Visirna Announce Positive Topline Results from Phase 3 Clinical Trial of Plozasiran (VSA001) in Chinese Patients in China with Familial Chylomicronemia Syndrome

Suzhou, China, Mar. 17, 2025 —Visirna Therapeutics (Suzhou) Co., Ltd. today announced positive topline results from the Phase 3 clinical trial conducted by Visirna (CTR20231418/NCT05902598) of investigational plozasiran in Chinese patients with familial chylomicronemia syndrome (FCS), a severe and rare genetic disease. The trial successfully met its primary efficacy endpoint and all key secondary endpoints. The randomized, double-blind, placebo-controlled, multicenter Phase 3 trial enrolled 37 patients with FCS, who were randomly assigned to receive subcutaneous injections of plozasiran (25 mg or 50 mg) or placebo every three months for 12 months. The study aimed to evaluate the efficacy and safety of plozasiran in adult patients with FCS in China. The primary endpoint of the study was the median percentage change from baseline in fasting triglycerides at month 10 compared to the placebo group. FCS patients treated with 25 mg and 50 mg of plozasiran at month 10 achieved reductions from baseline in fasting serum triglyceride levels of 86% and 89%, respectively. At month 12, FCS patients treated with 25 mg and 50 mg of plozasiran achieved reductions in fasting serum triglyceride levels of 72% and 79%, respectively. In the plozasiran 25 mg treatment group, 90% of FCS patients had fasting triglycerides reduced to below 500 mg/dL by month 10. FCS patients treated with 25 mg and 50 mg of plozasiran at month 10 showed reductions from baseline in serum apolipoprotein C-III (APOC3) median levels of 93% and 92%, respectively. In addition to successfully achieving the primary endpoint of the study, plozasiran also met all key secondary endpoints, including the percent change in fasting triglycerides at months 10 and 12 from baseline, and the percent change in APOC3 at months 10 and 12 from baseline. Plozasiran demonstrated a favorable safety profile in patients with FCS. The number of patients experiencing treatment-emergent adverse events (TEAEs) was similar between plozasiran and the placebo groups, with severe adverse events being less common in the plozasiran group compared to the placebo group. Familial chylomicronemia syndrome (FCS) is a severe and rare disease often caused by various monogenic mutations. FCS leads to extremely high triglyceride (TG) levels, typically over 880 mg/dL. Such severe elevations can lead to various serious signs and symptoms including acute and potentially fatal pancreatitis, chronic abdominal pain, diabetes, hepatic steatosis, and cognitive issues. Currently, there are limited therapeutic options to adequately treat FCS. Plozasiran (VSA001), previously called ARO-APOC3, is a first-in-class investigational RNA interference (RNAi) therapeutic designed to reduce production of apolipoprotein C-III (APOC3) which is a component of triglyceride rich lipoproteins (TRLs) and a key regulator of triglyceride metabolism. APOC3 increases triglyceride levels in the blood by inhibiting breakdown of TRLs by lipoprotein lipase and uptake of TRL remnants by hepatic receptors in the liver. The goal of treatment with plozasiran is to reduce the level of APOC3, thereby reducing triglycerides and restoring lipids to more normal levels. In multiple clinical studies, investigational plozasiran has demonstrated reductions in triglycerides and multiple atherogenic lipoproteins in patients with familial chylomicronemia syndrome (FCS), severe hypertriglyceridemia (SHTG), and mixed hyperlipidemia. Plozasiran has been generally well tolerated to date with treatment emergent adverse events reported that generally reflect the comorbidities and underlying conditions of the study populations. Across clinical studies and study populations, the most frequently reported treatment emergent adverse events for the 25 mg dose that is proposed for marketing approval were COVID-19, upper respiratory tract infection, headache, Type 2 diabetes mellitus, and abdominal pain. Plozasiran is being investigated in the SUMMIT program of clinical studies, including the PALISADE Phase 3 study in patients with FCS, the SHASTA studies in patients with SHTG, and the MUIR and CAPITAN studies in patients with mixed hyperlipidemia. Plozasiran in the treatment of patients with FCS has been granted Breakthrough Therapy Designation, Orphan Drug Designation, and Fast Track Designation by the U.S. Food and Drug Administration, Orphan Drug Designation by the European Medicines Agency and Breakthrough Therapy Designation, and Priority Review Designation by the China National Medical Products Administration. Investigational plozasiran has not been reviewed or approved to treat any disease. Visirna was founded in 2022 as part of a strategic transaction with Arrowhead Pharmaceuticals. Headquartered in China with a global perspective, Visirna strives to emerge as a frontrunner in the advancement of siRNA therapeutics. The existing product portfolio comprises four clinical stage siRNA candidates with a focus on cardiovascular/metabolic, and auto-immune ailments. By effectively integrating internal expertise with external resources, Visirna has cultivated a robust suite of industry capabilities spanning drug discovery, clinical development, manufacturing, and commercialization. For more information, please visit https://www.visirna.com, or email [email protected]

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