Visirna's First-in-Class APOC3 siRNA Therapy VSA001 (Plozasiran) Achieves Positive Phase III Results in China with Once-Every-Three-Months Dosing
Visirna
siRNA Drug Developer
On March 17, Visirna announced,VSA001 Injection（Pulersilan Sodium）InChinese Familial Chylomicronemia Syndrome（FCS）Phase III Clinical Trial of the Patient（CTR20231418/NCT05902598）Positive topline data achieved, successfully reaching the primary efficacy endpoint and all key secondary endpoints.
Screenshot source: Official WeChat account of the company
This is a randomized, double-blind, placebo-controlled, multi-center Phase III clinical trial, with a total of 37 FCS patients randomly assigned to receiveOnce every 3 monthsA 12-month treatment with VSA001 25 mg, 50 mg, or placebo subcutaneous injections aims to evaluate the efficacy and safety of VSA001 in Chinese adult patients with FCS.The primary endpoint of the study was compared with the placebo group,The median percentage change in fasting serum triglyceride levels from baseline at Month 10.
Data shows,Fasting serum triglyceride levels in FCS patients treated with 25 mg and 50 mg VSA001 at Month 10 compared to baselineThe reductions reached 86% and 89%, respectively.。Fasting serum triglyceride levels in FCS patients treated with 25 mg and 50 mg VSA001 at Month 12 compared to baselineThe reductions reached 72% and 79%, respectively.。
In the VSA001 25 mg treatment group,90% of FCS patients had fasting serum triglyceride levels drop below 500 mg/dL by the 10th month.。Serum from FCS patients treated with 25 mg and 50 mg VSA001 in the 10th monthThe median reduction of APOC3 compared to baseline reached 93% and 92%, respectively.。
In addition to successfully achieving the primary endpoint of the study, VSA001 alsoAchieved all key secondary endpoints, including the percentage change in serum fasting triglycerides from baseline at Month 10 and Month 12, as well as the percentage change in APOC3 levels from baseline at Month 10 and Month 12.
VSA001 Demonstrates Good Safety in FCS Patients. Experiencing Treatment-Related Adverse Events (TEAEs) The number of patients was similar between the VSA001 treatment group and the placebo group, while the incidence of severe adverse events was lower in the VSA001 treatment group than in the placebo group.
VSA001 is aFirst-in-classRNA Interference（RNAi）Therapeutic drugs, aimed at reducingApolipoprotein C3（APOC3）The mRNA level was targeted to reduce the expression of the APOC3 protein, thereby effectively lowering serum triglycerides and triglyceride-rich lipoprotein levels through both lipoprotein lipase-dependent and -independent pathways.
The drug is currently being studied in a series of trials within the SUMMIT clinical program, including the PALISADE Phase III study for patients with FCS,Hypertriglyceridemia（SHTG）The patient's SHASTA study, as well as forMixed HyperlipidemiaThe MUIR and CAPITAN Studies in Patients.
Insight database shows that, in the APOC3 siRNA track,The progress of VSA001 is far ahead.In November 2024, Visirna's partner Arrowhead Pharmaceuticals has submitted to the U.S. FDAVSA001 NDA for the treatment of FCS,PDUFA Action Date is November 18, 2025. In January 2025, the product inChina Report Goes Public。
In addition, four similar products have entered the clinical stage.(Only positive states are counted), more than 70% are produced in China, all in the early clinical stage.
Screenshot source: Insight database
Cover Source:Company Logo
Disclaimer:This article is for information sharing only and does not represent the position or viewpoint of Insight. It does not recommend or introduce any treatment plans. If necessary, please consult and contact正规医疗机构.
Editor：ccai
PR Article Coordination: WeChat insightxb
SubmissionWeChat: insightxb; Email: insight@dxy.cn
