OLEZARSEN


Associated tags: Hypertriglyceridemia, Breakthrough therapy, FCS, RNA, Orphan drug, FDA, Disease, Liver, Ionis, LICA, TGS, American College, Medicine, Annual general meeting, Triglyceride, ACC, Ionis Pharmaceuticals, Fasting, AP, NEJM, Patient, Webcast, IONS, Abdominal pain, Incidence, Pharmaceutical industry

Ionis presents positive results from Phase 3 Balance study of olezarsen for familial chylomicronemia syndrome

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Sunday, April 7, 2024
TGS, AP, Ionis, Incidence, Abdominal pain, IONS, Webcast, Patient, NEJM, Fasting, American College, Ionis Pharmaceuticals, FCS, ACC, Triglyceride, Annual general meeting, Medicine, Pharmaceutical industry

CARLSBAD, Calif., April 7, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced full results from the Phase 3 Balance study of Ionis' lead independent investigational medicine, olezarsen, for the treatment of adults with familial chylomicronemia syndrome (FCS). The olezarsen 80 mg monthly dose met the primary endpoint of significantly reducing triglycerides (TGs) in patients with genetically validated FCS at six months. In addition, olezarsen demonstrated robust and sustained reductions in TGs and serum apolipoprotein C-III (apoC-III) levels. Importantly, olezarsen reduced the incidence of acute pancreatitis (AP) events over the 12-month treatment period compared to placebo. Olezarsen also demonstrated a favorable safety and tolerability profile. These results 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). Based on these data, Ionis is pursuing regulatory approval of olezarsen as a potential breakthrough treatment for adults with FCS.

Key Points: 
  • Based on these data, Ionis is pursuing regulatory approval of olezarsen as a potential breakthrough treatment for adults with FCS.
  • In the study, patients were treated with olezarsen 80 mg (n=22), 50 mg (n=21) or placebo (n=23) once every four weeks.
  • Serious AEs occurred in 14% of patients treated with olezarsen 80 mg, 19% treated with olezarsen 50 mg, and 39% treated with placebo.
  • Ionis will host a webcast to discuss the detailed results from the Balance study on Monday, April 8 at 10:00 am ET.