Hepatology Publishes Six-Year Data Demonstrating Improved Clinical Outcomes in Patients with Alagille Syndrome Treated with Mirum’s LIVMARLI

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today 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 (ALGS) treated with LIVMARLI (maralixibat) oral solution when compared with a natural history control group (p<0.0001). The analysis evaluated time to clinical outcome from the pooled LIVMARLI clinical studies versus a control cohort from the Global Alagille Alliance (GALA) clinical database, the largest global ALGS natural history database.

“Patients with Alagille syndrome often undergo liver transplantation for complications related to cholestasis,” said Binita M. Kamath, MBBChir, Staff Physician and Senior Associate Scientist, The Hospital for Sick Children (SickKids), Toronto, Ontario, Canada, and senior author of the manuscript. “This seminal six-year analysis of LIVMARI versus a comparable natural history control group demonstrates a 70% risk reduction for clinical outcomes in patients treated with LIVMARLI.”

“We are grateful to the GALA Study Group for this important academic contribution and are thrilled about the recognition of this analysis by Hepatology,” said Pam Vig, PhD, chief scientific officer and head of research at Mirum. “We would like to thank the patients who participated in the LIVMARLI studies as well as the Alagille Syndrome Alliance for their continued partnership and dedication to advancing research in this rare liver disease.”

The pre-specified statistical analysis was conducted independently by the lead author Dr. Bettina Hansen, Professor of Clinical Biostatistics at Erasmus MC, Netherlands and compared time to first clinical event in the LIVMARLI-treated patients with ALGS (n=84) versus a well-selected external natural history cohort treated with standard of care from the GALA database (n=469). 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.