Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) on Sunday shared results from the pivotal Phase 3 CORE and CORE2 studies of olezarsen for severe hypertriglyceridemia (sHTG), a condition characterized by dangerously high triglyceride (fat) levels.
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The studies met the primary endpoint, with olezarsen achieving a highly statistically significant placebo-adjusted mean reduction in fasting triglyceride (TG) levels of up to 72% at six months.
The reductions were sustained through 12 months. Olezarsen showed a highly statistically significant 85% reduction in acute pancreatitis events.
Additionally, 86% of olezarsen-treated patients achieved triglyceride levels less than 500 mg/dL, below the risk threshold for acute pancreatitis, sudden inflammation of the pancreas.
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Olezarsen demonstrated favorable safety and tolerability.
These data were presented at the American Heart Association (AHA) Scientific Sessions and simultaneously published in The New England Journal of Medicine.
Data
Nearly 1,100 patients were enrolled in the CORE and CORE2 studies, which is the largest pivotal program ever conducted in sHTG, and patients were required to be on standard-of-care lipid-lowering therapy.
- TGs <880 mg/dL: 89% and 88% of patients on olezarsen 50 mg and 80 mg, respectively, achieved triglyceride levels less than 880 mg/dL, the level associated with the highest risk of acute pancreatitis.
- TGs <500 mg/dL: 86% of patients on olezarsen 50 mg and 80 mg achieved triglyceride levels less than 500 mg/dL, below the risk threshold for sHTG and acute pancreatitis.
- TGs <150 mg/dL: 34% and 54% of patients on olezarsen 50 mg and 80 mg, respectively, achieved normal triglyceride levels less than 150 mg/dL.
Olezarsen demonstrated a highly statistically significant 85% reduction in adjudicated acute pancreatitis events at 12 months.
These results were based on a total of 22 events in 17 patients in the placebo group, compared to seven events in five patients in the olezarsen group.
- In an overall pooled analysis of the number of patients needed to treat (NNT), treating 20 patients with olezarsen is estimated to prevent one acute pancreatitis event over one year.
- In the highest risk group, patients with triglyceride levels greater than or equal to 880 mg/dL and a history of acute pancreatitis, treating four patients is estimated to prevent one event over one year.
Olezarsen also showed an overall favorable lipid profile, with significant reductions in the secondary endpoints of apoC-III, remnant cholesterol, and non-HDL-C.
What Next?
Ionis is on track to submit a supplemental new drug application for both the 50 mg and 80 mg doses to the FDA by the end of the year.
An open-label extension (OLE) study of olezarsen for sHTG is ongoing. More than 90% of patients who completed CORE and CORE2 chose to continue into the OLE.
IONS Price Action: Ionis Pharmaceuticals shares were down 5.21% at $69.72 at the time of publication on Monday, according to Benzinga Pro data.
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