Gannex Pharma Co., Ltd. a wholly-owned company of Ascletis Pharma Inc.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 (Chinese Medical Association Rheumatology Branch), "Recommendations for diagnosis and treatment of primary biliary cholangitis in China (2021)" (Primary biliary cholangitis diagnosis and treatment standard (2021)) [J]. Zhong Hua Nei Ke Za Zhi. (Chinese Journal of Internal Medicine), 20 21, 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.

 

Source:gannexpharma.com

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