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

FOSTER CITY, Calif. — Mirum Pharmaceuticals, Inc., 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.

 

About Mirum Pharmaceuticals, Inc.

Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat) oral solution, Cholbam® (cholic acid) capsules, and Chenodal® (chenodiol) tablets.

LIVMARLI, an IBAT inhibitor, is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the U.S. (three months and older), in Europe (two months and older), and in Canada. Mirum has also submitted LIVMARLI for approval in the U.S. in cholestatic pruritus in PFIC patients three months of age and older and in Europe in PFIC for patients two months of age and older. Cholbam is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme defects and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms or liver disease. Chenodal has received medical necessity recognition by the FDA to treat patients with cerebrotendinous xanthomatosis (CTX).

Mirum’s late-stage pipeline includes two investigational treatments for debilitating liver diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2b VISTAS study for primary sclerosing cholangitis and Phase 2b VANTAGE study for primary biliary cholangitis. Lastly, Chenodal, has been evaluated in a Phase 3 clinical study, RESTORE, to treat patients with CTX.

 

Contacts