Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) has recently disclosed the positive results from the phase 3 balance study of olezarsen, their lead independent investigational medicine for the treatment of adults with Familial Chylomicronemia Syndrome (FCS). The study revealed that the olezarsen 80 mg monthly dose significantly reduced triglycerides (TGs) in patients with genetically validated FCS at six months. Furthermore, the medicine demonstrated substantial and clinically meaningful reductions in acute pancreatitis (AP) events over the 12-month treatment period compared to the placebo.
In the 80 mg group, olezarsen met the primary endpoint with a statistically significant placebo-adjusted reduction in TG levels from baseline to six months (44%, p<0.001). The reductions from six to 12 months were sustained, with olezarsen 80 mg achieving a placebo-adjusted 59% reduction in TGs. Additionally, the study revealed that olezarsen-treated patients had markedly fewer AP events during the 12-month period compared to the placebo, with eleven episodes of AP occurring in the placebo group versus one episode in the 80 mg olezarsen group and one episode in the 50 mg group.
Furthermore, the study indicated that olezarsen-treated patients experienced a placebo-adjusted 84% reduction in all-cause hospitalizations between baseline and 12 months. Notably, the medicine exhibited a favorable safety and tolerability profile, with a higher number of treatment-emergent adverse events (TEAEs) in the placebo group.
The global, multicenter, randomized, double-blind, placebo-controlled phase 3 balance study enrolled 66 patients aged 18 and older with confirmed FCS. Patients in the study received background therapies including statins, fibrates, and omega-3 fatty acids.
Olezarsen is designed to inhibit the body’s production of apolipoprotein C-III (ApoC-III), a protein produced in the liver that regulates triglyceride metabolism in the blood. The U.S. FDA granted olezarsen fast track designation for the treatment of FCS in January 2023, as well as orphan drug designation and breakthrough therapy designation in February 2024.
The results of the study were presented in an oral presentation at the 2024 American College of Cardiology (ACC) annual meeting in Atlanta, Georgia, and published simultaneously in the New England Journal of Medicine (NEJM). The company is pursuing regulatory approval of olezarsen as a potential breakthrough treatment for adults with FCS. As a result of these announcements, the company's shares have moved -2.1% on the market, and are now trading at a price of $42.33. Check out the company's full 8-K submission here.
