NGM Reports Top-Line Results From Phase 2 Study Of NGM282 In Patients With Primary Sclerosing Cholangitis (PSC)
NGM282, an Engineered Analogue of FGF19, Significantly Improves Biomarkers of Hepatic Injury and Fibrosis in PSC Patients
SOUTH SAN FRANCISCO, CA, February 5, 2018 – NGM Bio today announced initial data from an exploratory Phase 2 study of patients with PSC who were treated with NGM282, an engineered, non-tumorigenic FGF19 analogue that is being developed for the treatment of nonalcoholic steatohepatitis (NASH) and other liver diseases. This study did not meet the primary endpoint of statistically significant change in alkaline phosphatase (ALP), a presumptive biomarker of PSC disease progression, from baseline to week 12. However, NGM282 demonstrated both statistically significant improvements in biomarkers of hepatic injury and fibrosis as well as statistically significant reductions in biomarkers of bile acid synthesis and serum bile acids, all consistent with past studies of this experimental medicine in other liver diseases. NGM282 met the secondary endpoints in the study and was generally safe and well tolerated by study subjects. Complete results from this clinical trial will be presented at an upcoming medical meeting.
“These encouraging results highlight both the broad activity of NGM282 across multiple, important dimensions of cholestatic disease and the confounding challenge of treating diseases like PSC, for which a robust understanding of disease progression is lacking,” noted NGM’s Chief Medical Officer, Alex DePaoli. “We are grateful to the patients, investigators and study staff who participated in this study and look forward to working with those investigators to interpret the encouraging signals from these results.”
About the Exploratory Phase 2 Study
This Phase 2 proof of concept study evaluated NGM282 1mg or 3mg versus placebo for 12 weeks in PSC patients to assess biologic activity and safety. The primary endpoint was change in ALP from baseline, with secondary endpoints of safety and biomarkers of bile acid synthesis, as well as surrogates of hepatic injury and liver fibrosis. A total of 35 sites were activated in the United States, Netherlands, United Kingdom and France with 62 patients randomized across the three arms of the study.
About Primary Sclerosing Cholangitis
PSC is a rare, life-threatening, chronic cholestatic liver disease characterized by progressive destruction of bile ducts that leads to the development of cirrhosis and end-stage liver disease or cancer in a majority of patients. In patients with PSC, the bile ducts become blocked due to inflammation and scarring or fibrosis. This causes bile to accumulate in the liver, where it gradually damages liver cells and causes cirrhosis, or fibrosis of the liver. As cirrhosis progresses and the amount of scar tissue in the liver increases, the liver slowly loses its ability to function. The scar tissue may block drainage of the bile ducts, leading to infection of the bile. There are no approved therapies for PSC, and estimated survival time from PSC diagnosis to liver transplant is approximately 10 years. Males are slightly more likely to contract PSC, and as many as 75% of patients have concomitant inflammatory bowel disease, most often ulcerative colitis. Although it is a rare disease, PSC is the seventh leading indication for liver transplant in adults in the United States.
NGM282 is a non-tumorigenic, engineered variant of the human hormone FGF19 that has demonstrated dramatic reductions in liver fat content and improvement in liver function and biomarkers of fibrosis by targeting multiple pathogenic pathways of disease. NGM Bio has generated robust preclinical and clinical evidence supporting NGM282’s ability to improve steatosis, inflammation and fibrosis in NASH patients. This wholly-owned, single-agent therapeutic has been evaluated in four Phase 2 studies in primary biliary cholangitis, PSC, type 2 diabetes and NASH. NGM282’s safety database includes clinical data from more than 350 individuals.
About NGM Bio
NGM Bio is a research-driven biotechnology company committed to discovering and developing novel biologics for the treatment of life-threatening diseases. NGM Bio’s portfolio consists of multiple programs in clinical testing and more than a dozen additional programs in various stages of preclinical development. The company’s most advanced compound, NGM282, is a wholly-owned asset that recently completed a Phase 2 trial in NASH. NGM Bio has established a broad strategic collaboration with Merck. NGM Bio is backed by The Column Group, Merck, Prospect Ventures, Topspin Partners, Rho Ventures, Tichenor Ventures and other leading investors around the world. For more information, please visit www.ngmbio.com.