Mirum Pharmaceuticals publishes data from Livmarli analysis

Mirum Pharmaceuticals announced that Hepatology published an analysis demonstrating a statistically significant improvement in six-year event-free and transplant-free survival in patients with Alagille syndrome, or ALGS, treated with Livmarli oral solution when compared with a natural history control group. The analysis evaluated time to clinical outcome from the pooled Livmarli clinical studies versus a control cohort from the Global Alagille Alliance, or GALA, clinical database, the largest global ALGS natural history database. The pre-specified statistical analysis was conducted independently and compared time to first clinical event in the Livmarli-treated patients with ALGS versus a well-selected external natural history cohort treated with standard of care from the GALA database. Events were defined as liver transplantation, biliary diversion surgery, manifestations of portal hypertension, or death. The GALA control group was identified based on a pre-specified and blinded selection process to align with eligibility criteria from the Livmarli clinical studies. Multiple sensitivity and subgroup analyses were conducted to ensure statistical robustness of the primary result. Data from the analysis demonstrated a significant improvement in six-year event-free survival translating to a 70% overall reduction for clinical outcomes with Livmarli. The analysis also showed statistically significant improvements in transplant-free survival as compared to the GALA cohort. Sensitivity and subgroup analyses demonstrated overall consistency with the primary results.