Familial Primary Biliary Cirrhosis

Mirum Pharmaceuticals remains “excited about the clear response seen in reduction of serum bile acids and pruritus” in its study to date, CEO Chris Peetz said. Mirum Pharmaceuticals has blamed enrollment problems for its decision to discontinue a phase 2 study of volixibat in a pregnancy-associated liver disorder. The OHANA study launched in January 2021 with the aim of enrolling 280 pregnant participants with intrahepatic cholestasis, a potentially serious liver disorder that can develop in pregnancy and lead to fetal complications, according to ClinicalTrials.gov. The trial was designed to compare volixibat, an ileal bile acid transporter, to placebo in terms of safety and its ability to reduce elevated serum bile acid concentrations. However, the company faced “challenges in enrollment in this high-risk pregnancy setting,” which led it to halt the trial. Discontinuing OHANA allows Mirum to prioritize other programs, the company pointed out in a release Nov. 29. Mirum remains “excited about the clear response seen in reduction of serum bile acids and pruritus” in the study to date, CEO Chris Peetz said in the release. This has built confidence in phase 2 programs exploring volixibat as a treatment for two liver diseases: primary sclerosing cholangitis (PSC) and primary bilary cirrhosis (PBC). An interim analysis of the VISTAS trial in PSC is expected in the middle of 2023, while an interim analysis of the VANTAGE study in PBC is due in the second half of next year. The company also took the opportunity to celebrate a “very successful year” for Livmarli, its minimally absorbed ileal bile acid transporter inhibitor. In September 2021, Livmarli became the first medicine approved by the FDA for a genetic disorder in children called Alagille syndrome. By the end of this year, Mirum expects Livmarli to get the green light in Europe for the same indication—treating itchiness and skin lesions associated with the syndrome—but for children as young as two months. The company also intends to submit a supplemental new drug application for the drug in progressive familial intrahepatic cholestasis in the first half of next year, with a phase 2b readout in biliary atresia due for the second half of 2023.