▎WuXi AppTec Content Team Report

On September 8, the official website of the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) announced,Weierzhen Biotechnology (Shanghai) Co., Ltd. (hereinafter referred to as "Visirna") jointly applied forsiRNA DrugsVSA001 InjectionProposed for inclusion in the breakthrough therapy category, this drug can be used forReduce Triglyceride Levels in Adult Patients with Familial Chylomicronemia Syndrome (FCS)。

Screenshot source:CDE Official Website

Public information shows,Visirna, jointly established by Arrowhead Pharmaceuticals and Vivo Capital in April 2022, is a small nucleic acid therapy company.。The company has established a long-term strategic partnership with Arrowhead Pharmaceuticals, a small nucleic acid drug enterprise.Currently, Visirna's product pipeline includes three siRNA drugs in clinical development, targeting the cardiovascular and metabolic disease fields.

VSA001 is a liver-targeted siRNA drug for the treatment of lipid metabolism disorders., which can reduce the expression level of APOC3 protein by effectively and persistently silencing the messenger RNA of apolipoprotein C3 (APOC3 mRNA). Based on both lipoprotein lipase (LPL)-dependent and -independent pathways, this drug significantly reduces serum triglycerides (TG), triglyceride-rich lipoproteins (TRL), and their degradation remnants.In April 2023, the drug was approved for clinical use in China, targeting the indication of:On the basis of a low-fat diet and other lipid-lowering therapies (e.g., fibrates),Reduce triglyceride levels in adult patients with familial chylomicronemia syndrome (FCS).

2023In May, Visirna announced that the first subject dosing of VSA001 injection was successfully completed at Peking University Third Hospital.。ThisIt is a randomized, double-blind, placebo-controlled Phase 1 clinical trial designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VSA001 in healthy adult volunteers in China.In July 2023, Visirna...Visirna announces the first patient dosing in the Phase 3 clinical trial of VSA001 injection successfully completed at the Second Xiangya Hospital of Central South University.This is a randomized, double-blind, placebo-controlled, multi-center Phase 3 clinical trial aimed to evaluate the efficacy and safety of VSA001 in adult patients with familial chylomicronemia syndrome (FCS) in China.。

Visirna VSA001 Injection Phase 3Principal Investigator of the Leading Institution in Clinical TrialsProfessor Li Yong (Department of Cardiovascular Medicine, Huashan Hospital, Fudan University) onceStated in the press release: "FCS is a rare, life-threatening disease characterized by extremely high triglyceride levels, severe postprandial abdominal pain, xanthomas, and more. It can lead to various serious complications, including acute pancreatitis. Conventional lipid-lowering treatments fail to meet the therapeutic needs of patients with this condition.There is still a lack of safe and effective drug treatment options globally, and there are no drugs under research or approved for FCS in China."It is a great honor to lead the first Phase 3 clinical trial of VSA001 in China as the principal investigator. I would like to thank Professor Daoquan Peng (Department of Cardiology, Xiangya Second Hospital, Central South University) and his team for successfully completing the first dosing of an FCS patient in the Phase 3 clinical trial of VSA001 in China. I also look forward to the joint efforts of all clinical research participants and the Visirna team bringing the first small nucleic acid drug targeting this disease to Chinese FCS patients."

ParticipateReferences:
[1]Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of China. Retrieved Sep 09, 2023, from https://www.cde.org.cn/main/xxgk/listpage/9f9c74c73e0f8f56a8bfbc646055026d
[2]Visirna Announces First Patient Dosed in Phase III Clinical Trial of siRNA Innovative Drug VSA001 for Familial Chylomicronemia Syndrome in China. Retrieved July 25, 2023, From https://mp.weixin.qq.com/s/-RBIPyT4Vtdg2UaIoWK0JQ