Ionis Pharmaceuticals reported on Tuesday that a drug approved for a rare disease successfully lowered the triglycerides of patients with a common condition and averted pancreas damage in a pair of late-stage trials, opening up a potential blockbuster opportunity for the biotech.
The Phase 3 trials found that, compared to a placebo, the therapy, olezarsen, lowered triglycerides by up to 72% in patients with severe hypertriglyceridemia, a condition that affects 3 million people in the U.S. Patients taking the therapy also had 85% fewer episodes of pancreas inflammation than those on a placebo after a year. These findings were statistically significant.
The drug was generally safe and well tolerated, with serious adverse events rarer among those on olezarsen than in the placebo groups. The most common side effects were mild injection site reactions; 90% of participants continued onto an open-label extension study for long-term monitoring.
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