Visirna's APOC3 Targeted siRNA - VSA001 Receives IND Approval for Phase III International MRCT in China

(Shanghai, China, August 19th, 2024) Visirna Therapeutics (Shanghai) Co., Ltd. ("Visirna") announced that the National Medical Products Administration has approved the Investigational New Drug (IND) application for an international Multi-Regional Clinical Trial (MRCT) of Visirna’s VSA001 injection, an APOC3 targeted siRNA therapeutic. This Phase III clinical trial will be conducted in China to assess the safety, efficacy, and pharmacokinetics of VSA001 in adult patients with severe hypertriglyceridemia (SHTG). Visirna's partner, Arrowhead Pharmaceuticals, has already initiated this international MRCT globally, and Visirna will soon join the study in China. SHTG is a common lipid disorder, defined by triglyceride (TG) levels ≥ 500 mg/dl, which can be caused by various factors, including genetics, diet, lifestyle (such as excessive alcohol consumption), and comorbidities like obesity, metabolic syndrome, hypothyroidism, and type 2 diabetes [1]. SHTG increases the risk of acute pancreatitis, cardiovascular diseases, and atherosclerosis. Recurrent acute pancreatitis can lead to chronic pancreatitis and related pancreatic endocrine dysfunction [2, 3]. Dr. Xiaoming Zou, CEO of Visirna, stated: “Severe hypertriglyceridemia is a common condition where patients can have triglyceride levels more than three times the normal level. SHTG patients often face extremely high risks of acute pancreatitis and cardiovascular diseases, significantly impacting their quality of life. Dietary adjustments and current TG-lowering medications offer limited efficacy for SHTG, highlighting the urgent need for safer and more effective treatment options. The approval of this international MRCT accelerates the global and Chinese commercialization process of VSA001, bringing new treatment options to SHTG patients in China.” VSA001 is a hepatocyte-targeting small interfering RNA (siRNA) designed to inhibit the expression of apolipoprotein C3 (APOC3) by efficiently and consistently silencing messenger RNA (mRNA) of APOC3. The levels of serum TG, TG-rich lipoprotein (TRL), and TRL remnants are effectively lowered via both lipoprotein lipase (LPL)-independent and LPL-dependent pathways. 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. Visirna currently owns three first-in-class late clinical-stage RNAi candidates licensed from Arrowhead Pharmaceuticals for the treatment of cardiovascular and metabolic diseases. For more information, please visit www. visirna.com 1. Ulrich Laufs, et al., Clinical review on triglycerides. Eur Heart J. 2020 Jan 1;41(1):99-109c. 2. Robert A Hegele, et al., The polygenic nature of hypertriglyceridaemia: implications for definition, diagnosis, and management. Lancet Diabetes Endocrinol. 2014 Aug;2(8):655-66. 3. John Scherer et al., Issues in hypertriglyceridemic pancreatitis: an update. J Clin Gastroenterol. 2014 Mar;48(3):195-203.

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