News Release Details
Mirum Pharmaceuticals Announces First Patient Enrolled in Phase 2b VISTAS Clinical Study Evaluating Volixibat in Adult Patients With Primary Sclerosing Cholangitis
Patients 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.
The 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.
“This study is an important step forward in evaluating the potential clinical benefit of volixibat in adult patients with primary sclerosing cholangitis,” said
“We are excited to initiate the volixibat Phase 2 VISTAS study for patients with primary sclerosing cholangitis,” said
Mirum 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
About Primary Sclerosing Cholangitis
Primary sclerosing cholangitis (PSC) is a rare, serious, idiopathic chronic cholestatic liver disease characterized by cholestasis, progressive inflammation, and destruction of bile ducts, which may lead to fibrosis, cirrhosis, portal hypertension, cancer, and ultimately liver failure. It is estimated that approximately 29,000 people in
About Volixibat
Volixibat is an oral, minimally absorbed agent designed to selectively inhibit the apical sodium dependent bile acid transporter (ASBT). Volixibat may offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids, through inhibition of ASBT, thereby reducing bile acids systemically and in the liver. Phase 1 and Phase 2 studies of volixibat demonstrated on-target fecal bile acid excretion, a pharmacodynamic marker of ASBT inhibition, in addition to decreases in LDL cholesterol and increases in 7αC4 which are markers of bile acid synthesis. Volixibat has been evaluated in more than 400 individuals across multiple clinical trials. The most common adverse events reported were mild to moderate gastrointestinal events observed in the volixibat groups.
About Mirum
Mirum is also developing volixibat, also an oral ASBT inhibitor, to evaluate its potential in treating primary sclerosing cholangitis, intrahepatic cholestasis of pregnancy, and primary biliary cholangitis. For more information, visit MirumPharma.com.
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Forward Looking Statements
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the conduct and progress of Mirum’s ongoing and planned studies for volixibat, and the regulatory approval path for maralixibat and volixibat. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “plans,” “will,” ”may,” “expects,” “potential” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum’s business in general, the impact of the COVID-19 pandemic, and the other risks described in Mirum’s filings with the
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