Gannex Published Phase I Data of ASC42, a Novel Farnesoid X Receptor Agonist on the Journal Drugs in R&D

Shanghai, China, November 6, 2023-- Gannex Pharma Co., Ltd. (“Gannex”), a wholly-owned company of Ascletis Pharma Inc. (HKEX:1672) announces today that the safety, pharmacokinetics (PK), and pharmacodynamics (PD) data of ASC42, a novel farnesoid X receptor (FXR) agonist, in healthy subjects have been published on the journal Drugs in R&D.

In-house developed by Gannex, ASC42 is a novel non-steroidal, selective, potent FXR agonist with best-in-class potential and global intellectual property.

This study shows that ASC42 was in general safe and well tolerated when administered as single doses up to 100 mg and multiple daily doses for 14 days up to 15 mg in healthy subjects. ASC42 at therapeutic dose range (5 mg to 15 mg) had an acceptable safety profile and showed no drug‐induced pruritus or transient elevations in serum alanine aminotransferase (ALT), aspartate aminotransferase (AST), or γ-glutamyl transferase (GGT). Furthermore, ASC42 showed effective FXR target engagement in dose‐dependent elevations in Fibroblast Growth Factor 19 (FGF19) and reduction in 7α‐hydroxy‐4‐cholesten‐3‐one (C4), and caused no significant changes in total cholesterol (TC), high density lipoprotein cholesterol (HDL-C), and low density lipoprotein cholesterol (LDL-C) levels in healthy subjects up to 3 weeks following daily dosing for 14 days. Results of this study support the continued investigation of ASC42 in patients with non-alcoholic fatty liver disease (NAFLD)/non-alcoholic steatohepatitis (NASH)/primary biliary cholangitis (PBC).

An epidemiology study showed that there were approximately 656,000 PBC patients in China, including 440,000 in females over age 40 [1]. Ursodeoxycholic acid (UDCA) is the only drug which is approved in China for treatment of PBC and approximately 40% of PBC patients have an inadequate response to or are unable to tolerate UDCA [2]. Obeticholic Acid (OCA), which is not approved in China, is the only approved medicine in the U.S. for PBC patients who have an inadequate response to or are unable to tolerate UDCA. However, there are significantly increased pruritus rates and LDL-C levels in patients with OCA treatment [3].

On July 20, 2023, Gannex announced the completion of patient enrollment for Phase II clinical trial of ASC42 for PBC.

“PBC remains to be a huge unmet clinical need in China and the current treatment for patients is very limited. The Phase II clinical trial of Gannex's in-house developed farnesoid X receptor agonist ASC42 will be completed soon. The topline data of the Phase II clinical trial are expected to be released by the end of 2023.”Said Dr. Jinzi J. Wu, Founder, Chairman and CEO of Ascletis.

[1] Chinese Rheumatology Association (中华医学会风湿病学分会), “Recommendations for diagnosis and treatment of primary biliary cholangitis in China (2021)”(原发性胆汁性胆管炎诊疗规范(2021)) [J]. Zhong Hua Nei Ke Za Zhi. (中华内科杂志), 2021, 60(8): 709-15. DOI: 10.3760/cma.j.cn112138-20210520-00360.

[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.

[3] Nevens, Frederik et al. “A Placebo-Controlled Trial of Obeticholic Acid in Primary Biliary Cholangitis.” The New England journal of medicine vol. 375,7 (2016): 631-43. doi:10.1056/NEJMoa1509840.

About Ascletis

Ascletis is an innovative R&D driven biotech listed on the Hong Kong Stock Exchange (1672.HK), covering the entire value chain from discovery and development to manufacturing and commercialization. Led by a management team with deep expertise and a proven track record, Ascletis focuses on three therapeutic areas with unmet medical needs from a global perspective: viral diseases, non-alcoholic steatohepatitis (NASH) and oncology. Through excellent execution, Ascletis rapidly advances its drug pipeline with an aim of leading in global competition. To date, Ascletis has multiple drug candidates in its R&D pipeline. The most advanced drug candidates include ASC22 (CHB functional cure), ASC40 (acne), ASC40 (recurrent glioblastoma), ASC40 (NASH), ASC41 (NASH) and ASC61 (advanced solid tumors).

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