Recent Updates — MIRM
Mirum Pharmaceuticals announced positive Week 24 results from Cohort 2 of the pivotal PROGRESS study for ATEBRIOZ (zilurgisertib) in pediatric patients aged 6 to <12 years with fibrodysplasia ossificans progressiva. Treatment reduced total heterotopic ossification lesion volume by a mean of -8.09 cm³ compared to an increase of 9.14 cm³ for placebo, yielding a treatment difference of -14.0 cm³ (p=0.003). No treated patients developed new lesions, and joint mobility scores improved significantly versus placebo. The company plans to submit a supplemental New Drug Application in the first half of 2027 to expand the indication to this younger age group. Mirum Pharmaceuticals operates as a biopharmaceutical company focused on developing treatments for rare diseases.
Mirum Pharmaceuticals announced that the Phase 3 EXPAND study of LIVMARLI (maralixibat) for treating cholestatic pruritus in patients aged six months or older with ultra-rare cholestatic liver diseases, including biliary atresia, met its primary endpoint and achieved statistical significance across all five prespecified secondary endpoints. The trial enrolled 52 patients randomized to maralixibat or placebo during a 20-week double-blind period. Maralixibat demonstrated a statistically significant improvement in pruritus compared with placebo, showing a mean 1.81-point reduction from baseline versus 0.83 points for placebo, and a 70.1% reduction in serum bile acids versus 3.5% for placebo. The company plans to submit a supplemental New Drug Application in the United States in the first half of 2027 based on these results. Mirum Pharmaceuticals operates as a biopharmaceutical company focused on developing treatments for rare liver diseases.
Mirum Pharmaceuticals announced FDA approval of Atebrioz (zilurgisertib) for fibrodysplasia ossificans progressiva, with commercial availability expected in October. The company also reported that brelovitug met its primary endpoint in the Phase 3 AZURE-1 study for chronic hepatitis delta, achieving virologic response and ALT normalization in 56% of patients on the 300 mg dose. Mirum estimates annual revenue potential at over $200 million for Atebrioz and more than $1 billion for brelovitug. The company plans to submit a BLA for brelovitug in early 2027 with a potential launch in late 2027. Mirum Pharmaceuticals operates in the biopharmaceutical industry, developing treatments for rare diseases.
Mirum Pharmaceuticals announced that Rob Myers, M.D., will succeed Joanne Quan, M.D., as Chief Medical Officer. Dr. Quan ceased serving in this role on September 2, 2026, with her employment termination expected on October 19, 2026. The separation is treated as involuntary under the company's Severance Plan, entitling Dr. Quan to standard benefits and an extension of her stock option exercise period to 12 months post-separation. Additionally, her 2026 annual target bonus will be paid at the 100% achievement level upon separation. Dr. Myers is expected to begin employment on September 21, 2026. Mirum Pharmaceuticals operates in the biopharmaceutical industry, focusing on the development of treatments for liver diseases.
Mirum Pharmaceuticals reported second quarter 2026 net product sales of $176.2 million and raised full-year guidance to $680–$700 million. The company issued $690 million in convertible senior notes due 2032 and settled approximately 75% ($237.2 million) of its 2029 convertible notes, holding $561.3 million in unrestricted cash as of June 30, 2026. Regulatory updates include FDA Breakthrough Therapy designation for volixibat in primary sclerosing cholangitis and a pre-NDA meeting indicating a potential Phase 3 study before an H1 2027 submission. Zilurgisertib’s PDUFA date is September 26, 2026, while brelovitug topline results are expected in Q3 and Q4 2026. Mirum Pharmaceuticals operates as a rare disease company focused on liver and genetic diseases.