Vayazhen's First-in-Class APOC3-Targeting siRNA Drug Plozasiran (VSA001) Nominated for Priority Review in China
Visirna
siRNA Drug Developer
On December 23, Visirna's Class 1 new drug, Plersiran Sodium Injection（VSA001）Proposed for priority review, on the basis of dietary control, for the reduction ofFamilial Chylomicronemia Syndrome（FCS）Adult PatientsTriglyceride Levels, thereby reducing the risk of acute pancreatitis. This indication is currently in the application phase for market approval in the United States. This is also the first-in-class breakthrough for this target.The World's FirstDrugs submitted for marketing approval.
Screenshot source: CDE official website
VSA001 is a liver-targeted small interfering RNA（siRNA）Drug, by efficiently and durably silencing apolipoprotein 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.
Latest Results of the Pivotal Phase III PALISADE Study in FCS Adult Patients Announced at the Cardiovascular and Metabolic Pipeline R&D Webinar on June 25, 2024.
PALISADE Study（NCT05089084）It is a randomized, double-blind, placebo-controlled Phase III clinical trial. A total of 75 subjects were enrolled in the study.(Distributed across different research centers in 18 countries), randomly receiving VSA001 25 mg once every three months(26 cases)、50 mg(24 cases)or placebo(25 cases)Subcutaneous injection treatment for 12 months. Subjects who complete the randomized period are eligible to continue participating in the extension period study.
The primary endpoint of the PALISADE study was the median triglyceride level at the 10th month, adjusted for placebo.（TG）Horizontal changes. The results show,Patients treated with 25 mg or 50 mg VSA001 achieved median triglyceride level reductions of 80% and 78%, respectively.。
In addition to achieving the primary endpoint, VSA001 also met all critical secondary endpoints, including those at Month 10 and Month 12.(Average)Percentage change in fasting triglycerides from baseline; Percentage change in fasting APOC3 from baseline at Month 10; Percentage change in fasting APOC3 from baseline at Month 12; Incidence of acute pancreatitis events during the randomized controlled study. Study data also showed that VSA001, administered once every three months, demonstrated consistent results throughout the study period.Continuously Reduce Triglyceride Levels(Median and Mean), and with low variability.
In terms of safety, VSA001 demonstrated a good safety profile in the PALISADE study. Adverse events（AEs）The number of subjects was similar in the VSA001 and placebo groups. Severe and serious adverse events were less common in the VSA001 group than in the placebo group. The most common adverse events were abdominal pain, COVID-19, nasopharyngitis, headache, and nausea.
Screenshot source: Insight database
FCS is a severe and extremely rare genetic disorder, with an estimated prevalence of approximately 1/1,000,000 according to incomplete statistics, usually caused by various single-gene mutations.(e.g., LPL, GPIHBP1, APOC2, APOA5, or LMF1)Caused by loss-of-function mutations. FCS usually leads to extremely elevated fasting TG levels.(Above 880 mg/dL)，Severe elevation of TG can lead to various clinical diseases and serious complications, including atherosclerosis, acute pancreatitis, type 2 diabetes, and hepatic steatosis, etc.,There are currently no approved effective therapeutic drugs.VSA001 is expected to becomeThe First Effective Approved Drug for FCS。
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