-- ASC42 has completed Phase I trials in the U.S. and China. This approval from U.S. FDA enables Gannex to complete a critical drug-drug interaction (DDI) study to support upcoming Phase III trials in China, the U.S. and European Union       

-- Gannex expects to complete this DDI study at the beginning of Q4, 2022

-- Currently, Phase II clinical trial of FXR agonist ASC 42 in China is in progress. Gannex intends to start Phase III clinical trials in China, the U.S. and European Union after the completion of the Phase II clinical trial which is ongoing in China

Shanghai, China, June 8, 2022-- Gannex, a wholly owned company of Ascletis Pharma Inc. (HKEX: 1672) today announces that it has obtained the U.S. FDA clearance for its drug candidate, ASC42, to initiate a drug-drug interaction (DDI) study. This important DDI study is designed to provide more evidence to support upcoming Phase III clinical trials in China, the U.S. and European Union for treatment of primary biliary cholangitis (PBC).

ASC42 is an in-house developed, novel non-steroidal, selective, potent Farnesoid X receptor (FXR) agonist with best-in-class potential and global intellectual property. Previous Phase I clinical trial in the U.S. (ClinicalTrials.gov Identifier: NCT04679129) demonstrated that ASC42 might be a potentially best-in-class PBC drug candidate as low density lipoprotein cholesterol (LDL-C) levels were in normal range with no pruritus occurrence, and FXR target engagement biomarker FGF19 increased 1,780% when ASC42 was dosed at 15 mg, once daily (QD) during the 14-day treatment. Currently, Phase II clinical trial of FXR agonist ASC 42 in China is in progress. Gannex intends to start a Phase III trial in China, the U.S. and European Union once the ongoing Phase II clinical trial of ASC42 for PBC in China is completed.

PBC is a chronic autoimmune cholestatic disease and frequently progresses to liver fibrosis and cirrhosis requiring liver transplantation or resulting in death. An epidemiology study indicates that there were approximately 120,000 PBC patients in the U.S. in 2014^[1]. Ursodeoxycholic acid (UDCA) is the standard treatment for PBC, however, approximately 40% PBC patients have an inadequate response to or are unable to tolerate UDCA^[2]. For those patients with insufficient UDCA response or intolerance, Obeticholic Acid (OCA) is the only approved medicine in the U.S. while it has not been approved in China yet. Additionally, OCA may significantly cause pruritus and LDL-C levels to rise.

“I'm very excited that we will soon start a drug-drug interaction study on FXR Agonist ASC42 to support upcoming Phase III trials in China, the U.S. and European Union for PBC indication. Together with other 6 NASH/PBC drug candidates, Gannex reaffirms the commitment to address the huge unmet medical needs through bringing more innovative treatment for patients globally.” said Dr. Jinzi J. Wu, Founder, Chairman and CEO of Ascletis.

[1] Lu M, Zhou Y, Haller I V, et al. Increasing Prevalence of Primary Biliary Cholangitis and Reduced Mortality With Treatment [J]. Clin Gastroenterol Hepatol 2018, 16(8): 1342-50 e1. DOI: 10.1016/j.cgh.2017.12.033.

[2] Lindor K D, Bowlus C L, Boyer J, et al. Primary Biliary Cholangitis: 2018 Practice Guidance from the American Association for the Study of Liver Diseases [J]. Hepatology 2019, 69(1): 394-419. DOI: 10.1002/hep.30145.