b><\/p>\n \nMRM-3379 is an in-licensed investigational oral therapy being evaluated for the treatment of Fragile X syndrome (FXS). It is a selective phosphodiesterase-4D (PDE4D) inhibitor designed to enhance cAMP signaling. MRM-3379 may offer a novel approach to improving cognition, language, and daily function in individuals with FXS. MRM-3379 has been granted FDA Fast Track designation for the treatment of FXS.<\/p>\n \nThe BLOOM Phase 2 clinical study of MRM-3379 is currently underway in FXS. Males ages 16 to 45 will be randomly assigned to receive one of three dose levels of MRM-3379 or placebo for 12 weeks. An open-label cohort of boys ages 13 to 16 will receive the lowest dose, in order to explore effects of treatment in younger boys, closer to the age of diagnosis. The study\u2019s primary endpoint is safety and tolerability, the key secondary endpoint is the NIH Toolbox Crystallized Cognition Composite (CCC), and several exploratory endpoints will assess potential effects on mood, behavior, and other symptoms that are relevant to this population. Mirum owns worldwide rights to MRM-3379.<\/p>\n About Mirum Pharmaceuticals<\/b><\/p>\n \nMirum 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 focuses on rare liver and rare genetic diseases, where it has built deep expertise and strong connections to patient communities. The company\u2019s commercial portfolio includes LIVMARLI\u00ae (maralixibat) for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), CHOLBAM\u00ae (cholic acid) for bile-acid synthesis disorders, and CTEXLI\u00ae (chenodiol) for cerebrotendinous xanthomatosis (CTX).<\/p>\n \nMirum\u2019s 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), zilurgisertib, an ALK2 inhibitor under regulatory review with the FDA for fibrodysplasia ossificans progressiva (FOP), and MRM-3379, a PDE4D inhibitor being evaluated for Fragile X syndrome (FXS).<\/p>\n \nMirum\u2019s 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 www.mirumpharma.com<\/a> and follow Mirum on Facebook<\/a>, LinkedIn<\/a>, Instagram<\/a> and X<\/a>.<\/p>\n Forward-Looking Statements<\/b><\/p>\n Statements contained in this press release regarding matters that are not historical facts are \u201cforward-looking statements\u201d within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, commercial results for our approved products, including continued growth in year-over-year net product sales, achievement of our 2026 financial guidance, our anticipated successes in 2026, including continued commercial performance and financial discipline, the results, enrollment, conduct and progress of our ongoing and planned studies for our product candidates, including the timing and results of interim and topline analyses of our ongoing studies, potential approval of NDA filings, the timing of any approvals of NDA filings and the potential commercial launch of our product candidates. 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 \u201canticipate,\u201d \u201cexpected,\u201d \u201cwill,\u201d \u201ccould,\u201d \u201cwould,\u201d \u201cguidance,\u201d \u201cpotential\u201d and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum\u2019s 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 and uncertainties associated with Mirum\u2019s business in general, the impact of geopolitical and macroeconomic events, and the other risks described in Mirum\u2019s Annual Report on Form 10-K for the year ended December 31, 2025 and subsequent filings with the Securities and Exchange Commission. 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\u2019s 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.<\/i><\/p>\n Mirum Pharmaceuticals, Inc.<\/b><\/p>\n Condensed Consolidated Statement of Operations Data<\/b><\/p>\n (in thousands, except share and per share amounts)<\/b><\/p>\n (Unaudited)<\/b><\/p>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n Three Months Ended March 31,<\/b><\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n 2026<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n 2025<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nRevenue:<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nProduct sales, net<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n159,882<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n111,585<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nOperating expenses:<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nCost of sales (1)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n28,805<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n23,018<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nResearch and development<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n97,910<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n41,044<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nAcquired in-process research and development<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n726,302<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n5,000<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nSelling, general and administrative<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n96,330<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n57,706<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nTotal operating expenses (2)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n949,347<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n126,768<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nLoss from operations<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(789,465<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(15,183<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n<\/tr>\n \nOther income (expense):<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nInterest income<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n3,401<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n3,023<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nInterest expense<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(3,616<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(3,596<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n<\/tr>\n \nOther income, net<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u2014<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n2,108<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nNet loss before provision for income taxes<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(789,680<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(13,648<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n<\/tr>\n \nProvision for income taxes<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n475<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n1,029<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nNet loss<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(790,155<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(14,677<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nNet loss per share, basic and diluted<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(13.43<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n(0.30<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n<\/tr>\n \nWeighted-average shares of common stock used to compute net loss per share, basic and diluted<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n58,851,021<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n48,889,058<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n(1) Amounts include intangible amortization expense as follows:<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nIntangible amortization<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n6,685<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n5,893<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n(2) Amounts include stock-based compensation expense as follows:<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nCost of sales<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n348<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n331<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nResearch and development<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n24,189<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n5,229<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nSelling, general and administrative<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n32,937<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n10,243<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nTotal stock-based compensation<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n57,474<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n15,803<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n<\/table>\n Mirum Pharmaceuticals, Inc.<\/b><\/p>\n Condensed Consolidated Balance Sheet Data<\/b><\/p>\n (in thousands)<\/b><\/p>\n (Unaudited)<\/b><\/p>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n March 31, 2026<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n December 31, 2025<\/b><\/p>\n<\/td>\n<\/tr>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n Assets<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nCurrent assets:<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nCash and cash equivalents<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n324,907<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n296,683<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nShort-term investments<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n61,352<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n86,644<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nAccounts receivable<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n133,113<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n123,330<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nInventory<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n25,518<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n24,887<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nPrepaid expenses and other current assets<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n24,546<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n18,140<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nTotal current assets<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n569,436<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n549,684<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nRestricted cash<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n1,733<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n1,482<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nLong-term investments<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n34,353<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n8,105<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nIntangible assets, net<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n254,154<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n260,921<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nOther noncurrent assets<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n31,240<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n22,621<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nTotal assets<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n890,916<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n842,813<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n Liabilities and Stockholders\u2019 Equity<\/b><\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nCurrent liabilities:<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nAccounts payable<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n59,399<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n9,614<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nHoldback liabilities, current<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n25,800<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u2014<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nAccrued expenses and other current liabilities<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n187,625<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n196,185<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nTotal current liabilities<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n272,824<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n205,799<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nOperating lease liabilities, noncurrent<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n11,175<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n7,516<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nConvertible notes payable, net, noncurrent<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n310,251<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n309,797<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nHoldback liabilities, noncurrent<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n48,257<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u2014<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nOther liabilities<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n5,915<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n5,011<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nTotal liabilities<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n648,422<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n528,123<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nCommitments and contingencies<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nStockholders\u2019 equity:<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nPreferred stock<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u2014<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u2014<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nCommon stock<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n6<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n5<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nAdditional paid-in capital<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n1,700,215<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n981,878<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nAccumulated deficit<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(1,457,699<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(667,544<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n<\/tr>\n \nAccumulated other comprehensive (loss) income<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n(28<\/p>\n<\/td>\n \n)<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n351<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nTotal stockholders\u2019 equity<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n242,494<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n314,690<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n \nTotal liabilities and stockholders\u2019 equity<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n890,916<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n \n$<\/p>\n<\/td>\n \n842,813<\/p>\n<\/td>\n \n\u00a0<\/p>\n<\/td>\n<\/tr>\n<\/table>\n \n\u00a0<\/p>\n View source version on businesswire.com: <\/span>https:\/\/www.businesswire.com\/news\/home\/20260506520552\/en\/<\/a><\/span><\/p>\n Investor Contact: Media Contact: KEYWORDS:<\/b> California United States North America<\/p>\n INDUSTRY KEYWORDS:<\/b> Health Clinical Trials Research Pharmaceutical Science Biotechnology<\/p>\n\n
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International: +1 585 542 9983
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Access Code: 487977865<\/p>\n
\n<\/b><\/a>
<\/b>EU SmPC
\n<\/b><\/a>
<\/b>Canadian Product Monograph<\/b><\/a><\/p>\n\n
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\n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n
\n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n <\/p>\n
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Andrew McKibben
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ir@mirumpharma.com<\/a><\/p>\n
\n<\/b>
Meredith Kiernan
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media@mirumpharma.com<\/a><\/p>\n
![\"Logo\"](\"https:\/\/mms.businesswire.com\/media\/20260506520552\/en\/746217\/3\/Mirum_Inline_FullColor_RGB.jpg\")
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