Mirum Pharmaceuticals Highlights Data Demonstrating Impact of LIVMARLI (maralixibat) on Patients with Alagille Syndrome at European Society for Paediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN) Annual Meeting

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) presented data from clinical programs evaluating maralixibat (LIVMARLI) in patients with Alagille syndrome at the 54^th Annual ESPGHAN Meeting. These data further characterize LIVMARLI’s clinical potential in pediatric patients including evaluation of its impact on long-term event-free survival.

“We are thrilled for the opportunity to share key long-term findings from our clinical studies with the pediatric liver community during ESPGHAN,” said Dr. Pam Vig, head of research and development at Mirum. “The years of data that have been generated with maralixibat allow us to look critically at important long-term treatment goals such as growth, transplant rates and predictors of improved outcomes.”

Summaries of data presented during the congress are enclosed below. View the presentations in full within our Publications & Presentations section on our website.

H-O-018: Maralixibat-treated patients with ALGS demonstrate improved event-free survival in a natural history comparison with patients from the GALA database: Application of real-world evidence analytics

Dr. Bettina Hansen on behalf of Dr. Binita Kamath and the GALA Study Group (Oral presentation)

The presentation reported on an analysis independently conducted and presented by Dr. Bettina Hansen and the Global Alagille Alliance (GALA) Study Group, which has aggregated the largest global natural history clinical database established for ALGS. The analysis evaluated time to first clinical event using six years of follow-up data from pooled ALGS maralixibat studies (n=84) and compared it against an external natural history control cohort from the GALA clinical database. Events were defined as liver transplantation, biliary diversion surgery, decompensation events (ascites requiring therapy or variceal bleeding), or death. Additional analyses included transplant-free survival as well as several sensitivity and subgroup analyses to confirm robustness of the findings.

The analysis demonstrated a highly significant improvement in six-year event-free survival with a p-value of <0.0001 (HR: 0.305, 95% CI: 0.189-0.491) translating to a 70% overall reduction for clinical outcomes with maralixibat. The analysis also showed highly statistically significant improvements in transplant-free survival with a p-value of <0.0001 (HR: 0.332, 95% CI: 0.197-0.559).