Overview

• In the severe hypertriglyceridemia program, CORE showed 72% and 63% placebo-adjusted triglyceride reductions with 80 mg and 50 mg doses at six months, while CORE2 showed 55% and 49% reductions (all P<0.0001).
• A prespecified pooled analysis across CORE and CORE2 reported an 85% reduction in acute pancreatitis events at 12 months versus placebo (P=0.0002), with adverse and serious adverse events reported as balanced by the company.
• The pivotal severe-hypertriglyceridemia studies enrolled nearly 1,100 participants on background standard-of-care therapy, and more than 90% entered an open-label extension for continued monitoring.
• Separately, the ESSENCE‑TIMI 73b trial in 1,349 patients with moderate hypertriglyceridemia showed placebo-adjusted triglyceride reductions of 58.4 and 60.6 percentage points at six months for 50 mg and 80 mg, with over 80% achieving levels below 150 mg/dL and no significant rise in LDL-C.
• Ionis said it will submit a supplemental new drug application by the end of 2025 to add a severe hypertriglyceridemia indication to Tryngolza, which was approved in December 2024 for familial chylomicronemia syndrome.