Mirum Pharmaceuticals Inc（MIRM）资讯与事件MIRMMirum Pharmaceuticals Inc

Mirum Pharmaceuticals Announces Phase 3 Results for LIVMARLI

Mirum Pharmaceuticals announced topline results from the Phase 3 EXPAND study evaluating LIVMARLI for the treatment of cholestatic pruritus in patients aged 6 months or older with ultra-rare cholestatic liver diseases, including biliary atresia. The study met its primary endpoint and achieved statistical significance across all five prespecified secondary endpoints, including the three endpoints in patients with biliary atresia and a native liver. Treatment with maralixibat demonstrated a statistically significant improvement in pruritus compared with placebo in the primary analysis population of pediatric patients. Safety findings in EXPAND were consistent with prior clinical experience with maralixibat and no new safety signals were observed. Key safety data from the Phase 3 EXPAND study are presented below in the Safety Summary table.

Mirum Pharmaceuticals Meets Primary Endpoint in AZURE-1 Study

Mirum Pharmaceuticals announced the primary endpoint was met in the Phase 3 portion of the AZURE-1 study evaluating brelovitug, an investigational fully human monoclonal antibody that binds to hepatitis B surface antigen, for the treatment of chronic hepatitis delta virus. In addition, 48-week data from the Phase 2b portion of AZURE-1 demonstrated deepening viral suppression, with a greater proportion of patients achieving HDV RNA below the lower limit of quantification and target not detected, and increased rates of alanine aminotransferase normalization. AZURE-1 is one of two pivotal Phase 3 studies intended to form the basis of Mirum's U.S. registration package for brelovitug. "These results demonstrate the potential of brelovitug as a well-tolerated, convenient, single agent to deliver both deep viral suppression and ALT normalization, with responses that continue to deepen through 48 weeks of treatment. Notably, these results were achieved in a broad patient population, including those with significant liver inflammation, cirrhosis and clinically significant portal hypertension," said Nancy Shulman, M.D., Executive Vice President of Clinical Development at Mirum. "We look forward to reporting topline results from AZURE-4 later this year and advancing toward our planned BLA submission in the first half of 2027." The Phase 3 portion of the AZURE-1 study enrolled 153 treatment-naive patients randomized in a 2:2:1 ratio to receive brelovitug 300 mg self-administered once weekly by subcutaneous injection, brelovitug 900 mg administered once every four weeks by SC injection, or delayed treatment starting at Week 24. This global study enrolled a broad patient population including patients with advanced disease. At Week 24, treatment with brelovitug met the primary endpoint, with 56% of patients in the 300 mg QW arm and 45% in the 900 mg Q4W arm achieving the combined endpoint of virologic response nd ALT normalization, compared to 0% in the delayed treatment arm. The efficacy results by treatment arm at Week 24 are presented below in Phase 3 Key Efficacy Endpoints. Treatment with brelovitug was well tolerated across dose groups, with a safety profile consistent with previously reported AZURE-1 data.