Ionis Claims Curative Potential for Tryngolza in Severe Triglyceride Disease

Ionis Pharmaceuticals reported that Tryngolza, an antisense oligonucleotide, achieved significant reductions in triglyceride levels and acute pancreatitis events in two large Phase III trials for severe hypertriglyceridemia (sHTG)1 2 3 4.

At 6 months, Tryngolza delivered up to a 72% mean reduction in fasting triglyceride levels compared to placebo, sustained through 12 months of follow-up1 3 4.

Treatment with Tryngolza resulted in an 85% reduction in acute pancreatitis events, which analysts described as transformational and highly statistically significant1 2 3 4 6.

In the trials, 89% of patients on a 50-mg dose saw their triglyceride levels drop below 880 mg/dL (the highest risk threshold for pancreatitis), and 34% fell to below 150 mg/dL, within normal range1.

Ionis plans to file a supplemental submission to the FDA by the end of 2025, seeking label expansion for Tryngolza to include treatment for severe hypertriglyceridemia1 3.

Tryngolza is currently FDA-approved only for familial chylomicronemia syndrome (FCS), a rare genetic disease, but the new data support expansion to a broader patient population1 3 4.

Industry analysts expect Tryngolza could become a 'first-in-class' treatment for sHTG and anticipate a significant commercial impact if FDA approval is secured2 3 7.

Positive trial results led to a substantial increase in Ionis' share price, highlighting market confidence in the drug's potential2 3 4 7.