Visirna Completes Dosing of the First Patient in China Phase III Study of VSA003 for Treatment of Homozygous Familial Hypercholesterolemia

SHANGHAI, CHINA – December 18th, 2024, Visirna announced that it has dosed the first patient in a randomized, double-blind, placebo-controlled, multi-center Phase III clinical study (CTR20244397), to evaluate the efficacy and safety of VSA003 in Chinese adolescent and adult patients with homozygous familial hypercholesterolemia (HoFH) at Chinese Academy of Medical Sciences, Peking Union Medical College Hospital (PUMCH). Professor Shuyang Zhang, the principal investigator of this clinical study at Peking Union Medical College Hospital (PUMCH) of the Chinese Academy of Medical Sciences, emphasized the pressing need for effective therapies in homozygous familial hypercholesterolemia (HoFH) management. She remarked, "HoFH patients often contend with LDL-C levels significantly higher than the general population, and current treatments offer limited lipid-lowering effectiveness. The inability to adequately control LDL-C leaves many patients vulnerable to cardiovascular events. VSA003 presents a promising solution to overcome these therapeutic obstacles. We eagerly anticipate the advancement of this clinical trial and aim to expand treatment options for HoFH patients in China expeditiously." Dr. Xiaoming Zou, CEO of Visirna, underscored the groundbreaking nature of VSA003, an innovative siRNA drug targeting ANGPTL3. He emphasized its ability to effectively reduce LDL-C levels in HoFH patients through both LDLR-independent and LDLR-dependent mechanisms. VSA003's unique mechanism and therapeutic promise led to its designation as a Breakthrough Therapy for HoFH by the CDE earlier this year, potentially positioning it to be the first siRNA therapeutics targeting ANGPTL3 globally. Acknowledging the pivotal role of research institutions, clinical experts, and patients in this milestone, Dr. Zou expressed gratitude for their unwavering support. The successful initiation of the Phase III clinical trial and dosing of the first HoFH patient in China mark significant progress. Visirna remains dedicated to collaborating closely with research institutions and experts to further explore VSA003's therapeutic potential, with the aim of extending its benefits to more Chinese patients grappling with dyslipidemia, leveraging the unique advantages of siRNA drugs. Homozygous familial hypercholesterolemia (HoFH) represents a rare autosomal dominant genetic disorder characterized by markedly elevated LDL-C levels from birth, substantially heightening the susceptibility to atherosclerotic cardiovascular disease (ASCVD). The progression of systemic ASCVD in HoFH patients is swift and early, often resulting in angina or myocardial infarction and ultimately premature mortality between the ages of 20 and 30. This condition poses considerable risks of fatality and incapacitation, profoundly impacting the lives and well-being of affected individuals. The prevalence of HoFH is estimated to range from approximately 1 in 300,000 to 1 in 160,000 and has been officially recognized in China's "First Catalog of Rare Diseases." <br/><br/> When statins prove ineffective in attaining LDL-C lowering goals, non-statin lipid-lowering medications like cholesterol absorption inhibitors (e.g., ezetimibe) or proprotein convertase subtilisin/kexin type 9 inhibitors (PCSK9 inhibitors) may be combined for treatment. Studies suggest that around 85-90% of individuals with homozygous familial hypercholesterolemia (HoFH) experience exceptionally elevated LDL-C levels due to the loss or dysfunction of LDLR. The mechanisms of current lipid-lowering therapies predominantly rely on LDLR function, leading to constrained effectiveness and safety profiles in HoFH patients. VSA003 is a siRNA drug specifically targeting ANGPTL3 mRNA in liver cells, effectively reducing ANGPTL3 protein production. This leads to decreased levels of ANGPTL3 protein within the liver and circulation, thereby: 1. Inhibiting the secretion of triglyceride-rich lipoproteins (TRL), including VLDL (an upstream substance in LDL synthesis), from the liver into the bloodstream, consequently lowering blood LDL-C and TG levels. 2. Enhancing lipoprotein lipase (LPL) activity, increasing the hydrolysis of TG/TRL in peripheral tissues (such as fat and muscle), thereby reducing blood TG/TRL levels. 3. Enhancing liver cell uptake and clearance of LDL primarily through LDL receptor (LDLR) non-dependent/endothelial lipase (EL)-dependent pathways, as well as LDLR-dependent/EL non-dependent pathways, thus lowering levels of key lipid indicators including LDL-C, VLDL-C, and TG. The target action of VSA003, ANGPTL3, differs from the targets of statins, PCSK9 inhibitors, cholesterol absorption inhibitors, antioxidants, bile acid sequestrants, niacin, fibrates, and other drugs recommended in the "Chinese Blood Lipid Management Guidelines (2023)." Visirna was founded in 2022 in a strategic partnership with Arrowhead Pharmaceuticals (NASDAQ: ARWR). 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 three clinical stage siRNA candidates with a focus on cardiovascular and metabolic ailments. By effectively integrating internal expertise with external resources, Visirna has cultivated a robust suite of industry capabilities spanning drug discovery, clinical advancement, domestic manufacturing, and commercialization. For more information, please visit www. visirna.com

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