Mirum Pharmaceuticals (MIRM) Conference presentation summary

Study design

• AZURE-1 is a global, randomized, controlled Phase 2b/3 study of brelovitug versus delayed treatment in chronic hepatitis D; Phase 2b enrolled the first ~50 patients across 10 countries.

• The Week 24 analysis included 53 of 54 randomized participants: 300 mg weekly (n=21), 900 mg every 4 weeks (n=20), and delayed treatment (n=12).

• Primary endpoint: combined HDV RNA response (≥2 log10 reduction or below quantification/undetectable) and ALT normalization at Week 24.

Week 24 efficacy

• Primary endpoint achieved by 35% (7/20) with 300 mg weekly and 45% (9/20) with 900 mg every 4 weeks, versus 0% (0/12) with delayed treatment; p=0.024 and p=0.003, respectively.

• Virologic response rates were 100% (20/20) and 75% (15/20) for brelovitug doses, versus 0% (0/12) with delayed treatment.

• ALT normalization rates were 45% (9/20) and 40% (8/20), versus 8% (1/12) with delayed treatment; normalization at any time through Week 24 was 60% and 50% in the respective dose cohorts.

• HDV RNA and ALT declined from baseline through Week 24 in both brelovitug arms; no virologic breakthrough was observed to date.

Safety and follow-up

• Any adverse event occurred in 52% (11/21) with 300 mg weekly, 50% (10/20) with 900 mg every 4 weeks, and 25% (3/12) with delayed treatment.

• Treatment-related injection-site reactions were the most common adverse events and were mild Grade 1; flu-like syndrome was uncommon.

• Brelovitug was generally well tolerated; multiple Phase 3 trials evaluating brelovitug in chronic hepatitis D are ongoing.