Visirna Completes Enrollment of Phase 3 Clinical Trial evaluating VSA001 for Treatment of Familial Chylomicronemia Syndrome

Shanghai, China, January 24, 2024 — Visirna Therapeutics (Shanghai) Co., Ltd. ("Visirna") announced the enrollment of over 36 Chinese patients with familial chylomicronemia syndrome (FCS) in the Phase 3 clinical trial of VSA001 injection ("VSA001"), a Class I innovative drug, and anticipates completing the evaluation of all endpoints by the first quarter of 2025 to support subsequent registration review and approval. VSA001 is expected to become the first approved drug for FCS in China. This is a randomized, double-blind, placebo-controlled, multi-center Phase3 clinical trial (CTR20231418/NCT05902598) aimed to evaluate the effectiveness and safety of VSA001 in Chinese adults with FCS. The participants received subcutaneous injections randomized to placebo or VSA001 at 25mg or, 50mg every 3 months. The primary endpoint is the percentage change from baseline in fasting triglyceride (TG) levels at the end of 10 months. After the randomized phase, patients can continue participating in an open-label extension study for long-term observation of the effectiveness and safety of the drug. Prof. Li Yong, the leading Principal Investigator (PI) of the study (Department of Cardiology, Huashan Hospital, Fudan University) said: "FCS is a potentially life-threatening rare disease posing significant clinical burdens on patients. Individuals diagnosed with familial chylomicronemia syndrome (FCS) often experience recurrent acute pancreatitis at a young age, which significantly increases their risks of cardiovascular events and diabetes. Despite strict dietary management and medication, effectively controlling triglyceride levels in these patients remains challenging. This highlights the pressing clinical need for safer and more effective triglyceride-lowering drugs. VSA001 had demonstrated remarkable efficacy and durability in lowering TG while maintaining favorable safety profile. This underscore its immerse therapeutic potential and value for FCS. Notably, the unique advantages of prolonged duration of action hence longer dosing intervals of siRNAs greatly improves patients’ compliance with chronic conditions. We are delighted to announce the completion of patient enrollment for the Phase 3 clinical trial of VSA001, made possible by the collaborative efforts and support of researchers, research centers, and Visirna. This milestone marks a significant step forward in the treatment and management of chronic diseases like hypertriglyceridemia, including FCS. "The absence of approved drugs for FCS has significantly impacted the quality of life and life expectancy of affected patients. VSA001, being the first drug expected to receive approval for FCS in China, holds great promise for these patients. Notably, VSA001 has been recognized as a breakthrough therapy by the Center for Drug Evaluation (CDE), further emphasizing its potential and value for FCS treatment." said Dr. Xiaoming Zou, CEO of Visirna. "The completion of patient enrollment for the Phase 3 clinical trial in China within a short timeframe represents a significant milestone in Visirna's clinical development efforts. It instills confidence in the timely availability of an approved therapeutic agent for patients with FCS. Visirna remains committed to collaborating closely with research institutions and clinical experts throughout the study, supporting the drug's New Drug Application (NDA) submission in China. I would like to extend sincere gratitude to all the patients and researchers." 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. FCS is a severe rare genetic disease, with an estimated prevalence of approximately 1/1,000,000. It is typically caused by mutations or compound/heterozygous mutations in multiple single genes (e.g., <i>LPL, GPIHBP1, APOC2, APOA5</i>, or <i>LMF1</i>). FCS usually leads to extremely elevated fasting TG levels (above 880 mg/dL). Severe TG elevation can cause various clinical diseases and severe complications, including acute pancreatitis, atherosclerosis, type 2 diabetes, obesity, fatty liver, and chronic kidney disease. Currently, there are no approved drugs specifically for 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 leader in the research and development of siRNA therapeutics. The current product pipeline includes three siRNA programs in clinical development targeting cardiovascular and metabolic diseases. Through the comprehensive integration of internal and external resources, Visirna has established a full industry chain capabilities covering drug discovery, clinical development, local production, and commercialization. For more information, please visit www.visirna.com.

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