Visirna Completes First Patient Dosing in Phase III Trial of VSA003 for Homozygous Familial Hypercholesterolemia in China

Dec 19, 2024 11:57 CST Updated 11:57

Visirna

siRNA Drug Developer

On December 18, 2024, Weierzhen Biotechnology (Shanghai) Co., Ltd. (hereinafter referred to as "Weierzhen" or "Visirna") announced that the first patient dosing in the Phase III clinical trial of its investigational new drug VSA003 Injection (hereinafter referred to as VSA003) was successfully completed 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).

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Professor Zhang Shuyang, the principal investigator of the leading institution in this clinical trial (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 the general population. The lipid-lowering efficacy of existing treatments is relatively limited in HoFH patients, and most patients still cannot achieve effective control of LDL-C levels to prevent cardiovascular events. VSA003 is expected to break through the therapeutic bottleneck of current drugs. We look forward to the smooth progress of this clinical trial and hope it can soon provide more treatment options for HoFH patients in China."

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Visirna CEO—Dr. Zou Xiaoming

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 view 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, with the potential 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 the Phase III clinical trial of VSA003 in China and the completion of the first dosing in an HoFH patient. Visirna will continue to work closely with research institutions and clinical experts to further explore the therapeutic potential of VSA003, allowing more Chinese patients with dyslipidemia to 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). This can lead to angina or myocardial infarction 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 vast 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 existing lipid-lowering drugs are mostly dependent on LDLR, thus their lipid-lowering efficacy in HoFH patients often falls short of expectations, showing significant clinical limitations.

About VSA003

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, achieves the following: ① Inhibiting the secretion of TRL, including VLDL (the upstream substance of LDL), from the liver into the bloodstream, thereby reducing blood LDL-C and TG levels; ② Enhancing LPL activity, increasing the hydrolysis of TG/TRL in peripheral tissues (such as fat and muscle), and lowering 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, thereby 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)." VSA003 can reduce LDL-C through an LDLR-independent pathway.

About Visirna

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 established in 2022 and 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 nucleic acid drugs in clinical development stages, targeting cardiovascular and metabolic diseases.The company's existing pipeline is in a leading position among similar competing products. Its research targets are supported by clear genomic and biological evidence, and it utilizes Arrowhead Pharmaceuticals' proven chemical modification and delivery technology platform. The company adopts a China-US collaborative clinical development and registration strategy, which helps accelerate the registration and market launch of its products under research. At the same time, through the comprehensive integration of internal and external resources, it forms an entire industry chain layout from early-stage research, clinical research, localized production to commercialization.

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SoundMing: The content is sourced from the company's official release. This articleFor the purpose of knowledge sharing, all content is for reference only and does not constitute any advice.