Gannex Pharmaceutical Co Ltd said on Sunday that it has submitted an investigational new drug application (IND) to US FDA for its non-alcoholic steatohepatitis (NASH) drug candidate ASC42.

NASH is the progressive form of non-alcoholic fatty liver disease (NAFLD), which is characterized by the accumulation of fat in the liver, inflammation and fibrosis (scarring), and can eventually lead to cirrhosis and liver failure. NASH is a major cause of liver disease worldwide and the leading cause of liver transplants for people under 50 in the US. There are currently no approved treatments for NASH.

According to the company, ASC42 is an in-house developed Farnesoid X Receptor (FXR) agonist. In two NASH animal models, ASC42 demonstrated significant improvements in liver steatosis, inflammation and fibrosis. It has two additional drug candidates at clinical stage in its NASH pipeline, ASC40 and ASC41. ASC42 is expected to be used alone or in combination with ASC40 or ASC41.

Under the company's Phase II randomized, placebo-controlled trial of 99 patients in the USA, clinicians evaluated the safety and efficacy of oral, once-daily dosing of ASC40 for 12 weeks. The ASC40 reduced liver fat, the primary efficacy endpoint of this trial, with a 61% responder rate in the 50 mg group. ASC41 is an oral thyroid hormone receptor beta (THR-beta) agonist which received IND approval from China's National Medical Products Administration (NMPA) to conduct clinical trials for NASH indication. Topline data of Phase I safety, PK and preliminary efficacy (LDL-C) in healthy volunteers with LDL-C > 110 mg/dL is expected to be available by the end of 2020.

Gannex Pharma Co Ltd is a wholly owned company of Ascletis Pharma Inc (HKEX:1672), a R&D biotech company that is committed to developing and commercializing innovative drugs of viral hepatitis, NASH and HIV/AIDS, for unmet medical needs in China and globally.