Mirum Expects AZURE-4 Data in Fourth Quarter, Ties Licensing Application to Supportive Results

Key Facts

Mirum Pharmaceuticals said in its official update issued on November 3 that pivotal data from the Phase 3 AZURE-4 study of brelovitug remain expected in the fourth quarter of 2026. That places the results timeline at the center of attention on the drug’s development path for chronic hepatitis D, while the update did not announce a study outcome. The company wrote that "pivotal data from the Phase 3 AZURE-4 study are expected in the fourth quarter of 2026." Mirum tied its next regulatory step to the study’s findings, saying it plans to submit a biologics license application in the United States during the first half of 2027, provided AZURE-4 results are supportive. The awaited study results therefore remain a decisive factor in the timing of the licensing application the company has said it plans to file.

AZURE-4 follows positive results Mirum reported from the Phase 3 AZURE-1 study, which met its composite primary endpoint at week 24 in both dose groups. The rate was 56% among patients receiving 300 mg weekly and 45% among those receiving 900 mg every four weeks, compared with 0% in the treatment-delay group. The study included 153 treatment-naive patients, who were randomly assigned in a 2:2:1 ratio to the two treatment groups and the treatment-delay group. Both comparisons with the treatment-delay group were statistically significant, with a p-value of less than 0.0001 for each dose. The virologic response rate was also 86% with the weekly dose and 85% with the dose given every four weeks, versus 0% in the treatment-delay group.

AZURE-1 results also showed ALT normalization in 63% of the weekly-dose group and 54% of the group receiving a dose every four weeks, compared with 0% in the treatment-delay group at week 24. In follow-up data from Part 2b of the AZURE-1 study, the rate of achieving the composite endpoint with the 300 mg weekly dose rose from 45% at week 24 to 55% at week 48. With the 900 mg dose every four weeks, the rate rose from 35% to 55% over the same period. Follow-up data beyond week 24 were presented in a session at the American Association for the Study of Liver Diseases conference on November 8, from 2:00 to 2:15 p.m. These results relate to AZURE-1 in Phase 2b and do not change the company’s stated timeline for AZURE-4 data.

On safety through week 24, neither brelovitug group recorded serious adverse events or cases in which treatment was discontinued because of an adverse event. Adverse events were reported in 45.8% of participants in the 300 mg weekly group and 61.5% in the 900 mg every-four-weeks group. At the company level, total revenue was $185.7 million in the quarter ended September 30, 2026, compared with $133.0 million in the corresponding quarter of 2025. Unrestricted cash and investments totaled $500.5 million on September 30, 2026, versus $391.4 million on December 31, 2025. MIRM shares closed at $93.18 on November 9, according to EL7 data.

Brelovitug targets patients with chronic hepatitis D, a disease that can progress rapidly to complications including cirrhosis and liver cancer, according to the U.S. Food and Drug Administration. On May 22, 2026, the agency approved Hepcludex as the first treatment for the disease approved in the United States. The American Association for the Study of Liver Diseases estimates that 12 million to 72 million people worldwide are infected with hepatitis D, including up to 75,000 in the United States. Brelovitug has received Breakthrough Therapy designation from the FDA, as well as PRIME and orphan drug designations from the European Medicines Agency. The next milestone is the expected publication of AZURE-4 data in the fourth quarter and whether they support the plan to submit a licensing application in the first half of 2027.