Long-Term LIVMARLI Data for ALGS and PFIC to be Presented at EASL Congress

Mirum Pharmaceuticals Inc. announced significant findings from extended studies on LIVMARLI, an oral solution aimed at treating Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC). Scheduled to be presented at the European Association for the Study of the Liver (EASL) Annual Congress in Milan, Italy, these studies underscore the sustained clinical benefits of LIVMARLI.

Alagille Syndrome Efficacy

Mirum's research, particularly data from the MERGE study, reveals that patients with ALGS exhibit durable clinical benefits over a seven-year treatment period with LIVMARLI. This study, identified as THU-094, will be presented by Dr. Doug Mogul. The data indicates significant improvement in clinical outcomes for ALGS patients, emphasizing the long-term efficacy of this treatment.

Progressive Familial Intrahepatic Cholestasis Insights

For patients with PFIC, LIVMARLI shows promising results in enhancing growth and improving key liver markers. Key presentations, THU-129 and THU-158, will be led by Professor Richard J. Thompson and Dr. Lorenzo D’Antiga, respectively. Findings from the MARCH and MARCH-ON trials demonstrate significant improvements in serum bile acid levels, which are linked to better liver health markers in children receiving LIVMARLI treatment.

Symposium on Pediatric and Adult Liver Disease Treatment

A Mirum-sponsored symposium on June 5 will focus on the role of elevated serum bile acids in cholestatic liver diseases, exploring the potential application of pediatric experience to adult hepatology. Drs. Verena Keitel-Anselmino, Marco Carbone, Kris Kowdley, and Richard Thompson will discuss the unmet needs in primary sclerosing cholangitis (PSC) and the potential of ileal bile acid transporter (IBAT) inhibitors as emerging therapies.

About LIVMARLI

LIVMARLI (maralixibat) is an orally administered IBAT inhibitor approved for treating cholestatic pruritus in ALGS patients aged three months and older in the U.S., and in PFIC patients aged five years and older. It is also approved in Europe and Canada for treating cholestatic pruritus in ALGS. The drug has received Breakthrough Therapy designation for ALGS and PFIC type 2, and orphan designation for both conditions.

Safety and Side Effects

While LIVMARLI has shown significant benefits, it is not recommended for PFIC type 2 patients with severe bile salt export pump (BSEP) protein defects. Potential side effects include liver injury, gastrointestinal problems, and fat-soluble vitamin (FSV) deficiency. Regular monitoring through blood tests and physical exams is advised to manage these risks.

Mirum Pharmaceuticals

Mirum Pharmaceuticals is dedicated to developing treatments for rare diseases affecting children and adults. Besides LIVMARLI, the company offers CHOLBAM for bile acid synthesis disorders and CHENODAL for cerebrotendinous xanthomatosis (CTX). Mirum's pipeline includes investigational treatments like volixibat for PSC and primary biliary cholangitis, and CHENODAL, which has shown positive results in Phase 3 trials for CTX.

Future Prospects

Mirum's participation in the EASL Annual Congress highlights the company's commitment to advancing treatment options for rare liver diseases. With ongoing research and promising clinical data, Mirum aims to expand the therapeutic potential and commercial viability of LIVMARLI across various liver disease indications.

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