Date: 16-Sep-2020

Gannex Pharma Files IND Application With US FDA For NASH Drug Candidate ASC42

Gannex Pharma, a wholly owned company of Ascletis Pharma, announced that it has filed investigational new drug application (IND) with US FDA for its non-alcoholic steatohepatitis (NASH) drug candidate ASC42.

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.

Gannex 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.

ASC40 is an oral fatty acid synthase (FASN) inhibitor. In the phase II (FASCINATE-1) randomized, placebo-controlled trial of 99 patients in the USA, clinicians evaluated the safety and efficacy of oral, once-daily dosing of ASC40 (TVB-2640) for 12 weeks. The preliminary data showed that ASC40 (TVB-2640) significantly reduced liver fat, the primary efficacy endpoint of this trial, with a 61% responder rate in the 50 mg group. Participants also showed improvement in markers of liver function and fibrosis. ASC40 (TVB-2640) phase II (FASCINATE-1) data were presented as a Late Breaker at virtual European Association for the Study of the Liver (EASL) International Liver Congress 2020 (ILC) on August 28, 2020.

ASC41 is an oral thyroid hormone receptor beta (THR-beta) agonist which recently 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.

“We are excited about US IND filing of the in-house developed ASC42 that has the potential to be a best-in-class FXR agonist,” said Dr. Handan He, chief scientific officer of Ascletis, “ASC42 IND filing demonstrated again our confidence and capability to develop first-in-class or best-in-class NASH drug candidates through our internal effort.”

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.