Mirum Pharmaceuticals’ LIVMARLI Data Showcased at ESPGHAN Annual Meeting

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced data presented during the 56^th European Society for Paediatric, Gastroenterology, Hepatology, and Nutrition (ESPGHAN) Annual Meeting which took place this week in Milan, Italy. Data from LIVMARLI (maralixibat) oral solution clinical studies and real-world settings in Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC) were presented in oral and poster presentations during the meeting.

“We continue to build upon the strong body of evidence demonstrating LIVMARLI’s potential to provide long-term benefit to PFIC patients across key quality of life and liver disease parameters, as well as improvements in varied genetic types of PFIC,” said Pam Vig, PhD, chief scientific officer and head of research at Mirum. “Further, we are pleased to demonstrate data that shows children treated with LIVMARLI are reducing or discontinuing some antipruritic medications and nutritional supplements.”

Abstract 587: Long-term Maintenance of Response and Improved Liver Health with Maralixibat in Patients with Progressive Familial Intrahepatic Cholestasis: 2-year Data From the MARCH-ON Study
*Plenary Session: Hepatology Highest Scoring Abstracts*
Presented by Professor Richard J. Thompson, MD, King’s College London, United Kingdom
Patients with PFIC showed significant and sustained improvements in pruritus severity, serum bile acid (sBA) levels, total bilirubin and growth following up to two years of LIVMARLI treatment. Similar improvements in pruritus and sBA were seen in patients originally randomized to placebo who received LIVMARLI in the open-label study.

Abstract 594: Maralixibat Leads to Significant Reductions in Bilirubin for Patients with Progressive Familial Intrahepatic Cholestasis: Data from the MARCH Trial
Presented by Lorenzo D’Antiga, MD, Papa Giovanni XXIII Hospital, Bergamo, Italy
Patients treated with LIVMARLI experienced significant decreases in both total and direct bilirubin compared to placebo. 40% of the patients with abnormal bilirubin at baseline treated with LIVMARLI achieved normalization versus none in the placebo group. Further, these reductions in bilirubin were consistent with reductions in sBAs.

Abstract 600: Maralixibat Impact on Concomitant Medication Use for the Treatment of Cholestatic Pruritus in Alagille Syndrome: Real-World Experience
Presented by Jolan Terner-Rosenthal, PhD, Mirum Pharmaceuticals, Inc., Foster City, California, USA