Mirum Pharmaceuticals has reported top-line data from the Phase II EMBARK clinical trial of Livmarli (maralixibat) oral solution in biliary atresia patients.

The double-blind, randomised, placebo-controlled trial analysed the efficacy and safety of Livmarli versus placebo in biliary atresia patients following a Kasai surgery. 

Subjects enrolled in the trial were ≥21 days old and <90 days old during the time of the surgery with biliary atresia.

To be part of the trial, the participants should have undergone Kasai surgery within three weeks before randomisation in the trial.

Mean variation in total bilirubin over week 26 was the trial’s primary endpoint.

Proportion of subjects needing liver transplants or had some liver-associated event over this duration and mean variation in total serum bile acids were the trial’s secondary endpoints.

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The trial failed to meet the primary endpoint and crucial secondary endpoints. 

Baseline characteristics were found to be proportionate across both arms of the trial. 

Livmarli was also found to be well-tolerated in the trial without any new safety findings reported.

Mirum Pharmaceuticals president and CEO Chris Peetz said: “We are disappointed in the outcome of the study in this post-surgery, high-need disease setting. 

“We are grateful to the patients, families, and clinicians who participated in the study and advanced the science around this rare liver disease.”

Mirum focuses on developing therapies for rare diseases that impact children and adults. 

Currently, three medications of the company, Livmarli oral solution, Cholbam capsules, and Chenodal tablets have received approvals for various indications.

In the US, Livmarli is indicated to treat cholestatic pruritus in Alagille syndrome patients. 

Cholbam is approved by the FDA to treat bile acid synthesis disorders while Chenodal is indicated for use under medical necessity recognition in cerebrotendinous xanthomatosis (CTX) patients.

In October 2023, Mirum reported positive data from the Phase III RESTORE trial of Chenodal tablets in CTX patients.