Expanded Access Policy

Gannex Pharma Co.,Ltd. (Gannex),  a wholly-owned company of Ascletis, is dedicated to the R&D and commercialization of drugs in the field of Non-alcoholic steatohepatitis (NASH). Gannex conducts clinical trials in individuals to evaluate investigational medicines in order to obtain information on safety and efficacy that may be used to support marketing approval and subsequent wider accessibility of the product to patients. Investigational medicines are drugs that have not been approved by regulatory authorities.


Gannex has three clinical stage drug candidates against three different NASH targets — Fatty Acid Synthase (FASN), Thyroid Hormone Receptor-Beta (THR-β) and Farnesoid X Receptor (FXR) — and three NASH combination therapies. ASC42 is an in-house developed novel non-steroidal, selective, potent FXR agonist. In two NASH animal models, ASC42 demonstrated significant improvements in liver steatosis, inflammation and fibrosis. ASC42 is a proprietary oral tablet formulation and is stable at room temperature. The compound showed a wide safety margin and is currently in clinical trials. Gannex received investigational new drug application (IND) approval and Fast Track designation for ASC42 from the U.S. Food and Drug Administration (FDA).


Gannex has two additional drug candidates at clinical stage in its NASH pipeline, ASC40 and ASC41. ASC40(TVB-2640), an oral fatty acid synthase (FASN) inhibitor, has been shown to significantly reduce liver fat with a 61% responder rate in the 50 mg group in a Phase II clinical trial (FASCINATE-1). ASC41 is a liver-targeted prodrug and its active metabolite (ASC41-A) is selective for THR-β. ASC42 is expected to be used alone or in combination with ASC40 or ASC41.


Expanded Access, which is sometimes known as “compassionate use”, is a potential pathway for a patient with an immediately life-threatening condition or serious disease to gain access to an investigational therapy for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.  The U.S. FDA advises that Expanded Access may be appropriate when all the following apply:

• Patient has a serious disease or condition, or whose life is immediately threatened by their disease or condition. 

• There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition.

• Patient enrollment in a clinical trial is not possible.

• Potential patient benefit justifies the potential risks of treatment.

• Providing the investigational medical product will not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication.

For more information about expanded access in the U.S., please visit the FDA website at https://www.fda.gov/news-events/public-health-focus/expanded-access.


Gannex seeks to retain the ability to manufacture and supply investigational medicines in a fair and equitable manner and in a volume that assures adequate supply for ongoing clinical trials and development programs. Hence, at this time, Gannex is unable to provide its investigational medicines on an expanded access or right to try basis.  To access our investigational medicines before they are approved by a regulatory authority, patients should participate in one of  Gannex’s clinical trials. To learn more about available clinical trials by Gannex, please visit Gannex Clinical Trials, or visit https://clinicaltrials.gov and search by company, disease or medicine.


If you have additional questions, please speak with your physician or contact Gannex at ea@gannexpharma.com.


Consistent with the 21st Century Cures Act, Gannex may revise this policy at any time.