Visirna's APOC3-Targeting siRNA Drug VSA001 Receives Chinese Approval for Global Phase III Trial in Severe Hypertriglyceridemia

Aug 19, 2024 11:43 CST Updated 11:43

Visirna

siRNA Drug Developer

On August 19, 2024, Weierzhen Biotechnology (Shanghai) Co., Ltd. (hereinafter referred to as "Weierzhen" or "Visirna") recently announced,The international multi-center clinical trial application for Visirna's investigational new drug Class 1, targeting APOC3 small nucleic acid—VSA001 Injection (hereinafter referred to as VSA001)—has been approved by the National Medical Products Administration., conducting a Phase III clinical trial in China to reduce severe hypertriglyceridemia (hereinafter referred to as "SHTG"), aiming to evaluate the safety, efficacy, and pharmacokinetics of VSA001 in adult patients with SHTG. Visirna's partner, Arrowhead Pharmaceuticals, has initiated this international multicenter clinical trial globally, and Visirna will soon join the study in China.

SHTG is a common lipid disorder defined as triglyceride (TG) levels ≥ 500 mg/dl, which may be caused by various factors, including genetics, diet, lifestyle (such as excessive alcohol consumption), and comorbidities like obesity, metabolic syndrome, hypothyroidism, and type 2 diabetes. SHTG can lead to an increased risk of acute pancreatitis, cardiovascular diseases, and atherosclerosis. Recurrent acute pancreatitis may result in chronic pancreatitis and associated pancreatic endocrine dysfunction.

Dr. Xiaoming Zou, CEO of Visirna"Severe hypertriglyceridemia is a common condition where patients have triglyceride levels 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 TG-lowering medications show limited effectiveness 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 SHTG patients as soon as possible."

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 the APOC3 protein. It then effectively lowers serum triglycerides (TG) and TG-rich lipoproteins (TRL) as well as their degradation remnants through both lipoprotein lipase (LPL)-dependent and -independent dual pathways.

AboutVisirna

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 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. The company’s current pipeline includes three small interfering RNA drugs targeting cardiovascular and metabolic diseases, all at the clinical development stage. The pipeline holds a leading position among similar competitive products and adopts a US-China collaborative clinical development and registration strategy to accelerate the registration and market launch of its products under development. 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 R&D, clinical development, localized siRNA drug production, and commercialization.

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