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Mirum Pharmaceuticals Announces First Patient Enrolled in Phase 2b VISTAS Clinical Study Evaluating Volixibat in Adult Patients With Primary Sclerosing Cholangitis
FOSTER CITY, Calif.--(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that the first patient has been enrolled in Mirum’s Phase
About this update from Mirum Pharmaceuticals, Inc.
[{"type":"text","content":" FOSTER CITY, Calif.--(BUSINESS WIRE)--\nMirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that the first patient has been enrolled in Mirum’s Phase 2b VISTAS study evaluating volixibat in adult patients with primary sclerosing cholangitis (PSC), an idiopathic chronic cholestatic liver disease characterized by progressive inflammation and destruction of bile ducts often leading to serious liver disease, cancer, and ultimately liver failure. Volixibat, an oral, minimally absorbed medication, is designed to offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids through inhibition of the apical sodium dependent bile acid transporter (ASBT), thereby reducing bile acids systemically and in the liver.\n\nPatients meeting eligibility criteria for the study are randomized to receive volixibat 20 mg twice daily (BID) or 80 mg BID, or placebo for 28 weeks, after which time all patients will receive volixibat through the open-label extension phase of the study.\n\nThe primary endpoint will evaluate the change in pruritus from baseline for volixibat versus placebo using the Adult ItchRO tool. Secondary endpoints evaluated will include safety and tolerability, quality of life measures, and serum bile acids. Exploratory endpoints will include assessments of liver fibrosis and other markers of PSC progression.\n\n“This study is an important step forward in evaluating the potential clinical benefit of volixibat in adult patients with primary sclerosing cholangitis,” said Christopher Bowlus, MD, chief of gastroenterology and hepatology at UC Davis Health. “There are currently no approved therapies indicated for the treatment of patients with PSC suffering from pruritus, a common and often debilitating symptom of the disease.”\n\n“We are excited to initiate the volixibat Phase 2 VISTAS study for patients with primary sclerosing cholangitis,” said Ed Tucker, MD, chief medical officer at Mirum. “Our learnings from the maralixibat development program have informed the design of our volixibat study with the hope of alleviating the burdensome effects experienced by patients with PSC.”\n\nMirum has ongoing studies evaluating cholestatic liver diseases in pediatric patients with maralixibat, also an ASBT inhibitor. The company has initiated a rolling NDA submission to the U.S. Food and Drug...