The U.S. Food and Drug Administration (FDA) approved on Wednesday Ionis Pharmaceuticals, Inc.’s (NASDAQ:IONS) Tryngolza (olezarsen) to treat severe hypertriglyceridemia.
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The company also struck a global licensing agreement with Italy-based Recordati for its investigational Alexander disease therapy, zilganersen, outside the U.S.
The developments bolster Ionis’ commercial portfolio as it prepares for a potential U.S. launch of zilganersen later this year.
FDA Clears TRYNGOLZA for Severe Hypertriglyceridemia
The FDA approved Tryngolza as an adjunct to diet to lower triglyceride levels and reduce the risk of acute pancreatitis in adults with severe hypertriglyceridemia, defined as triglyceride levels of at least 500 mg/dL.
The therapy, available in 50 mg and 80 mg doses, is administered once monthly through an autoinjector.
The approval was supported by data from the Phase 3 CORE and CORE2 studies
In the studies, Tryngolza lowered fasting triglyceride levels by as much as 72% versus placebo after six months, with reductions maintained through 12 months. The treatment also cut acute pancreatitis events by up to 91%.
Among patients with baseline and one-year data, 86% achieved triglyceride levels below 500 mg/dL, a threshold associated with lower pancreatitis risk.
Tryngolza is expected to become available in the U.S. in July.
Ionis Partners with Recordati on Zilganersen Commercialization
Separately, Ionis entered into a licensing agreement with Recordati, granting the company exclusive rights to develop and commercialize Zilganersen in all markets outside the U.S.
Ionis will retain sole commercial rights in the U.S. and continue leading global development efforts, while Recordati will oversee regulatory filings and commercialization activities internationally.
Zilganersen, currently under FDA review with a Prescription Drug User Fee Act action date of Sept. 22, could become the first disease-modifying treatment for Alexander disease and would represent Ionis’ first independent neurology launch in the U.S.
Pivotal Study Data Supports Zilganersen Filing
Ionis recently reported additional positive data from the pivotal Zilganersen study. The trial met its primary endpoint in patients aged five years and older.
The 50 mg dose produced statistically significant stabilization in gait speed at week 61 compared with controls.
Secondary and exploratory measures also favored treatment with Zilganersen.
The investigational therapy demonstrated a favorable safety profile, with most adverse events classified as mild or moderate.
Ionis will receive a $30 million upfront payment and remain eligible for milestone payments and tiered royalties reaching the mid-20% range on annual net sales.
William Blair on Wednesday wrote, “With Tryngolza available in July for sHTG and a likely meaningful contributor to cash flow breakeven guidance in 2028.”
Analyst Myles Minter reiterated the Outperform rating.
IONS Stock Price Activity: Ionis Pharmaceuticals shares were up 2.30% at $78.28 at the time of publication on Thursday, according to Benzinga Pro data.
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