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Phase 3 primary endpoint (virologic response and ALT normalization) achieved in both dose arms at Week 24
– 48-week data from Phase 2b portion of AZURE-1 demonstrate deepening viral suppression and increased rates of ALT normalization with longer-term treatment
– Favorable safety and tolerability profiles observed; no new safety signals
– Mirum to host conference call to discuss topline results today, September 28, at 8:30 a.m. ET
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today 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 (HBsAg), for the treatment of chronic hepatitis delta virus (HDV). 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 (LLOQ, <10 IU/mL) and target not detected (TND), and increased rates of alanine aminotransferase (ALT) 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 goal of treating chronic HDV is to prevent progression to cirrhosis, liver cancer and liver failure, and that requires controlling both the virus and liver inflammation," said Norah Terrault, M.D., MPH, Professor of Medicine and Chief of the Division of GI and Liver at the Keck School of Medicine of USC, and an AZURE-1 investigator. "ALT is a marker of liver injury, so seeing virologic response alongside ALT normalization is encouraging in a long-term therapy. For patients facing the most aggressive form of viral hepatitis, these are important results."
"Chronic hepatitis delta is a lifelong disease, and people living with it need treatment options that are effective, safe and tolerable enough to stay on over time. That's especially true for patients with more advanced disease, who often have the fewest choices," said Chari A. Cohen, DrPH, MPH, President of the Hepatitis B Foundation. "Results like these bring hope to a community that has had few options until recently."
Phase 3 AZURE-1 Results: Brelovitug Met the Primary Endpoint at Week 24
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 (QW) by subcutaneous (SC) injection, brelovitug 900 mg administered once every four weeks (Q4W) 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 (≥2 log10 reduction in HDV RNA from baseline or undetectable HDV RNA
