Visirna Announces First Patient Dosed in Phase III Clinical Trial of VSA003, a Novel siRNA Therapy for Homozygous Familial Hypercholesterolemia

December 202418DayWeierzhen Biotechnology (Shanghai) Co., Ltd. ("Visirna" or "Weierzhen") announced that the first patient dosing has been successfully completed in the Phase III clinical trial of its investigational new drug VSA003 Injection (hereinafter referred to as VSA003) at Peking Union Medical College Hospital, Chinese Academy of Medical Sciences. This trial is a randomized, double-blind, placebo-controlled, multi-center Phase III clinical trial aimed at evaluating the efficacy and safety of VSA003 Injection in Chinese adolescent and adult patients with homozygous familial hypercholesterolemia (HoFH) (CTR20244397).

The principal investigator of the leading unit of this clinical trial, Professor Zhang Shuyang (Peking Union Medical College Hospital, Chinese Academy of Medical Sciences), stated: "LDL-C levels in HoFH patients are often several times higher than those in normal individuals, and the lipid-lowering efficacy of existing lipid-lowering drugs is relatively limited in HoFH patients. In most patients, LDL-C levels still cannot be effectively controlled to prevent cardiovascular events. VSA003 is expected to break through the therapeutic bottleneck of existing drugs, and we look forward to the smooth progress of this clinical trial.""Provides more treatment options for HoFH patients in China."

Dr. Zou Xiaoming, CEO of Visirna, stated, "VSA003 is an innovative siRNA drug targeting ANGPTL3. This drug can effectively reduce LDL-C levels in HoFH patients through dual lipid-lowering mechanisms, both LDLR-dependent and LDLR-independent. In light of VSA003's novel mechanism and therapeutic potential, it was granted Breakthrough Therapy Designation by the CDE in January this year for the treatment of HoFH. It is expected to become the world’s first approved small nucleic acid drug targeting ANGPTL3. We are grateful for the strong support from research institutions, clinical experts, and patients, enabling the smooth initiation of VSA003’s Phase III clinical trial in China and the completion of the first dosing in an HoFH patient. Visirna will continue to closely collaborate with research institutions and clinical experts to further explore the therapeutic potential of VSA003, benefiting more patients in China."Patients with dyslipidemia benefit from the unique therapeutic advantages of small nucleic acid drugs."

About Homozygous Familial Hypercholesterolemia (HoFH)

HoFH is a severe autosomal recessive genetic disorder caused by homozygous or compound heterozygous mutations in key genes responsible for the catabolism of low-density lipoprotein cholesterol (LDL-C). Patients are exposed to extremely high levels of LDL-C from an early age, significantly increasing the risk of atherosclerotic cardiovascular disease (ASCVD). Angina or myocardial infarction can occur during childhood, adolescence, or adulthood, with notable mortality and disability, posing a serious threat to the patient's life and health. The extreme elevation of LDL-C in the majority of HoFH patients is due to the loss or defect of function in the low-density lipoprotein receptor (LDLR). Moreover, the mechanisms of action of most existing lipid-lowering drugs are LDLR-dependent; therefore, their lipid-lowering efficacy in HoFH patients often falls short of expectations, with clearSignificant clinical limitations.

AboutVSA003

VSA003 is an siRNA drug that specifically targets ANGPTL3 mRNA in hepatocytes, effectively reducing the production of ANGPTL3 protein and lowering the levels of ANGPTL3 protein in the liver and circulation. This, in turn, lowers blood LDL-C and TG levels by: ① Inhibiting the secretion of TRLs, including VLDL (the upstream substance for LDL synthesis), from the liver into the bloodstream; ② Enhancing LPL activity, increasing the hydrolysis of TG/TRL in peripheral tissues (such as fat and muscle), thereby reducing blood TG/TRL levels; ③ Enhancing the uptake and clearance of LDL by hepatocytes, primarily through LDLR-independent/EL-dependent pathways, as well as LDLR-dependent/EL-independent mechanisms for LDL-C clearance, ultimately reducing a series of key lipid indicators including LDL-C, VLDL-C, and TG. The target of VSA003, ANGPTL3, differs from the targets of statins, PCSK9 inhibitors, cholesterol absorption inhibitors, antioxidants, bile acid sequestrants, niacin, and fibrates recommended in the "Chinese Guidelines for Lipid Management (2023)," and can reduce LDL-C via an LDLR-independent pathway.

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 in research and development, production, and commercialization. The company was founded in 2022 and has established a long-term strategic partnership with Arrowhead Pharmaceuticals (NASDAQ: ARWR), an internationally leading small nucleic acid drug company. Currently, the company's pipeline includes three small nucleic acid drugs targeting cardiovascular and metabolic diseases that are in the clinical development stage. The company’s existing pipeline is in a leading position among similar competing products, with its investigational targets supported by clear genomic and biological evidence, utilizing Arrowhead Pharmaceuticals' proven chemical modification and delivery technology platform. The company adopts a U.S.-China collaborative clinical development and registration strategy, which helps accelerate the registration and market launch of its investigational products.