Mirum Pharmaceuticals Announces Data from EMBARK Phase 2b Study for Biliary Atresia

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), today announced top-line results of the Phase 2 EMBARK study evaluating LIVMARLI (maralixibat) oral solution versus placebo given as an adjuvant therapy to Kasai surgery in patients with biliary atresia. The study did not meet its primary endpoint of mean change in bilirubin from baseline to Week 26, or the key secondary endpoints. Baseline characteristics were well-balanced between the groups. LIVMARLI was generally well-tolerated, with no new safety findings.

“We are disappointed in the outcome of the study in this post-surgery, high-need disease setting,” said Chris Peetz, president and chief executive officer at Mirum. “We are grateful to the patients, families, and clinicians who participated in the study and advanced the science around this rare liver disease.”

About the EMBARK Study

The Phase 2b EMBARK study is a randomized, double-blind, placebo-controlled clinical trial evaluating the safety and efficacy of LIVMARLI (maralixibat) oral solution in patients with biliary atresia who have undergone a Kasai surgery. Patients enrolled in the study were ≥21 days old and <90 days old at the time of the Kasai surgery and had a clinical diagnosis of biliary atresia; patients underwent a Kasai surgery within three weeks prior to randomization in EMBARK. The primary endpoint was mean change in total bilirubin through Week 26, followed by secondary endpoints including mean change in total serum bile acids, and proportion of patients who required liver transplant or had another liver related event over the 26 weeks.

IMPORTANT SAFETY INFORMATION

LIVMARLI can cause side effects, including:

Changes in liver tests. Changes in certain liver tests are common in patients with Alagille syndrome and can worsen during treatment with LIVMARLI. These changes may be a sign of liver injury and can be serious. Your healthcare provider should do blood tests before starting and during treatment to check your liver function. Tell your healthcare provider right away if you get any signs or symptoms of liver problems, including nausea or vomiting, skin or the white part of the eye turns yellow, dark or brown urine, pain on the right side of the stomach (abdomen) or loss of appetite.