Mirum Pharmaceuticals develops and commercializes therapies for rare liver diseases. Its lead drug LIVMARLI (maralixibat) is an oral ASBT inhibitor that lowers bile acid buildup to relieve itching in cholestatic conditions such as Alagille syndrome, and the company is pursuing LIVMARLI for additional rare liver diseases, with volixibat as another ASBT inhibitor in its pipeline. The company differentiates itself by focusing on rare pediatric and adult cholestatic diseases and building dedicated commercialization infrastructure in the U.S. and other major markets, including partnerships with physicians, payers, and patient groups to improve patient access. Mirum’s goal is to bring effective therapies to patients with rare bile-duct and liver disorders, expand LIVMARLI indications, and establish a sustainable market presence in key markets.
Company Size
201-500
Company Stage
IPO
Headquarters
Foster City, California
Founded
2018
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Mirum's liver infection drug lowers virus levels in late-stage study. By Thomson Reuters Sep 28, 2026 | 8:49 AM By Sneha S K Sept 28 (Reuters) - Mirum Pharmaceuticals on Monday said its experimental hepatitis drug reduced levels of the virus in patients, meeting the main goal of a late-stage study. Here are some key details: - Mirum was testing brelovitug, an experimental monoclonal antibody that binds to the hepatitis B surface antigen, a protein found on the surface of the virus - The company was testing the treatment for chronic hepatitis delta virus, a liver infection that affects people already infected with hepatitis B - The disease, which the company estimates affects about 230,000 people in the US and Europe, can cause severe symptoms that can lead to liver damage and, in some cases, death - At 24 weeks, 56% of patients given 300 milligrams of brelovitug and 45% of those given 900 milligrams showed reduced hepatitis D virus levels and normal liver enzyme levels, compared with 0% in the delayed-treatment group, the company said - The company expects the drug to generate peak sales of at least $1 billion - The only FDA-approved treatment for the condition is Gilead's Hepcludex - "Brelovitug has a better benefit/risk profile," said RBC Capital Markets analyst Lisa Walter, adding that the latest results position it as the best-in-class treatment option - The drug was well tolerated across dose groups, with a safety profile consistent with previously reported data, Mirum said - Full results from the study will be presented at an upcoming medical congress, with the company expecting to submit a marketing application for the drug to the US FDA in the first half of 2027. (Reporting by Sneha S K in Bengaluru; Editing by Jonathan Ananda and Diti Pujara)
The US Food and Drug Administration has approved Atebrioz (zilurgisertib) for adult and paediatric patients aged 12 and older with fibrodysplasia ossificans progressiva, a rare genetic disease affecting approximately 300 people in the United States. Mirum Pharmaceuticals and Incyte announced the approval, which makes Atebrioz the latest treatment option for the progressive condition that causes bone to form in muscles, tendons, and other soft tissues. The once-daily oral tablet works by inhibiting ALK2, targeting the disease-driving pathway. Clinical trial data showed that at 24 weeks, mean total new heterotopic ossification volume decreased by 3.2 cm³ in patients receiving the drug compared with an increase of 24.6 cm³ in placebo-treated patients. Atebrioz is expected to be commercially available in October through Mirum Access Plus, with eligible patients paying as little as $0 per month.
Mirum Pharmaceuticals has appointed Dr Rob Myers as chief medical officer, effective 21 September 2026. Dr Myers will lead the company's global medical and clinical development functions. Dr Myers brings over 20 years of experience in drug development, with particular expertise in hepatology. He joins from OrsoBio, where he served as chief medical officer and head of development. Previously, he held senior leadership positions at Gilead Sciences, overseeing clinical development programmes across liver fibrosis, cholestatic liver diseases, and gastrointestinal inflammation. Dr Myers has published more than 200 peer-reviewed papers and has contributed to advancing understanding of primary sclerosing cholangitis. He holds an MD from Western University and a master's degree in epidemiology from the University of Calgary.
US FDA approves Regeneron's rare bone disorder drug. Regeneron's Pasatru has received US FDA approval for adults with FOP after clinical trials showed a significant reduction in abnormal bone formation. By Reuters August 20, 2026, 8:59:40 AM IST (Published) The US FDA on Wednesday (August 19) approved Regeneron's drug for a rare genetic disorder after it significantly reduced abnormal bone formation in certain soft tissues. New York-based Regeneron's shares rose 4%. The drug, garetosmab, branded as Pasatru, was approved to treat adults with fibrodysplasia ossificans progressiva (FOP) - a condition in which muscle, tendon and ligament tissue gradually turns into bone, leading to a "second skeleton" that causes progressive loss of mobility and reduced life expectancy. In a 56-week trial involving 63 participants, Pasatru reduced the development of new bone abnormalities by 94% in patients treated with a 3 mg per kg dose and by 90% in those given a 10 mg per kg dose, compared with a placebo. The drug works by blocking Activin A, a protein involved in triggering abnormal bone growth in patients with FOP. Susan Rhee, a member of Regeneron's clinical team, told Reuters last week that the company plans to start a trial for children later this year. The drug will compete with French drugmaker Ipsen's oral treatment Sohonos, which in 2023 became the only other treatment approved by the US Food and Drug Administration. Incyte and partner Mirum Pharma, as well as privately held Ashibio, are also developing treatments for the condition. In 2020, Regeneron paused dosing in a mid-stage trial of the drug after five patient deaths, ultimately discontinuing the study and working with global regulators to design the late-stage trial, according to the company. The condition affects roughly 1 in 2 million people worldwide, with around 800 to 900 active diagnosed cases globally, according to data from the National Institutes of Health.
Mirum Pharmaceuticals shares plunged 15% on Thursday after the FDA recommended an additional Phase 3 study for its liver disease candidate Volixibat, potentially delaying the new drug application until the first half of 2027. Citizens lowered its price target to $137 from $146 whilst maintaining an Outperform rating. The regulatory setback overshadowed strong second-quarter results. Mirum reported revenue of $176.2 million, beating analysts' estimates of $167.3 million. The company raised its full-year net product sales guidance to $680 million to $700 million, from $660 million to $680 million. Mirum ended the quarter with $561.3 million in cash and equivalents. Volixibat has already received FDA Breakthrough Therapy and Orphan Drug designations.