Visirna Therapeutics, a Shanghai-based biopharmaceutical company, has announced the completion of patient enrollment in a Phase III clinical study for its investigational drug VSA001, which is being studied for the treatment of familial chylomicronemia syndrome (FCS) in China. A total of 36 FCS patients have been enrolled and will be randomly assigned to receive subcutaneous injections of 25mg VSA001, 50mg VSA001, or a placebo every three months.
The primary endpoint of the trial is the percentage change in abdominal serum triglycerides (TG) compared to baseline at the 10th month of treatment. Following the completion of the randomized double-blind study, patients may opt to continue in an open-label study for long-term efficacy and safety observation and follow-up.
FCS is a rare disease that significantly impacts patients’ health and can be life-threatening. Patients experience recurrent acute pancreatitis from adolescence, with a significantly increased risk of cardiovascular disease and diabetes. Despite strict dietary management and medication, triglyceride levels cannot be effectively controlled, necessitating safer and more effective drugs to lower TG levels in clinical practice.
VSA001 is a synthetic, double-stranded, hepatocyte-targeted RNA interference (RNAi) trigger designed to specifically silence messenger RNA (mRNA) transcripts from the APOC3 gene using an RNAi mechanism. Given APOC3’s role in serum TG level modulation and its primary synthesis in hepatocytes, reducing APOC3 through a hepatocyte-targeted RNAi strategy is expected to lower circulating TG, benefiting patient populations, including those with FCS.
Early research data for VSA001 indicates sustained, stable, and significant TG-lowering effects, as well as good safety, suggesting great therapeutic potential and value for FCS. The unique therapeutic advantages of small nucleic acid drugs, such as long-lasting efficacy and long administration intervals, are anticipated to enhance medication convenience and compliance for chronic disease patients, making VSA001 highly suitable for the treatment and management of chronic diseases, including high triglyceridemia and FCS.- Flcube.com
