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On August 19, 2024, Visirna announced that the application for the international multi-center clinical trial of its investigational Class 1 new drug targeting APOC3 small nucleic acid drug – VSA001 Injection (hereinafter referred to as VSA001) has been approved by the National Medical Products Administration. A Phase III clinical trial will be conducted in China to evaluate the safety, efficacy, and pharmacokinetics of VSA001 in adult patients with severe hypertriglyceridemia (hereinafter referred to as "SHTG"). Visirna's partner, Arrowhead Pharmaceuticals, has already initiated this international multi-center clinical trial globally, and Visirna will soon join the study in China.VSA001 is a liver-targeted small interfering RNA (siRNA) drug that effectively and persistently silences the mRNA level of apolipoprotein C3 (APOC3) to reduce the expression of APOC3 protein. It then effectively lowers serum triglycerides (TG) and triglyceride-rich lipoproteins (TG-rich lipoprotein, TRL) and their degradation remnants through both lipoprotein lipase (LPL)-dependent and -independent dual pathways.Dr. Xiaoming Zou, CEO of Visirna, stated"Severe hypertriglyceridemia (SHTG) is a common condition where patients' triglyceride levels can reach more than three times the normal level. Patients with SHTG typically face extremely high risks of acute pancreatitis and cardiovascular diseases, significantly impacting their quality of life. Moreover, dietary adjustments and existing triglyceride-lowering medications have limited efficacy in treating SHTG, creating an urgent need for safer and more effective therapeutic options. The approval of this international multicenter clinical trial accelerates the commercialization process of VSA001 globally and in China, bringing new treatment options to Chinese patients with SHTG as soon as possible."Visirna is a small nucleic acid drug therapy company based in China with a global outlook, aiming to build a biopharmaceutical enterprise with comprehensive capabilities ranging from research and development, production to commercialization. Established in 2022, Visirna has formed a long-term strategic partnership with Arrowhead Pharmaceuticals (NASDAQ: ARWR), an internationally leading small nucleic acid drug company. Currently, the company’s product pipeline includes three small interfering RNA drugs targeting cardiovascular and metabolic diseases at the clinical development stage. The pipeline holds a leading position among similar competing products and adopts a U.S.-China coordinated clinical development and registration strategy to accelerate the registration and market launch of its products under research. At the same time, through the comprehensive integration of internal and external resources, the company has established an entire industry chain layout covering early-stage research and development, clinical development, localized siRNA drug production, and commercialization.Source of the article:Visirna
