Shanghai-based Visirna Therapeutics announced positive topline results from the Phase III study of plozasiran in Chinese patients with familial chylomicronemia syndrome (FCS). The trial successfully met its primary efficacy endpoint and all key secondary endpoints, marking a significant advancement in the treatment of this rare lipid disorder.
Study Details and Results
The randomized, double-blinded, placebo-controlled, multi-center Phase III study assessed the efficacy and safety of plozasiran in adult patients with FCS. A total of 37 patients were enrolled and randomly assigned to receive subcutaneous injections of plozasiran (25 mg or 50 mg) or placebo every three months for 12 months. The primary endpoint was the median percentage change from baseline in fasting triglycerides at month 10 compared to the placebo group.
Efficacy Outcomes
- At month 10, FCS patients treated with 25 mg and 50 mg of plozasiran achieved reductions from baseline in fasting serum triglyceride levels of 86% and 89%, respectively.
- At month 12, patients in the 25 mg and 50 mg plozasiran groups achieved reductions of 72% and 79%, respectively.
- In the 25 mg treatment group, 90% of FCS patients had fasting triglycerides reduced to below 500 mg/dL by month 10.
- Patients treated with 25 mg and 50 mg of plozasiran at month 10 showed median reductions in serum apolipoprotein C-III (APOC3) levels of 93% and 92%, respectively.
Mechanism of Action
Plozasiran is a first-in-class investigational RNA interference (RNAi) therapeutic designed to reduce the production of apolipoprotein C-III (APOC3). APOC3 is a key regulator of triglyceride metabolism, increasing triglyceride levels by inhibiting the breakdown of triglyceride-rich lipoproteins (TRLs) and their uptake by hepatic receptors. By reducing APOC3 levels, plozasiran aims to lower triglycerides and restore lipid levels to more normal ranges.-Fineline Info & Tech
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