Gannex, a wholly-owned company of Ascletis，is fully 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 pre-clinical stage combination therapies. 

ASC40, a Global First-in-class, Oral FASN Inhibitor for NASH

FASN: fatty acid synthase  NASH: non-alcoholic steatohepatitis   NAFLD: non-alcoholic fatty liver disease

C. Estes et al., J HEP 2018 (69): 896–904

Efficacy and Safety of ASC40 Treatment in Patients with NASH

Rohit Loomba et al. 2020, Hepatology 72; 103.EASL 2020 Oral Presentation

ASC41, an Oral THR-β Inhibitor for NASH

• Liver-targeted prodrug (ASC41) and active moiety (ASC41-A) is selective for THR-β 

• In two NASH animal models, at 1/10 dose of MGL-3196, ASC41 demonstrated the same improvement in liver steatosis, inflammation and fibrosis 

• A highly potent and selective THR-β agonist with anticipated human efficacious dose <10 mg QD

• Proprietary oral tablet formulation stable at room temperature and whose exposure is same as solution formulation in dogs 

• Topline data of Phase I safety, PK  and preliminary efficacy (LDL-C) in healthy volunteers with LDL-C > 110 mg/dL is expected to be available by the end of 2020 

• Combination opportunities with ASC42 (FXR) and ASC40 (FASN)

Phase I Clinical Trial Results of ASC41

• 65 subjects with elevated low-density lipoprotein cholesterol (LDL-C) (> 110 mg/dL), a population characteristic of non-alcoholic fatty liver disease (NAFLD) 

• In the single-ascending dose portion of the study, preliminary data suggest that ASC41 is safe and well tolerated up to a dose of 20 mg. Furthermore, ASC41 tablet formulation showed a dose-proportional pharmacokinetic profile from 1 mg to 20 mg 

• In the multiple-ascending dose (MAD) portion of the study, preliminary data suggest that after 14 days of once daily oral dosing, subjects demonstrate clinically meaningful and statistically significant reduction in LDL-C and triglycerides compared to placebo, as shown in the table below：

• ASC41 had a benign adverse event profile at all doses following 14-day treatment, with no grade 3 or above adverse events, no serious adverse events or premature discontinuations

• ASC41 tablet formulation displayed a dose-proportional pharmacokinetic profile from 1 mg to 5 mg following once daily, 14-day dosing

ASC42, an Oral FXR Inhibitor for NASH

• A novel non-steroidal, selective, potent FXR agonist 

• U.S. IND approved in October 2020 

• In two NASH animal models, ASC42 demonstrated the significant improvement in liver steatosis, inflammation, and fibrosis 

• An oral tablet formulation developed with proprietary therapy, stable at room temperature 

• Combination opportunities with ASC41 (THR-β) and ASC40 (FASN)

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