What's Happening?
Ionis Pharmaceuticals has reported positive outcomes from its Phase 3 CORE and CORE2 studies on olezarsen, a treatment for severe hypertriglyceridemia (sHTG). The studies demonstrated a significant reduction
in fasting triglyceride levels, achieving up to a 72% placebo-adjusted mean reduction at six months, with sustained results through 12 months. Notably, 86% of patients treated with olezarsen reached triglyceride levels below 500 mg/dL, reducing the risk of acute pancreatitis. The treatment also showed an 85% reduction in acute pancreatitis events, marking a first in sHTG treatment. These findings were presented at the American Heart Association Scientific Sessions and published in The New England Journal of Medicine.
Why It's Important?
The results of the olezarsen studies are significant as they offer a new potential treatment for sHTG, a condition affecting approximately 3 million people in the U.S. This condition is associated with a high risk of acute pancreatitis, a serious and potentially life-threatening condition. Current treatments and lifestyle changes often fail to adequately manage triglyceride levels in sHTG patients. Olezarsen's ability to significantly lower triglyceride levels and reduce pancreatitis events could transform treatment protocols, offering a new hope for patients who are at high risk of severe complications.
What's Next?
Ionis Pharmaceuticals plans to submit a supplemental new drug application for olezarsen to the FDA by the end of the year. An open-label extension study is ongoing, with over 90% of participants from the initial studies opting to continue. If approved, olezarsen could be launched in 2026, marking Ionis's first entry into a broad patient population. The company will host a webcast to discuss the study results, indicating ongoing engagement with the medical community and stakeholders.
