Ionis inches toward first solo drug launch with late-stage trial success for rare genetic disease therapy

Ionis Pharmaceuticals, a California biotech that has spearheaded the development of RNA-targeting medicines, announced on Tuesday that its drug against familial chylomicronemia syndrome, a rare and serious disease that prevents the body from breaking down fats, succeeded in a late-stage clinical trial.

The Phase 3 trial, dubbed Balance, enrolled 66 adults who were randomly divided into three groups and given a monthly injection of placebo or one of two doses of olezarsen, the company’s experimental drug. Treatment with the higher dose of the therapy significantly lowered triglycerides in the blood of those on the drug for six months compared to participants given placebo, though a lower dose did not have this effect. Participants on the high dose didn’t have any instances of painful inflammation of the pancreas, one of the disease’s most serious symptoms, while there were 11 such episodes among those on placebo.

Researchers reported that the treatment was also generally safe, with fewer adverse events among those on olezarsen than among the placebo group.

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