Policy & Regulation
Mirum Pharmaceuticals treats first PFIC paediatric patient in Phase 3 MARCH-PFIC clinical trial of maralixibat
10 July 2019 -

Biopharmaceutical company Mirum Pharmaceuticals stated on Tuesday that it has dosed the first paediatric patient with progressive familial intrahepatic cholestasis (PFIC) under the Phase 3 MARCH-PFIC clinical trial of its lead drug candidate maralixibat, with topline results in fourth quarter of 2020.

The company said maralixibat is being developed as a therapy for cholestatic liver diseases including PFIC and Alagille syndrome. These diseases result in impaired bile acid flow and accumulation of toxic levels of bile acids in the liver, leading to progressive liver damage that may ultimately result in liver failure.

According to the company, Maralixibat is an inhibitor of the apical sodium-dependent bile acid transporter (ASBT), which recycles bile acids from the intestine to the liver.

This Phase 3 MARCH-PFIC, global randomized, placebo-controlled clinical trial will evaluate the company's maralixibat compared to placebo for six months followed by a long-term open label extension study in which all patients will receive maralixibat. The primary endpoint is a reduction in severity of pruritus as measured by the ItchRO(Obs) scale and will include up to 30 patients with residual BSEP function (PFIC2 non-truncating) aged one to 17 years.

In conjunction, the MARCH-PFIC trial will be available to patients in more than 40 pediatric hepatology centers spanning the US, Canada, Europe, the Middle East, Asia and Latin America, disclosed the company.

Under the company's prior Phase 2 INDIGO clinical trial, the blocking bile acid transport from the intestine to the liver with maralixibat resulted in profound and durable reductions in serum bile acids (sBA), clinically meaningful improvements in pruritus and acceleration of growth in some patients with PFIC2.