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Hepatology Communications Publishes Data from Mirum’s LIVMARLI in Primary Sclerosing Cholangitis
First and only data evaluating IBAT inhibition in treating primary sclerosing cholangitis (PSC) Data provided proof-of-concept for Phase 2b VISTAS study
About this update from Mirum Pharmaceuticals, Inc.
[{"type":"text","content":"\n\nFirst and only data evaluating IBAT inhibition in treating primary sclerosing cholangitis (PSC)\n\n\n\nData provided proof-of-concept for Phase 2b VISTAS study evaluating volixibat, an IBAT inhibitor, in patients with PSC\n\n\n\n FOSTER CITY, Calif.--(BUSINESS WIRE)--\nMirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that Hepatology Communications published data from the CAMEO study evaluating the safety, tolerability, and efficacy of LIVMARLI® (maralixibat) oral solution in patients with primary sclerosing cholangitis (PSC).\n\n\nThe CAMEO study was the first proof-of-concept study evaluating an ileal bile acid transporter (IBAT) inhibitor in patients with pruritus associated with PSC, a cholestatic liver disease. The open-label study (n=27) evaluated the safety and tolerability of LIVMARLI for 14 weeks in adults with PSC measuring pruritus, serum bile acid levels, and liver function.\n\n\nIn this open-label Phase 2 pilot study, pruritus scores and serum bile acid concentration decreased over the 14 weeks of treatment with LIVMARLI. Participants whose sBA was elevated above normal at baseline achieved a 40% reduction (n=18). Similarly, participants with an average pruritus score of ≥3 out of 10 at baseline achieved a 70% improvement in pruritus (n=8). (The most common treatment emergent adverse events (TEAEs) were gastrointestinal related (diarrhea, nausea, and abdominal pain). The TEAEs were generally tolerable and consistent with the expected mechanism of action of IBAT inhibitors.\n\n\nThe full publication including additional data from the analysis can be accessed in Hepatology Communications.\n\n\n“PSC is a rare, chronic, progressive cholestatic liver disease. Patients with PSC frequently suffer from severe pruritus that negatively impacts their quality of life,” said Christopher Bowlus, MD, chief of gastroenterology and hepatology at University of California, Davis, and lead author on the publication. “Treatment options for pruritus in patients with PSC are limited and unproven. The early data observed in the CAMEO study provides evidence that IBAT inhibitors may provide benefit to patients with PSC suffering from pruritus.”\n\n\n“These are the first published data on the potential for IBAT inhibition for the treatment of patients with PSC,” said Pam Vig, PhD, head of research and development at Mirum. “The ...