Visirna Completes Dosing of the First Patient in China Phase II Study of VSA006 for Treatment of Non-Alcoholic Steatohepatitis (NASH)

SHANGHAI, CHINA – April 26, 2024, Visirna announced that it has dosed the first patient in a Phase II, randomized, double-blind, placebo-controlled, and multi-center clinical study (CTR20233245), to evaluate the efficacy and safety of VSA006 in adults with non-alcoholic steatohepatitis (NASH) at the First Affiliated Hospital of Wenzhou Medical University. Professor Lai Wei, M.D., Leading Principal Investigator at Beijing Tsinghua Changgung Hospital stated, "NASH is a severe form of metabolic-related fatty liver disease that can lead to end-stage liver diseases such as cirrhosis and hepatocellular carcinoma, posing serious threats to patients' health and increasing the global burden of liver diseases. However, there are very few drug options available for NASH treatment currently, so it is encouraging to see the successful dosing of the first patient in the Phase II clinical trial of VSA006. VSA006 is an innovative siRNA drug targeting HSD17β13 with novel mechanism of action supported by whole-genome association studies. Combined with the unique therapeutic advantages of siRNA modality, such as long duration of action and dosing intervals, this product has the potential to become a new generation of drug therapy for NASH. As one of the principal investigators of VSA006, I am honored to have the support of clinical experts in advancing this study in China. I would like to thank Professor Minghua Zheng and his team at the First Affiliated Hospital of Wenzhou Medical University for successfully dosing the first NASH patient in this Phase II clinical trial. We also look forward to rapidly advancing the clinical trials with the joint efforts of all clinical research participants and the Visirna, bringing the first siRNA drug for Chinese NASH patients." Dr. Xiaoming Zou, CEO of Visirna, stated, "With changes in lifestyle and dietary patterns, the prevalence of NASH in China continues to rise, leading to rapidly growing demand for disease treatment. Currently, there are no drugs approved for NASH indication in China. VSA006 is the first siRNA drug in China to enter Phase II clinical trial for NASH indication and is also the most advanced clinical pipeline targeting HSD17β13 in China. We are grateful for the strong support from various clinical sites and experts. The successful completion of the first patient dosing in the Phase II clinical trial of VSA006 is not only another important milestone in Visirna’s clinical development efforts but also a milestone for siRNA drugs in the field of NASH drug development in China. We look forward to the progress of subsequent clinical trials, bringing better drug treatment options to Chinese NASH patients as soon as possible." 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. For more information, please visit www. visirna.com

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