Gannex Announces Clinical Trial Application of ASC42, an FXR Agonist, for Treatment of Primary Biliary Cholangitis Accepted by China NMPA

SHANGHAI,Sept. 6,2021 --Gannex Pharma Co.,Ltd.,a wholly owned company of Ascletis Pharma Inc. (HKEX:1672) announces today that the clinical trial application of ASC42 to treat patients with primary biliary cholangitis (PBC) has been accepted for review by China National Medical Products Administration (NMPA).

ASC42 is an in-house developed,novel non-steroidal,selective,potent FXR agonist with best-in-class potential. On June 16,2021,Gannex announced positive topline results of safety and pharmacodynamic biomarkers from the U.S. Phase I trial of ASC42. The data indicated there was no pruritus observed during 14-day treatment of the once-daily human therapeutic dose of 15 mg and FXR target engagement biomarker FGF19 increased 1632% on Day 14 of treatment with 15 mg dose.

According to the "Recommendations for diagnosis and treatment of primary biliary cholangitis in China (2021)1",PBC is a chronic autoimmune cholestatic disease and frequently progresses to liver fibrosis and cirrhosis requiring liver transplantation or resulting in death. The epidemiology study in 2010 showed that the prevalence of PBC in China was 49.2 cases per 100,000 persons and as high as 155.8 cases per 100,000 in women older than 40 years old,indicating a total of 656,000 PBC patients in China including 440,000 in females over age 40. Ursodeoxycholic acid (UDCA) is the only drug been approved in China with the effect of delaying disease progression. However,approximately 40% PBC patients have an inadequate response to UDCA or are unable to tolerate UDCA.

Therefore,there are significant unmet medical needs to develop new drugs like ASC42 for PBC patients.

About Ascletis

Ascletis is an innovative R&D driven biotech listed on the Hong Kong Stock Exchange (1672.HK). Ascletis is committed to developing and commercializing innovative drugs in the areas of NASH,cancer lipid metabolism and oral checkpoint inhibitors,and viral diseases to address unmet medical needs both in China and globally. Led by a management team with deep expertise and a proven track record,Ascletis has developed into a fully integrated platform covering the entire value chain from discovery and development to manufacturing and commercialization. Ascletis has three marketed products and a robust R&D pipeline of drug candidates.

(1) NASH: Gannex,a wholly-owned company of Ascletis,is dedicated to the R&D and commercialization of new drugs in the field of NASH. Gannex has three clinical stage drug candidates against three different targets – FASN,THR-beta and FXR,and three fixed-dose combinations. (2) Cancer lipid metabolism and oral checkpoint inhibitors: a pipeline of oral inhibitors targeting FASN,which plays a key role in cancer lipid metabolism,and a pipeline of oral PD-L1 small molecule next generation checkpoint inhibitors. (3) Viral diseases: (i) Hepatitis B: focus on breakthrough therapies for CHB functional cure with a subcutaneously-injected PD-L1 antibody – ASC22 and Pegasys® as cornerstone drugs. (ii) Hepatitis C: successfully launched an all-oral regimen of combining ASCLEVIR® and GANOVO® (RDV/DNV regimen). (iii) HIV/AIDS: ASC22,an immune therapy to restore HIV-specific immune responses and eventually lead to a functional cure of HIV-infected patients. For more information,please visit www.ascletis.com.

[1] Source: Chin J Intern Med,August 2021,60(8): 709-715. DOI: 10.3760/cma.j.cn112138-20210520-00360