Mirum Pharmaceuticals publishes phase 3 MARCH maralixibat data in The Lancet

James Spargo | May 8, 2024 | News story | Research and Development Hepatology, Mirum Pharmaceuticals, PFIC, The Lancet, clinical trials 

Biopharma company Mirum Pharmaceuticals has announced data from its phase 3 MARCH-PFIC study which evaluated maralixibat in patients with progressive familial intrahepatic cholestasis (PFIC). Results were published in The Lancet Gastroenterology and Hepatology.

MARCH-PFIC was a randomised, double-blind, placebo-controlled study – the largest in patients with PFIC. The aim was to confirm the efficacy and safety of maralixibat, or LIVMARLI – an IBAT inhibitor – across a PFIC genetic range. Efficacy analyses were tested in two groups: BSEP deficiency (BSEP or PFIC2) and All-PFIC, which included patients with FIC1 (PFIC1), BSEP (PFIC2), MDR3(PFIC3), TJP2 (PFIC 4) or MYO5B deficiencies.

Results show that the study reached its primary efficacy endpoint of change in pruritus severity score between baseline and the last 12 weeks of treatment, and key secondary endpoint of a change in total serum bile acid concentration between baseline and the average of weeks 18, 22 and 26 of the study.

Dr Alexander Miethke, Cincinnati Children’s Hospital and lead author on the publication, commented, “This data advances our understanding of LIVMARLI’s potential to provide meaningful improvements in pruritus and a number of parameters impacting patients with various PFIC types. The clinically meaningful reductions in serum bile acid and bilirubin levels also signify the potential for improvements in native liver survival in PFIC, a step forward in considering the long-term care and health of patients with PFIC. LIVMARLI presents a new non-surgical treatment option in PFIC with strong efficacy data and safety demonstrated across a range of PFIC types, which is important for patients with PFIC types previously unstudied.”

Pam Vig PhD, chief scientific officer and head of research at Mirum, stated: “We are pleased to see the data published and recognised by The Lancet as they demonstrate meaningful improvements in many clinical signs and symptoms seen in PFIC patients. Patients with PFIC suffer from cholestasis which can lead to debilitating cholestatic pruritus, as well as poor growth and progressive liver disease. These data demonstrate the significant impact LIVMARLI can have in this severe cholestatic setting.”

James Spargo

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