Mirum Pharmaceuticals, Inc. develops and commercializes medicines for rare diseases, with recurring updates centered on product sales, clinical readouts, regulatory interactions and employee equity grants. The company’s commercial portfolio includes LIVMARLI® (maralixibat) for Alagille syndrome and progressive familial intrahepatic cholestasis, CHOLBAM® (cholic acid) for bile-acid synthesis disorders, and CTEXLI® (chenodiol) for cerebrotendinous xanthomatosis.
News about MIRM also covers clinical-stage programs including volixibat, an IBAT inhibitor for primary sclerosing cholangitis and primary biliary cholangitis; brelovitug, a monoclonal antibody designed to bind hepatitis B surface antigen for chronic hepatitis delta virus; and MRM-3379, a PDE4D inhibitor for Fragile X syndrome. Company updates report financial results, pipeline licensing activity and inducement awards under Nasdaq Listing Rule 5635(c)(4).
Mirum Pharmaceuticals (MIRM) reported third-quarter 2026 net product sales of $185.7 million and raised its full-year sales guidance. Revenue compared with $133.0 million a year earlier; guidance is now $715 million to $720 million. LIVMARLI sales rose 45% to $133.4 million, while Bile Acid Medicines sales grew 28% to $52.3 million.
Mirum received FDA approval and launched ATEBRIOZ for fibrodysplasia ossificans progressiva in patients aged 12 years and older. It also reported positive results for younger ATEBRIOZ patients, LIVMARLI EXPAND and brelovitug AZURE-1. Mirum plans several regulatory submissions in H1 2027, with brelovitug dependent on supportive AZURE-4 results. Quarterly operating expenses increased to $245.5 million from $130.4 million. Cash, cash equivalents and investments reached $500.5 million, versus $391.4 million at year-end 2025; Mirum repurchased $20.0 million principal of convertible notes.
Mirum Pharmaceuticals (MIRM) will present rare liver disease data at AASLD’s The Liver Meeting in Denver on November 5–9, 2026. Presentations cover investigational brelovitug for chronic hepatitis delta virus, investigational volixibat for primary sclerosing cholangitis and approved LIVMARLI.
Brelovitug’s Phase 3 AZURE-1 study met its combined viral-response and liver-enzyme-normalization endpoint at Week 24 in both dose arms; longer-term data show responses deepening with continued treatment. Volixibat analyses show itch improvement across baseline severity levels, including in patients with inflammatory bowel disease without worsening disease activity. LIVMARLI presentations include itch improvement in patients with progressive familial intrahepatic cholestasis carrying compound heterozygous variants.
Mirum Pharmaceuticals (Nasdaq: MIRM) will report third quarter 2026 financial results on November 3, 2026, and host a conference call. The call begins at 4:30 p.m. ET / 1:30 p.m. PT and will cover financial results and recent corporate progress. A webcast will be accessible through the Investors section of Mirum’s website, with an archived replay available afterward.
Mirum Pharmaceuticals (NASDAQ: MIRM) reported reduced abnormal bone growth with ATEBRIOZ in children with fibrodysplasia ossificans progressiva in PROGRESS Cohort 2. The randomized study enrolled 25 patients aged 6 to <12 years. At Week 24, mean heterotopic ossification (HO), or abnormal bone, lesion volume fell 8.09 cm³ with zilurgisertib versus a 9.14 cm³ increase with placebo (nominal p=0.003). No treated patients developed new HO lesions, versus one placebo patient. Joint-mobility impairment scores improved by 0.80 points versus 0.09 points.
No new safety signals or growth plate closures occurred in treated patients, and no patients discontinued treatment during the double-blind period. Mirum plans an application in H1 2027 to expand FDA approval to ages 6 to <12. ATEBRIOZ received FDA approval in September 2026 for patients aged 12 years and older with FOP.
Mirum Pharmaceuticals (Nasdaq: MIRM) will participate in upcoming investor conferences, with its first fireside chat scheduled for November 10, 2026.
The Wolfe Research Healthcare Conference chat begins at 8:00 a.m. ET. A Stifel Healthcare Conference chat follows November 11 at 1:20 p.m. ET, and a Jefferies Global Healthcare Conference presentation takes place November 17 at 12:30 p.m. GMT. Live webcasts and archived replays will be accessible through Mirum’s Investors website section.
Mirum Pharmaceuticals (MIRM) granted equity inducement awards to new employees and its newly appointed chief medical officer on October 9, 2026. The awards included options for 18,663 shares and 24,687 restricted stock units (RSUs) for 18 new employees, plus options for 40,000 shares and 25,000 RSUs for Chief Medical Officer Rob Myers. All options have an exercise price of $86.01 per share, equal to the stock’s closing price that day.
Mirum Pharmaceuticals (MIRM) reported that LIVMARLI met the primary endpoint in its Phase 3 EXPAND study of ultra-rare cholestatic liver diseases. All five prespecified secondary endpoints also achieved statistical significance. The 52-patient trial included 46 children and six adults treated during a 20-week double-blind period.
In children, observer-rated itch scores improved 1.81 points with maralixibat versus 0.83 with placebo over Weeks 13–20, a 0.98-point treatment difference (p=0.0004). Serum bile acids showed a 69.0% treatment effect versus placebo (p<0.0001). Patients with biliary atresia and a native liver had a 1.15-point itch-score treatment difference (p=0.0002). No new safety signals were observed, but diarrhea occurred in 64.0% of treated patients versus 11.1% with placebo. Mirum plans a U.S. supplemental New Drug Application, seeking an additional approved use, in the first half of 2027.
Mirum Pharmaceuticals (MIRM) will host an investor call on October 12, 2026, to share topline results from the Phase 3 EXPAND study.
The call begins at 8:30 a.m. ET/5:30 a.m. PT. EXPAND evaluates LIVMARLI (maralixibat) for cholestatic pruritus, or itching, in patients aged 6 months or older with ultra-rare cholestatic liver diseases, including biliary atresia. A webcast and archived replay will be available through the company’s Investors website section.
Mirum Pharmaceuticals (MIRM) reported that brelovitug met the Phase 3 AZURE-1 primary endpoint in chronic hepatitis delta at Week 24. The combined measure of viral response and normalized alanine aminotransferase (ALT), a marker of liver injury, was reached by 56% on 300 mg weekly and 45% on 900 mg every four weeks, versus 0% with delayed treatment (p<0.0001 for each comparison).
Through Week 24, neither treated arm reported a serious or grade 3-plus adverse event. Adverse events occurred in 45.8% and 61.5% of treated patients, versus 27.6% in the delayed-treatment arm. In the Phase 2b portion, the combined response reached 55% in each dose arm at Week 48, up from 45% and 35%, respectively, at Week 24; no new safety signals were observed through Week 48. Mirum expects results from a second pivotal study in Q4 2026 and plans a U.S. biologics license application in H1 2027.
Mirum Pharmaceuticals (MIRM) will host an investor call on September 28, 2026, to share topline Phase 3 AZURE-1 results.
The call will also cover 48-week data from the study’s Phase 2b portion. AZURE-1 studies brelovitug in chronic hepatitis delta virus. The call begins at 8:30 a.m. ET, and an archived webcast will be available for replay.