Visirna Announces Breakthrough Therapy Designation Granted for VSA001 for Treatment of Adults with Familial Chylomicronemia Syndrome in China

SHANGHAI, CHINA – September 18th, Visirna today announced that the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) granted Breakthrough Therapy Designation (BTD) for VSA001 for the treatment of adults with familial chylomicronemia syndrome (FCS) in China. FCS is a life-threatening and rare genetic disease that is characterized by extremely high plasma TG levels leading to various clinical disorders, while there are no effective therapies currently approved for FCS in China. VSA001 is an innovative apolipoprotein C3 (APOC3)-targeting RNAi therapeutic that can effectively lower triglyceride (TG) and TG-rich lipoprotein (TRL) levels. Visirna announced the dosage of the first FCS patient in China Phase III clinical trial for VSA001 in July 2023 and is actively recruiting patients. "We are grateful to the NMPA for acknowledging the potential impact of VSA001 on patients with FCS and granting BTD for VSA001," said Dr. Xiaoming Zou, CEO of Visirna. "BTD designation is another important milestone for VSA001 program. We are also excited about the further recognition of the unique clinical advantages of RNAi therapeutics by CDE. We look forward to working closely with regulatory authorities, our principal investigators, and patients to expedite the clinical development of VSA001 in China." The Breakthrough Therapy Designation review policy is designed to facilitate the development and review of novel medicines that are intended for the prevention or treatment of serious, life-threatening diseases or diseases that severely impact the quality of life for which lacks effective treatment, or where sufficient evidence indicates advantages of the novel drug over current treatment options. Drugs granted Breakthrough Therapy Designations receive additional supports from CDE during clinical development and review process, including: 1) Priority communications and guidance from CDE to expedite the drug development process; 2) Complete review of an NDA within 130 days by following a Priority Review pathway; 3) Rolling submission during NDA and prioritized on-site inspection and registration testing. Familial chylomicronemia syndrome 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 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. 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 RNAi therapeutics. We currently have three first-in-class late clinical-stage RNAi candidates licensed from Arrowhead Pharmaceuticals for the treatment of cardiovascular and metabolic diseases.

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