Phase II efficacy data for NGM Biopharmaceuticals’ developmental non-alcoholic steatohepatitis (NASH) drug, NGM-282, was presented at the 2018 International Liver Congress (ILC) in Paris, France. The investigational treatment was found to produce a relative reduction in liver fat content in patients, as well as improvements in liver fibrosis. These results place NGM Biopharmaceuticals in a position to initiate Phase IIb placebo-controlled studies to further assess the efficacy of its product in NASH patients.

NGM-282 is a first-in-class drug, developed through the adaptive engineering of a human hormone known as FGF19, which plays a role in bile acid synthesis and liver function. Overexpression of this hormone has been shown to increase the risk of hepatocellular carcinoma. NGM-282 has been engineered so that it no longer carries the risk of tumorigenesis, while still maintaining its ability to modulate liver function.

The key role of FGF19 in bile acid synthesis initially drove NGM Biopharmaceuticals to position NGM-282 as a developmental drug for primary sclerosing cholangitis (PSC), a rare condition which results in a buildup of bile in the liver. Although the drug remains in development for PSC, the company has begun exploring the potential for the drug in treating NASH, a disease with a much greater prevalence than PSC.

NGM-282’s potential in NASH

The potential of NGM-282 for the treatment of NASH was assessed in an exploratory open-label Phase II trial that enrolled 22 NASH patients. A 3mg daily dose of the study drug was administered by injection over a 12-week period. The primary endpoint was a reduction in the absolute liver fat content of greater than 5%, with other measures of liver histology included as exploratory endpoints. Overall, 19 of 22 patients completed treatment and were included in the study data analysis.

NGM-282 was found to meet its primary endpoint, with 100% of patients who completed treatment having an absolute reduction of liver fat content greater than 5%. The average absolute reduction in liver fat content after 12 weeks was 11.2%, which represents an average relative reduction of 67%. Furthermore, NGM was found to have an effect on liver fibrosis, with 28% of participants experiencing a single-stage reduction, and 16% experiencing a two-stage reduction. The drug was found to have a safety profile in line with previous studies carried out on NGM-282 in other indications, and on the most part was well-tolerated, with one participant complaining of mild gastrointestinal issues.

NGM Biopharmaceuticals will be very pleased with these results. With an average absolute reduction in liver fat content of more than double the 5% cut off chosen for the primary endpoint, GlobalData believes that these efficacy results will translate well into the more rigorous study environment of a placebo-controlled Phase IIb trial. Not only has the drug been shown to treat the cause of the disease by reducing liver fat content, it also reduced the symptoms through a marked reduction in fibrosis.

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Despite these strong efficacy data, NGM-282 may struggle to compete amongst a burgeoning NASH pipeline due to its intravenous route of administration, particularly in patients who would require therapeutic treatment in an outpatient setting over an extended period of time. GlobalData therefore anticipates that NGM Biopharmaceuticals could benefit from positioning its drug as a rescue therapy for severe cases of NASH, rather than for longer-term use in mild or moderate cases.

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