Gannex Announces the Completion of Patient Enrollment for Phase II Clinical Trial of ASC42, an FXR Agonist, for Primary Biliary Cholangitis

-- Gannex is expected to release topline data of the Phase II clinical trial by the end of 2023

SHANGHAI,July 20,2023 -- Gannex Pharma Co.,Ltd. ("Gannex"),a wholly-owned company of Ascletis Pharma Inc. (HKEX:1672) announces today the completion of enrollment of 98 patients with primary biliary cholangitis (PBC) in the Phase II clinical trial of ASC42,a novel Farnesoid X receptor (FXR) agonist.

The 12-week Phase II study (ClinicalTrials.gov Identifier: NCT05190523) consists of three ASC42 active treatment arms (5 mg,10 mg and 15 mg) and one placebo control arm and enrolled a total of 98 patients who have an inadequate response to or are unable to tolerate Ursodeoxycholic acid (UDCA). Topline data are expected to be available by the end of 2023.

ASC42 is an in-house developed,novel non-steroidal,selective,potent FXR agonist with best-in-class potential and global intellectual property. The data from the U.S. Phase I trial of ASC42 indicated that there was no pruritus observed during 14-day treatment of the once-daily human therapeutic dose of 15 mg and FXR target engagement biomarker Fibroblast Growth Factor 19 (FGF19) increased by 1,780% on Day 14 of treatment at the dose of 15 mg. Furthermore,mean low density lipoprotein cholesterol (LDL-C) values remained within the normal range during 14-day,once-daily treatment at the dose of 15 mg.

UDCA is the only drug which is approved in China for PBC and approximately 40% of PBC patients have an inadequate response to or are unable to tolerate UDCA [1]. 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 [2]. Absence of pruritus and mean LDL-C values within the normal range at the therapeutic dose make ASC42 a potential best-in-class PBC drug candidate.

An epidemiology study in China in 2010 showed that there were approximately 656,000 PBC patients in China including 440,000 in females over age 40 [3]. An epidemiology study in the U.S. indicated that there were approximately 120,000 PBC patients in the U.S. in 2014 [4].

Dr. Jinzi J. Wu,Founder,Chairman and CEO of Ascletis said,"We are excited that ASC42 for PBC has completed the patient enrollment for the Phase II clinical trial. We are working very hard to complete this Phase II trial by the end of 2023."

[1] 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.

[2] 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.

[3] 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.

[4] 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.

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 20+ drug candidates in its R&D pipeline. The most advanced drug candidates include ASC10 (RSV infection),ASC22 (CHB functional cure),ASC40 (acne),ASC40 (recurrent glioblastoma),ASC40 (NASH),ASC41 (NASH) and ASC61 (advanced solid tumors).

For more information,please visit www.ascletis.com.