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NGM reports positive Phase II trial results of NGM282 in PBC patients

PBR Staff Writer Published 25 March 2015

US-based NGM Biopharmaceuticals has reported positive results from a Phase II trial of NGM282, an engineered protein variant of the human hormone FGF19, in patients with primary biliary cirrhosis (PBC).

In the trial, NGM282 showed statistically significant, dose-dependent and clinically meaningful reductions in alkaline phosphatase (ALP), a primary indicator of disease activity, as well as substantial reductions in 7a-Hydroxy-4-cholesten-3-one (C4), a marker of bile acid synthesis.

The company said the results suggest that NGM282 is a potent regulator of bile acid synthesis.

NGM chief executive officer William Rieflin said: "This Phase II study demonstrated that NGM282 causes significant improvements on established markers of disease progression and could deliver meaningful clinical benefits to patients with PBC.

"However, in light of treatment alternatives that may be available to PBC patients before our daily injectable therapeutic could gain approval, we are pursuing additional studies of NGM282 in other orphan bile acid-related diseases where the pronounced activity on bile acid synthesis as demonstrated by C4 reduction may provide benefit."

Around 45 patients were enrolled in the double blind, placebo-controlled trial and they were randomized 1:1:1 to daily injections of 0.3mg or 3mg doses of NGM282 or placebo.

Patients, who participated in the trial, had a confirmed PBC diagnosis with an inadequate response to ursodeoxycholic acid (UDCA), the current standard of care, defined as an elevated ALP level greater than 1.67 times the upper limit of normal while on therapeutic doses of UDCA for at least 12 months.