Gilead released results from a Phase II clinical trial of GS-0976 for NASH.
Gilead Sciences released results from a Phase II clinical trial of GS-0976 for nonalcoholic steatohepatitis (NASH).
GS-0976 is an oral inhibitor of Acetyl-CoA caroboxylase (ACC). NASH, sometimes called the “silent liver disease,” resembles alcoholic liver disease, but appears in people who drink little or no alcohol. It can be quite severe and lead to cirrhosis. According to the National Institute of Diabetes and Digestive and Kidney Diseases, NASH affects 2 to 5 percent of people in the U.S. There are currently no specific treatments aside from weight loss, increased physical activity, and avoiding alcohol and unnecessary medications.
The clinical trial included 126 patients. Patients either received 20 mg (49 patients) or 5 mg (51 patients) of the drug, with the remainder (26 patients) receiving a placebo once a day for 12 weeks. All patients had been diagnosed with NASH and liver fibrosis stages F1 through F3 based on a biopsy or magnetic resonance elastography (MRE) and MRI proton density fat fraction (MRI-PDFF). The patients receiving the 20 mg dose showed significant decreases in liver fat content compared to placebo after 12 weeks. They also had a significant decrease in TIMP-1, a blood serum marker associated with liver fibrosis. The differences between placebo and patients receiving 5 mg doses of the drug were not statistically significant.
“In patients with advanced fibrosis, NASH may lead to severe complications including end-stage liver disease, hepatocellular carcinoma and the requirement for liver transplantation,” said Rohit Loomba, lead study author and director of the NAFLD Research Center, Director of Hepatology, Professor of Medicine, and vice chief of the Division of Gastroenterology at the University of California San Diego School of Medicine, in a statement. “Unfortunately, there are no treatments available for these patients. In this first randomized, placebo-controlled, Phase II study of an ACC inhibitor in NASH, the data suggest that GS-0976 has the potential to play an important role in treating patients with this disease.”
Gilead will be presenting the data at The Liver Meeting 2017 held in Washington, DC, in addition to other abstracts related to primary sclerosing cholangitis (PSC).
Because there are no specific treatments for NASH, it is an area a number of companies are turning their attention to. Genfit, for example, projected the worldwide market for NASH treatments to exceed $30 billion. Other analysts believe that’s unrealistic because it assumes that 86 percent of NASH population could be treated, equating the global NASH market size to around $30 to $40 billion.
Because NASH patients have a range of severity, and the advanced fibrosis stage of F3 to F4 is when patients typically note symptoms that make them feel poorly and want treatment, more conservative estimates for addressable NASH patients is about 6 to 10 million people. That would make the global NASH market size at least $10 billion.
Dublin-based Allergan made several strategic acquisitions last year in the NASH market, buying San Francisco-based Tobira Therapeutics for a deal that could hit $1.695 billion. Tobira has two complementary development programs, Cenicriviroc (CVC) and Evogliptins, for NASH. Also in September 2016, Allergan bought Akarna Therapeutics, headquartered in San Diego. Akarna’s lead candidate is also a NASH therapeutic.
Other leaders in the NASH arena include Intercept, Novo Nordisk, Shire, Galmed and Contus.
