Ionis Posts Strong Results for Tryngolza in Severe Blood Fat Disease

Ionis Pharmaceuticals announced positive results from its Phase III Essence study of Tryngolza (olezarsen) in people with moderate to severe hypertriglyceridemia, a condition marked by high levels of triglycerides in the blood12.

Tryngolza achieved a 61% reduction in triglyceride levels at the 80 mg dose and 58% at the 50 mg dose over six months compared to placebo, with most treated participants' levels returning to normal (<150 mg/dL)12.

The drug met both its primary and key secondary endpoints, including lowering triglycerides and other lipid parameters while maintaining a favorable safety profile12.

Tryngolza was recently approved by the FDA for familial chylomicronemia syndrome (FCS), a rare genetic disorder with extremely high triglycerides, marking Ionis’ first independent drug launch24.

Ionis plans to seek expanded FDA approval for severe hypertriglyceridemia based on these results, potentially broadening use to a multi-billion dollar market34.

Analysts have responded positively, with Ionis shares rising sharply and projections of blockbuster sales if approved for broader indications34.

Sources:

1. https://www.clinicaltrialsarena.com/news/ionis-olezarsen-hypertriglyceridemia/

2. https://www.nasdaq.com/articles/ionis-tryngolza-cuts-triglyceride-levels-late-stage-study

3. https://www.biopharmadive.com/news/ionis-tryngolza-triglycerides-core-trial-results/758987/

4. https://www.statnews.com/2024/12/19/ionis-fda-approval-tryngolza-familial-chylomicronemia-syndrome-triglycerides/