Mirum Pharmaceuticals Provides AZURE Clinical Program Update for brelovitug in Chronic Hepatitis Delta Virus
Mirum Pharmaceuticals, Inc. a leading rare disease company, today announced completion of enrollment in the Phase 3 AZURE-1 study and completion of screening in the Phase 3 AZURE-4 study, both evaluating brelovitug for the treatment of chronic hepatitis delta virus (HDV). Achievement of these milestones confirms the expected timing of topline 24-week data in the second half of 2026. AZURE-1 and AZURE-4 together will form the basis of Mirum’s U.S. BLA submission for brelovitug, which has received Breakthrough Therapy designation from the FDA for the treatment of chronic HDV infection.
Brelovitug is being developed for HDV, the most severe form of viral hepatitis, and a disease with no approved therapies in the U.S. HDV occurs in people already infected with hepatitis B virus (HBV) and is associated with rapid progression to liver fibrosis, cirrhosis, liver cancer, and liver-related death.
The completion of AZURE-1 enrollment and AZURE-4 screening represent important execution milestones as we advance brelovitug through the Phase 3 AZURE program,” said Joanne Quan, M.D., Chief Medical Officer at Mirum Pharmaceuticals. “We look forward to reporting interim data from AZURE-1 in the second quarter and topline results from AZURE-1 and AZURE-4 in the second half of 2026. Both studies are evaluating a composite endpoint of virologic response and ALT normalization at 24 weeks, aligned with FDA guidance for accelerated approval in HDV, and building on the strong antiviral activity observed in our Phase 2 study.
HDV is an aggressive disease that progresses quickly and leaves patients with very limited treatment options,” said Tatyana Kushner, M.D., MSCE, Associate Professor of Medicine, Division of Gastroenterology and Hepatology at Weill Cornell Medicine. “Having a well-tolerated single-agent therapy that achieves viral suppression and improves liver inflammation is critical for patients with HDV, and the progress of these Phase 3 studies brings us closer to potentially delivering a new therapy to patients in urgent need.
Topline data from AZURE-1 and AZURE-4 are expected in 2H 2026, with a potential BLA submission and launch in the U.S. in 2027.
About Brelovitug
Brelovitug is an investigational, highly potent, pan-genotypic, fully human immunoglobulin G1 (IgG1) monoclonal antibody (mAb) that targets the surface antigen (anti-HBsAg) on both the chronic hepatitis delta virus (HDV) and the hepatitis B virus (HBV). Brelovitug is designed to neutralize and remove hepatitis B and hepatitis D virions and deplete HBsAg-containing subviral particles. Brelovitug has FDA Breakthrough Therapy designation for the treatment of chronic HDV infection and PRIME and Orphan designations from the European Medicines Agency.
In the Phase 2 study, brelovitug demonstrated strong antiviral activity in HDV, achieving a 100% HDV RNA response, along with improvements in liver enzyme levels and a favorable safety profile, with the most common adverse event being injection-site erythema. Mirum owns worldwide rights to brelovitug.
About the AZURE Clinical Program
The AZURE program is a global, registrational Phase 3 clinical development program evaluating brelovitug for the treatment of chronic hepatitis delta virus (HDV). The program includes multiple open-label studies designed to assess the primary endpoint of combined virologic and biochemical response. Together, the studies are intended to support regulatory filings in the United States and Europe.
About AZURE-1 and AZURE-4
AZURE-1 and AZURE-4 are Phase 3 studies that form the basis of Mirum’s U.S. FDA BLA submission for brelovitug in chronic hepatitis delta virus (HDV).
AZURE-1 is evaluating approximately 200 treatment-naïve patients randomized in a 2:2:1 ratio to receive brelovitug 300 mg once weekly, brelovitug 900 mg once monthly, or 24-week delayed therapeutic start. AZURE-4 is evaluating approximately 80 treatment-naïve patients randomized in a 2:1:1 ratio to receive brelovitug 300 mg once weekly, brelovitug 900 mg once monthly, or 12-week delayed therapeutic start. Patients randomized to delayed treatment initiate brelovitug 300 mg once weekly upon completion of the treatment delay in both studies. AZURE-4 is being conducted in partnership with PSI CRO.
The primary endpoint for both studies is the proportion of patients achieving combined virologic response and ALT normalization at Week 24, consistent with FDA guidance for accelerated approval in HDV. Both studies include open-label extension periods of up to 96 weeks.
About Mirum Pharmaceuticals
Mirum Pharmaceuticals is a leading rare disease company with a global footprint of approved products and a broad pipeline of investigational medicines. Purpose-built to bring forward breakthrough medicines for people with overlooked conditions, Mirum combines deep rare disease expertise with strong connections to patient communities.
The company’s commercial portfolio includes LIVMARLI® (maralixibat) for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), CHOLBAM® (cholic acid) for bile-acid synthesis disorders, and CTEXLI® (chenodiol) for cerebrotendinous xanthomatosis (CTX). Mirum’s clinical-stage pipeline includes volixibat, an IBAT inhibitor in late-stage development for primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), brelovitug, a fully human monoclonal antibody in late-stage development for chronic hepatitis delta virus (HDV) and MRM-3379, a PDE4D inhibitor being evaluated for Fragile X syndrome (FXS).
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