Mirum Pharmaceuticals, Inc. is a biopharmaceutical company. The firm focuses on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases. Its products include Maralixibat and Volixibat. The company was founded by Niall O'Donnel, Michael Grey and Christopher Peetz in May 2018 and is headquartered in Foster City, CA.
Clinical Trials
82
40 active
Approvals
11
Total approvals
Agencies
3
Regulatory bodies
Founded
2018
Active, not recruiting
39
47.6%
Approved For Marketing
1
1.2%
Completed
21
25.6%
Not yet recruiting
1
1.2%
Recruiting
15
18.3%
Terminated
2
2.4%
Unknown
1
1.2%
Withdrawn
2
2.4%
- The FDA approved Atebrioz (zilurgisertib) tablets at 100 mg once daily to reduce total new heterotopic ossification volume in patients aged 12 and older with FOP. - In the Phase 2 PROGRESS study, mean total new HO lesion volume fell 3.2 cm3 with zilurgisertib versus a 24.6 cm3 increase with placebo at Week 24. - Zilurgisertib was generally well tolerated, with headache, arthralgia, upper respiratory tract infection, epistaxis and nausea as the most common adverse reactions. - Mirum licensed zilurgisertib from Incyte for worldwide development; an EMA marketing authorization application is under review and pediatric cohorts continue enrolling.
- Milestone Pharmaceuticals reported an operating loss of $62 million over the last twelve months with an operating margin of -4019%, signaling severe profitability challenges. - Mirum Pharmaceuticals posted a $70 million operating loss (LTM) despite a 130% stock price surge over the past 12 months, raising valuation concerns. - Both companies remain cash-flow negative, with Milestone's free cash flow margin at -3192% and Mirum's at -32%, underscoring unsustainable burn rates. - Share-based compensation remains elevated at both firms, reaching 483% of revenue at Milestone and 16% at Mirum, potentially diluting shareholder value.
- Mirum Pharmaceuticals announced a $620 million acquisition of Bluejay Therapeutics to gain worldwide rights to brelovitug, a monoclonal antibody with FDA Breakthrough Therapy designation for chronic hepatitis delta virus (HDV). - Brelovitug demonstrated 100% HDV RNA response in Phase 2 studies and is currently in the global AZURE Phase 3 registrational program, with top-line results expected in the second half of 2026. - HDV affects approximately 230,000 people in the United States and Europe, with more than 50% of patients dying from liver-related causes within 10 years of diagnosis. - The acquisition strengthens Mirum's rare liver disease portfolio and leverages the company's existing expertise in rare liver disorders and established provider relationships.
- DelveInsight's 2025 analysis reveals over 30 companies are actively developing more than 30 therapeutic candidates for liver cirrhosis, driven by rising global prevalence of liver diseases, particularly NAFLD associated with obesity and metabolic syndrome. - Sagimet Biosciences received FDA Breakthrough Therapy and Fast Track designations for denifanstat in February 2025, while Madrigal Pharmaceuticals plans to launch REZDIFFRA in Europe starting with Germany in the second half of 2025. - Multiple Phase III trials are progressing, including Akero Therapeutics' completed enrollment in the SYNCHRONY Real-World study with results expected in the first half of 2026, and ongoing ENLIGHTEN trials for MASH cirrhosis. - Key investigational therapies advancing through clinical development include Volixibat, TVB-2640, belapectin, RTX001, and LPCN 1148, representing diverse mechanisms of action from sodium-bile acid cotransporter inhibition to macrophage cell therapy.
- Mirum Pharmaceuticals has enrolled the first patient in the BLOOM phase II study evaluating MRM-3379, a PDE4D inhibitor, for treating Fragile X syndrome, marking a significant milestone for this rare genetic neurocognitive disorder. - MRM-3379 is an orally available, brain-penetrant selective phosphodiesterase-4D inhibitor designed to enhance cAMP signaling, with preclinical studies showing improvements in cognitive and behavioral symptoms linked to FXS. - The study targets male participants with confirmed genetic diagnosis of FXS, addressing a critical unmet medical need as no approved therapies currently exist for this condition affecting approximately 50,000 males in the US and Europe. - Top-line safety and tolerability data from the BLOOM study are expected in 2027, potentially offering the first novel therapeutic approach for improving cognition and daily function in FXS patients.
- Mirum Pharmaceuticals has completed enrollment in the Phase 2b VISTAS study evaluating volixibat, an oral IBAT inhibitor, for treating cholestatic pruritus in primary sclerosing cholangitis patients. - The trial previously met pre-specified efficacy and safety thresholds at a blinded interim analysis in 2024, with topline results expected in the second quarter of 2026. - Primary sclerosing cholangitis is a rare, progressive liver disease with no currently approved therapies, leaving patients with limited treatment options focused on symptom management. - Volixibat works by selectively inhibiting the ileal bile acid transporter to reduce bile acid recycling and has received breakthrough therapy designation for cholestatic pruritus in primary biliary cholangitis.
- Mirum Pharmaceuticals reported second quarter revenue growth to $127.79 million and raised full-year 2025 revenue guidance to between $490 million and $510 million, exceeding analyst expectations of $108.1 million. - The company narrowed its net loss to $5.86 million for the quarter while earnings per share came in at negative 12 cents, significantly better than the predicted negative 33 cents. - Mirum filed a new $200 million shelf registration covering common and preferred stock, debt securities, and warrants to support future growth initiatives. - Analysts upgraded price targets with Citigroup raising from $79 to $81 and Raymond James increasing to $82, citing the commercial strength of Livmarli driving sales projections.
- GSK's linerixibat received FDA acceptance for review in June 2025 to treat cholestatic pruritus in primary biliary cholangitis patients, with a PDUFA goal date of March 24, 2026. - Gilead Sciences secured European Commission conditional marketing authorization for seladelpar in February 2025 for PBC treatment in combination with UDCA or as monotherapy. - COUR Pharmaceuticals' CNP-104 obtained FDA Orphan Drug Designation in January 2025, positioning it as a potentially first-in-class disease-modifying treatment for PBC. - The PBC pipeline demonstrates robust activity with 18+ companies developing 20+ therapies, including promising candidates like elafibranor, ASC42, and OP-724 in various clinical stages.
- DelveInsight's 2025 pipeline analysis reveals over 30 companies developing 30+ liver cirrhosis therapies, with promising candidates like Rezdiffra, efruxifermin, and belapectin showing significant clinical progress. - Madrigal Pharmaceuticals' Rezdiffra demonstrated reduced liver scarring in compensated cirrhosis patients in Phase 3 MAESTRO-NAFLD-1 trial data, while Akero Therapeutics reported preliminary results from its Phase IIb SYMMETRY study of efruxifermin. - The liver cirrhosis treatment market is experiencing robust growth driven by rising global prevalence of liver diseases, particularly NAFLD, fueled by lifestyle factors including obesity and metabolic syndrome. - Key pipeline therapies span diverse mechanisms including sodium-bile acid cotransporter inhibitors, regulatory T-lymphocyte stimulants, and Wnt signaling pathway inhibitors across oral, intravenous, and subcutaneous administration routes.
- Mirum Pharmaceuticals' LIVMARLI (maralixibat) has received FDA approval for a new tablet formulation to treat cholestatic pruritus in patients with Alagille syndrome and Progressive Familial Intrahepatic Cholestasis. - The tablet version provides a convenient one-tablet per dose option for older patients, while maintaining the liquid formulation for younger patients, enhancing treatment flexibility for physicians and patients. - LIVMARLI, an ileal bile acid transporter (IBAT) inhibitor, is now approved in over 40 countries for ALGS and 30 countries for PFIC, with the new tablet formulation expected to be available in June 2025.
