Visirna Completes Enrollment of China Phase III Study of VSA003 for Treatment of Homozygous Familial Hypercholesterolemia (HoFH)

Shanghai, China, August 7th, 2025 — Visirna Therapeutics (Suzhou) Co., Ltd. ("Visirna") announced the enrollment of 46 Chinese patients with Homozygous Familial Hypercholesterolemia (HoFH) in the Phase 3 clinical trial of VSA003 injection ("VSA003"), a Class I innovative drug, and anticipates completing the evaluation of double-blind primary endpoint by the first quarter of 2026 to support subsequent registration review and approval. This is a randomized, double-blind, placebo-controlled, multi-center Phase III clinical study, to evaluate the efficacy and safety of VSA003 in Chinese adolescent and adult patients with HoFH (CTR20244397). Professor Shuyang Zhang, the principal investigator of this clinical study at Peking Union Medical College Hospital (PUMCH) of the Chinese Academy of Medical Sciences stated, "Currently available lipid-lowering therapies remain insufficient in effectively controlling LDL-C levels in HoFH patients. There is an urgent need for more effective and safer lipid-lowering drugs to reduce cardiovascular risk in this population. We are pleased that patient enrollment for the Phase III clinical trial of VSA003 has been successfully completed, and we look forward to further follow-up data." Dr. Xiaoming Zou, CEO of Visirna, said “As the second Visirna product to successfully complete Phase III clinical patient enrollment, VSA003 is poised to become the world’s first ANGPT3-tareting siRNA drug near market approval. VSA003 has previously been granted Breakthrough Therapy Designation by the CDE, underscoring regulatory recognition of its therapeutic potential and value in treating HoFH. We sincerely thank all the patients and investigators who participated in this study. Visirna will continue to work closely with clinical site and experts to complete the trial, supporting New Drug Application (NDA) submission in China and offering more treatment options for patients with HoFH.” HoFH (Homozygous Familial Hypercholesterolemia) is a rare autosomal dominant genetic disorder caused by homozygous or compound heterozygous mutations in key genes involved in the metabolic degradation of low-density lipoprotein cholesterol (LDL-C). Patients are exposed to extremely high levels of LDL-C from an early age, significantly increasing their risk of developing atherosclerotic cardiovascular disease (ASCVD). This can lead to angina or myocardial infarction during childhood, adolescence, or adulthood, posing serious threats to life and health due to its high morbidity and mortality. In the vast majority of HoFH patients, the extreme elevation of LDL-C is caused by the absence or dysfunction of low-density lipoprotein receptors (LDLR). Most existing lipid-lowering therapies rely on LDLR-dependent mechanisms, which results in suboptimal efficacy in HoFH patients and presents clear clinical limitations. VSA003 is a first-in-class investigational RNA interference (RNAi) therapeutic designed to reduce production of angiopoietin-like protein (ANGPTL3), which is a hepatocyte expressed regulator of lipid and lipoprotein metabolism with multiple potential modes of action, including inhibition of lipoprotein lipase (LPL) and endothelial lipase (EL). ANGPTL3 is an emerging therapeutic target with relevance to hypercholesterolemia, hypertriglyceridemia, and mixed hyperlipidemia. Genetic studies suggest that individuals with ANGPTL3 loss-of-function variants have enhanced lipoprotein lipase and endothelial lipase activity, resulting in lower levels of atherogenic lipoproteins and a reduced risk of ASCVD. Visirna was founded in 2022 as part of a strategic transaction with Arrowhead Pharmaceuticals. Headquartered in China with a global perspective, Visirna strives to emerge as a frontrunner in the advancement of siRNA therapeutics. The existing product portfolio comprises four clinical stage siRNA candidates with a focus on cardiovascular/metabolic, and auto-immune ailments. By effectively integrating internal expertise with external resources, Visirna has cultivated a robust suite of industry capabilities spanning drug discovery, clinical development, manufacturing, and commercialization. For more information, please visit https://www.visirna.com, or email [email protected]

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