Mirum Pharmaceuticals Inc.
04/27/2026 | Press release | Distributed by Public on 04/27/2026 06:08
Material Event (Form 8-K)
| Item 8.01 | Other Events. |
On April 27, 2026, Mirum Pharmaceuticals, Inc. (the “Company”) announced the primary endpoint was met in the Phase 2b portion of the AZURE-1 study evaluating brelovitug, an investigational monoclonal antibody designed to bind hepatitis B surface antigen (HBsAg), for the treatment of chronic hepatitis delta virus (HDV).
The Phase 2b portion of the AZURE-1 study included the first 53 patients evaluated at Week 24 of treatment.
At Week 24, treatment with brelovitug demonstrated robust antiviral activity across both dose groups. 100% of patients in the 300 mg once weekly (QW) arm and 75% of patients in the 900 mg once every four weeks (Q4W) arm achieved virologic response (≥2 log10 reduction in HDV RNA from baseline or undetectable HDV RNA [
Consistent with these antiviral effects, the primary composite endpoint of virologic response and alanine aminotransferase (ALT) normalization was achieved in 45% and 35% of patients in the 300 mg QW and 900 mg Q4W arms, respectively, as compared to 0% of patients in the delayed treatment arm. After 24 weeks of treatment, further reductions in ALT and HDV RNA levels have been observed. These results support the potential of brelovitug as a single agent therapy to treat HDV.
The efficacy results by treatment arm in the Phase 2b portion of AZURE-1 at Week 24 are presented below in Key Efficacy Endpoints.
Treatment with brelovitug was well tolerated across dose groups. The safety profile summary is presented below in Summary of Safety.
The full results from the Phase 2b portion of the AZURE-1 study will be presented in a late-breaking poster presentation at the European Association for the Study of the Liver (EASL) Congress, May 27-30, 2026. Topline data from the Phase 3 AZURE-1 and AZURE-4 studies are expected in H2 2026, with potential BLA submission and commercial launch in the U.S. in 2027.
Key Efficacy Endpoints
| Endpoint | 300 mg QW (n=20) | 900 mg Q4W (n=20) | Delayed Treatment Arm (n=12) | |||
| Virologic Response | ||||||
| (HDV RNA ≥2 log10 reduction or TND) | 100% | 75% | 0% | |||
| HDV RNA | 30% | 5% | 0% | |||
| ALT Normalization | 45% | 40% | 8% | |||
| Primary Endpoint | ||||||
| (Virologic Response + ALT Normalization) | 45% | 35% | 0% | |||
| P-value* | 0.003 | 0.024 |
Full analysis set, participants receiving at least one post baseline efficacy assessment
| * | P-values compare each treatment group against delayed treatment using a stratum-adjusted Cochran-Mantel-Haenszel (CMH) test. |
2
Summary of Safety
| Participants who experienced, n (%) | 300 mg QW N=21 | 900 mg Q4W N=20 | Delayed Treatment Arm N=12 | |||||||||
| AEs | ||||||||||||
| Any | 11 (52) | 10 (50) | 3 (25) | |||||||||
| Related to treatment | 7 (33) | 7 (35) | 0 | |||||||||
| Grade 3+ | ||||||||||||
| Any | 1 (5)† | 0 | 0 | |||||||||
| Related to treatment | 0 | 0 | 0 | |||||||||
| Serious | ||||||||||||
| Any | 0 | 1 (5)# | 0 | |||||||||
| Related to treatment | 0 | 0 | 0 | |||||||||
| AE Leading to Discontinuation of study drug | 0 | 0 | 0 | |||||||||
| Injection site reactions | 3 (14) | 4 (20) | 0 | |||||||||
| Flu-like Symptoms | 0 | 1 (5) | 0 | |||||||||
| † | Grade 3 AE of musculoskeletal pain, not related |
| # | Hospitalization for liver cirrhosis, class B, in a patient with recent history of ascites and hypoalbuminemia, not related and resolved |
Mirum Pharmaceuticals Inc. published this content on April 27, 2026, and is solely responsible for the information contained herein. Distributed via EDGAR on April 27, 2026 at 12:14 UTC. If you believe the information included in the content is inaccurate or outdated and requires editing or removal, please contact us at [email protected]