NGM Bio hits endpoint in Phase 2 NASH study
- NGM Bio's engineered hormone drug reached a mid-stage study's primary endpoint of reduction in absolute liver fat content by 5% or more in all patients with nonalcoholic steatohepatitis, also known as NASH, at 12 weeks.
- The South San Francisco biotech's agent, NGM282, also improved liver fibrosis by one or more stages in 42% of patients, by two stages in 16% of patients, and improved the non-alcoholic fatty liver disease (NAFLD) activity score in over 80% of patients, according to results presented at the International Liver Congress 2018 in Paris on Saturday.
- The next step will be a Phase 2b study in NASH patients later in 2018.
NASH and its precursor, NAFLD, are on the rise, with a global prevalence as high as 24%. NAFLD affects both adults and children and is on track to become the most common cause of end-stage liver disease over the next few decades. Many, but not all, cases are linked with obesity.
NAFLD can lead to a number of different disorders including cancer, putting a strain on the healthcare system. Resolving NAFLD and reversing fibrotic changes in NASH could have a significant impact on patient outcomes.
"It is unprecedented to see such profound histological improvement in well-established NASH after only twelve weeks of treatment, confirming that NGM282 is potently impacting many clinical dimensions of the disease," said Stephen Harrison, medical director at Pinnacle Clinical Research and principal investigator on the Phase 2 study.
Data for the drug reported at last year's EASL meeting showed similar effects on liver fat, but the more recent findings looked at histological data too, which demonstrated the additional and all-important impact on fibrosis.
"These new histology data support the translation of [non-invasive measures of NASH] into reversals of liver fibrosis in many patients, a clinically meaningful measure of disease outcomes," noted Alex DePaoli, chief medical officer at NGM Bio.
Further investigation will look more into the anti-steatotic, anti-inflammatory and anti-fibrotic activity of NGM282.
In 2018 alone, the NASH field has seen much activity.
Poxel SA, funded by a diabetes deal with Roivant Sciences Ltd., is advancing its pipeline, with plans for a Phase 2a trial of the candidate PXK-770 in NASH and NAFLD. MediciNova Inc., meanwhile, cut short a Phase 2 trial of tipelukast after early positive results, and will push forward with more advanced studies.
Big pharma and biotech is active in the space as well. Regeneron Pharmaceuticals Inc. and Alnylam Pharmaceuticals Inc. have signed a discovery deal for RNAi-based NASH therapeutics. Gilead Sciences Inc. is hoping for key clinical data for filgotinib in 2018, with a potential launch in NASH by 2020. And Eli Lilly & Co has licensed out three NASH candidates to California biotech Terns Pharmaceuticals Inc., making use of Terns' expertise in China.
- NGM Bio Statement
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