Visirna Announces IND Approval from China CDE for VSA006 for Treatment of NASH

SHANGHAI, CHINA – September 26th, Visirna announced that the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) approved Investigational New Drug (IND) for the initiation of a phase 2 clinical trial of VSA006 for the treatment of adults with non-alcoholic steatohepatitis (NASH) in China. VSA006 is the third RNAi therapeutic developed by Visirna receiving IND approval for clinical trials in China. VSA006 is an investigational RNAi therapeutic targeting hydroxysteroid 17-beta dehydrogenase 13 (HSD17β13). The safety, tolerability, and pharmacodynamics of VSA006 were evaluated in a phase 1/2 study with healthy volunteer and patients with confirmed/clinically suspected NASH, including patients recruited from Hong Kong. The upcoming phase 2 clinical trial is designed as a randomized, double-blind, placebo-controlled to evaluate the safety and efficacy of VSA006 in patients with NASH in China. “Visirna has advanced three first-in-class RNAi therapeutics to clinical trials in China, building up a leading pipeline in cardiovascular and metabolic disease areas since company’s inception in 2022”, said Dr. Xiaoming Zou, CEO of Visirna. “Visirna also locally manufactured VSA006 drug product to support China phase 2 clinical trial. It is an important milestone for Visirna in establishing local manufacturing capabilities for RNAi therapeutics. We look forward to working closely with investigators and providing potential treatment options for patients with NASH.” Non-alcoholic steatohepatitis (NASH) is a highly prevalent chronic liver disease. It is characterized by chronic and progressive liver pathology and can cause advanced fibrosis, cirrhosis, hepatocellular carcinoma, end-stage liver disease, and liver-related death. Due to hampered drug discovery for NASH by the limited understanding of the disease mechanism, there are currently no approved medical therapies for NASH. VSA006 is an investigational RNAi therapeutic specially targeting hydroxysteroid 17-beta dehydrogenase 13 (HSD17β13), a member of the hydroxysteroid dehydrogenase family involved in the metabolism of hormones, fatty acids, and bile acids. Truncated HSD17β13 has been associated with reduced risk of non-alcoholic fatty liver disease (NAFLD) and NASH from published human genetics studies. In the completed phase 1/2 clinical trial, the safety, tolerability, and pharmacodynamics of VSA006 were evaluated in normal healthy volunteers and patients with confirmed or clinically suspected NASH. VSA006 treatment was well tolerated with no treatment-related serious adverse events or drug discontinuations. This proof-of-concept study also demonstrated that short-term treatment with VSA006 reduces hepatic HSD17β13 mRNA and protein expression, which is accompanied by reductions in alanine aminotransferase. Visirna was founded in 2022 in a strategic partnership with Arrowhead Pharmaceuticals (NASDAQ: ARWR). Based in China, with a global vision, Visirna aims to be a leading player in the research and development of RNAi therapeutics. Visirna currently owns three first-in-class late clinical-stage RNAi candidates licensed from Arrowhead Pharmaceuticals for the treatment of cardiovascular and metabolic diseases.

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