Ionis Pharmaceuticals' Drug Candidate Shows Promise in Rare Genetic Disorder

Key Goals Met in Phase 3 Study

Ionis Pharmaceuticals announced on Tuesday that its olezarsen drug candidate has achieved key goals in a Phase 3 study involving familial chylomicronemia syndrome, a rare genetic disorder. With these positive results, the company is now set to move forward with planned regulatory filings.

Significant Reduction in Triglyceride Levels

The study demonstrated that the olezarsen 80-milligram monthly dose led to a statistically significant reduction in triglyceride levels compared to the placebo. Moreover, this triglyceride-lowering effect continued to improve even at the 12-month mark.

Dramatic Decrease in Acute Pancreatitis Events

Another noteworthy finding was that the olezarsen 80-milligram dose exhibited a 100% reduction in acute pancreatitis events compared to the placebo, which serves as a significant secondary endpoint for the study.

Seeking Regulatory Approval

Ionis Pharmaceuticals has announced its intention to file for approval from both the U.S. Food and Drug Administration and the European Union early next year. The approval of olezarsen would mark a significant milestone as it would become the first available treatment in the U.S. for familial chylomicronemia syndrome, a disorder that can lead to acute, potentially fatal pancreatitis attacks.