In what looks to be a transformative year for potential treatments of nonalcoholic steatohepatitis, the liver disease better known as NASH, another biotech has revealed that its experimental drug appears to improve the condition compared to a placebo.
NGM Biopharmaceuticals (NASDAQ: NGM) revealed preliminary data on Monday showing that 22 percent of patients who received its experimental NASH drug, aldafermin, as part of the last of four cohorts enrolled in its Phase 2 study of the drug met a composite goal of an improvement in liver fibrosis, or scarring, and resolution of NASH after 24 weeks of treatment. Zero percent of patients who received a placebo met the goal.
“It is safe to say the result of this study exceeded our expectations,” CEO David Woodhouse said on a conference call Monday.
Patients who were enrolled in the study had NASH with stage 2 or stage 3 liver scarring. (Stage 1 is minimal scarring; stage 4 is cirrhosis, or severe and irreversible scarring). A total of 53 patients received a 1 mg daily dose of aldafermin; 25, the placebo.
After 24 weeks, 38 percent of patients who received aldafermin saw the scarring improve by at least one stage, compared to 18 percent of the patients who received a placebo, and in 24 percent, NASH was resolved compared to 9 percent of those who got the placebo. Previous cohorts, which weren’t placebo-controlled and tested a range of doses of aldafermin, received 12 weeks of treatment.
NASH is a type of fatty liver disease which, unlike other such conditions, is unrelated to alcohol abuse or medication side effects. The condition, which is closely related to type 2 diabetes and has increased in incidence along with the obesity epidemic, is anticipated to become the leading cause of liver transplants in the US in coming years.
NGM’s NASH drug, an engineered variant of the human hormone FGF19, is designed to be taken once daily by patients. The company said it will present more data from the study in April at the European Association for the Study of the Liver’s meeting in London.
The most common adverse events experienced by patients in the study were diarrhea, headache, abdominal distension, nausea, fatigue, diabetes mellitus, and peripheral edema. Those problems occurred with comparable frequency in the aldafermin and placebo arms, and no patients who received aldafermin withdrew from the study because of side effects.
South San Francisco-based NGM (NASDAQ: NGM) saw its stock price rise about 17 percent to $18.87 per share over Friday’s close.
Aldafermin is NGM’s lead drug candidate. The company was also testing an antibody, which was being developed as a once-monthly injection, as a treatment for NASH and for type 2 diabetes; Merck (NYSE: MRK) exercised its option to license the molecule last year with plans to evaluate it in NASH patients in a Phase 2b study.
With no FDA-approved treatments for NASH, a number of companies are racing to get their experimental therapies across the finish line. SVB Leerink analyst Thomas Smith calls 2020 a “market-defining year” for NASH given the expectation that the FDA will review what could be the first-ever medicine OK’d for the condition.
The most advanced such drugs are by New York-based Intercept Pharmaceuticals (NASDAQ: ICPT) and French firm Genfit (NASDAQ: GNFT). Intercept’s daily pill for NASH, obeticholic acid (Ocaliva)—already FDA-approved to treat a different liver disorder, primary biliary cholangitis—is under review by the agency, with a decision due in late June.
Data from Genfit’s Phase 3 trial of its NASH drug, elafibranor, is anticipated in the second quarter. (The company recently announced it would delay the unblinding of the data, initially anticipated in the first quarter.)
Smith says the NASH therapy market remains nascent, but predicts US and Europe revenues of about $9.6 billion by 2030.