\nMirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today announced that the primary endpoint was met in the Phase 3 AZURE-4 study evaluating brelovitug, an investigational fully human monoclonal antibody that binds to hepatitis B surface antigen (HBsAg), for the treatment of chronic hepatitis delta virus (HDV). AZURE-4 is the second of two Phase 3 studies that will form the basis of Mirum’s U.S. registration package for brelovitug. Mirum also highlighted brelovitug data presented at the American Association for the Study of Liver Diseases\u2019 (AASLD) The Liver Meeting\u00ae. Across three presentations, the data demonstrated deepening viral suppression, increased rates of alanine aminotransferase (ALT) normalization, and improvements in liver stiffness with longer brelovitug treatment, including in patients with advanced cirrhosis.<\/p>\n
\n\u201cAZURE-4 marks the second U.S. registrational Phase 3 study in which brelovitug met the primary endpoint of combined virologic response and ALT normalization, demonstrating consistent efficacy in patients with substantial disease burden, including higher baseline viral loads and ALT levels,\u201d said Nancy Shulman, M.D., Executive Vice President of Clinical Development at Mirum. \u201cTogether with the favorable safety and tolerability profiles observed across both AZURE studies and the deepening responses observed with longer-term treatment in our Phase 2 studies, we believe these results support the potential of brelovitug as a potent, single-agent treatment for chronic HDV and our planned Biologics License Application (BLA) submission in the first half of 2027.\u201d<\/p>\n
\n\u201cHepatitis delta is the most severe form of viral hepatitis, and many patients are living with advanced liver disease, including cirrhosis,\u201d said Tatyana Kushner, M.D., MSCE, Associate Professor of Medicine, Division of Gastroenterology and Hepatology at Weill Cornell Medicine. \u201cNew 2026 AASLD expert opinion (EXPO) recommendations recognize that all adults with HDV viremia and compensated liver disease, including compensated cirrhosis, should be considered for HDV-specific antiviral therapy. The improvements in viral suppression, ALT normalization, and liver stiffness observed with brelovitug treatment are encouraging for patients facing significant risk of disease progression.\u201d<\/p>\n
Phase 3 AZURE-4 Results: Brelovitug Met the Primary Endpoint at Week 24<\/b><\/p>\n
\nThe AZURE-4 study enrolled 113 treatment-na\u00efve patients randomized 2:1:1 to receive brelovitug 300 mg self-administered once weekly (QW) by subcutaneous (SC) injection, brelovitug 900 mg administered once every four weeks (Q4W) by SC injection, or delayed treatment starting at Week 12. The global study population had substantial disease burden: 53% had cirrhosis, approximately half had baseline HDV RNA levels >6 log10<\/sub> IU\/mL, and mean baseline ALT was 122 U\/L.<\/p>\n \nAt Week 24, brelovitug met the primary endpoint, with 59% of patients receiving 300 mg QW and 44% receiving 900 mg Q4W achieving the combined endpoint of virologic response and ALT normalization, compared with 0% in the delayed-treatment arm (p\u22640.0001 for both comparisons).<\/p>\n \nThe table below summarizes key efficacy findings from the Phase 3 AZURE-1 and AZURE-4 studies and longer-term Phase 2 data presented at The Liver Meeting, highlighting deepening antiviral and ALT responses with longer-term brelovitug treatment.<\/p>\n Summary of Phase 3 and Phase 2 Key Efficacy Endpoints<\/b><\/p>\n \nNo patients in the delayed-treatment arms of the AZURE-1 or AZURE-4 Phase 3 studies achieved the primary endpoint.<\/p>\n
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