Visirna Announces Dosing of the First Patient in China Phase III Study of VSA001 for Treatment of Familial Chylomicronemia Syndrome

SHANGHAI, CHINA – July 25ᵗʰ, Visirna today announced the dosing of the first patient in a China Phase III, randomized, double-blind, placebo-controlled, and multi-center clinical study (CTR20231390) to evaluate the efficacy and safety of VSA001 in adults with familial chylomicronemia syndrome (FCS). VSA001 is an innovative RNAi therapeutic that targets apolipoprotein C3 (<span class="fi">APOC3</span>). VSA001 can effectively reduce the expression of the <span class="fi">APOC3</span> protein by silencing <span class="fi">APOC3</span> mRNA and lowering triglyceride (TG) and TG-rich lipoprotein (TRL) levels through both lipoprotein lipase (LPL)-dependent and independent pathways. Professor Yong LI, M.D., Leading Principal Investigator at Huashan Hospital of Fudan University, said: “FCS is a life-threatening and rare genetic disease that is characterized by extremely high plasma TG levels leading to various clinical disorders, including atherosclerotic cardiovascular disease, acute pancreatitis, etc. Conventional lipid-lowering therapy cannot meet the treatment needs of patients with this disease. There is still a lack of safe and effective treatment options worldwide, and there is no drug under development or approved for FCS in China. It is my great honor to be the principal investigator of VSA001 to lead the first phase III clinical trial in China. I would like to thank Professor Daoquan PENG (Department of Cardiovascular Medicine, Second Xiangya Hospital of Central South University) and his team for successfully completing dosing of the first FCS patient in the Phase III clinical trial of VSA001 in China. We look forward to the launch of the first siRNA drug for Chinese FCS patients with the joint efforts of all clinical research participants and the Visirna team.” “The successful initiation of the phase III clinical trial of VSA001 in China and dosing of the first patient with FCS is a significant milestone of Visirna as well as a testimony of our team execution. We will continuously commit to advancing siRNA therapeutics in China”. Dr. Xiaoming Zou, CEO of Visirna, said: "VSA001 is the first siRNA drug targeting <span class="fi">APOC3</span> to enter the phase III clinical trial in China. We aim to expedite VSA001’s phase III clinical trial in China through further cooperation with research institutions and clinical experts. We will continue to expand the treatment potential of VSA001 through international multi-center clinical trials, so that more Chinese patients with dyslipidemia can benefit from the unique therapeutic advantages of siRNA drugs." Visirna was founded in 2022 in a strategic partnership with Arrowhead Pharmaceuticals (NASDAQ: ARWR). Based in China, with a global vision, Visirna aims to be a leading player in the research and development of oligonucleotide therapeutics. We currently have three first-in-class late clinical-stage siRNA candidates licensed from Arrowhead Pharmaceuticals for the treatment of cardiovascular and metabolic diseases. Familial chylomicronemia syndrome (FCS) is a severe and ultrarare genetic disease. It affects approximately 1 in 1,000,000 people and is often caused by various monogenic mutations. FCS leads to extremely high fasting triglyceride (TG) levels, typically over 880 mg/dL. Such severe elevations lead to various clinical disorders, including atherosclerotic cardiovascular disease, acute pancreatitis, type 2 diabetes mellitus, hepatic steatosis, etc. Currently, there are limited approved therapeutic options that can adequately treat FCS.

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