Mirum Pharmaceuticals dosed the first patient in a phase 3 trial of maralixibat, its lead drug candidate for the treatment of pediatric patients with progressive familial intrahepatic cholestasis, according to a press release.
“PFIC is a devastating condition that negatively affects children and their families,” Chris Peetz, president and CEO of Mirum, said in the release. “With no approved drug therapy, the start of the phase 3 MARCH-PFIC clinical trial is an important milestone for Mirum and puts us one step closer to potentially offering children a new treatment option that targets bile acid overload, a primary driver of liver damage and pruritus in this progressive disease.”
Maralixibat is an inhibitor of the apical sodium-dependent bile acid transporter that is being developed for cholestatic liver diseases including PFIC and Alagille syndrome. Results from the phase 2 INDIGO trial showed significant and durable reductions in serum bile acids and clinically meaningful improvements in pruritus.
The randomized phase 3 trial will comprise 30 patients with residual bile salt exporter pump function aged 1 years to 17 years. The investigators will evaluate the reduction in severity of pruritus as measured by the ItchRO(Obs) scale.
Additional endpoints will include mean change in pruritus frequency, mean change in serum bile acids, mean change in quality of life and the proportion of patients who experience an improvement from baseline in height and weight.
“There is an immediate need for effective treatment options for children with PFIC who currently suffer from the life-dominating manifestations of this disease,” Robert Squires, MD, from the University of Pittsburgh Medical Center Children’s Hospital of Pittsburgh, said. “The potential of maralixibat to alleviate the intense itch and control elevated bile acid levels associated with PFIC2, could represent an important new option for physicians, patients and caregivers.”