Ionis Pharmaceuticals presents results from Phase 3 Balance study olezarsen

Ionis Pharmaceuticals announced full results from the Phase 3 Balance study of its lead independent investigational medicine, olezarsen, for the treatment of adults with familial chylomicronemia syndrome. The olezarsen 80 mg monthly dose met the primary endpoint of significantly reducing triglycerides in patients with genetically validated FCS at six months. In addition, olezarsen demonstrated robust and sustained reductions in TGs and serum apolipoprotein C-III levels. Importantly, olezarsen reduced the incidence of acute pancreatitis events over the 12-month treatment period compared to placebo. Olezarsen also demonstrated a favorable safety and tolerability profile. In the study, patients were treated with olezarsen 80 mg, 50 mg or placebo once every four weeks. In the 80 mg group, olezarsen met the primary endpoint, with a statistically significant placebo-adjusted reduction in TG levels from baseline to six months. Reductions from six to 12 months were sustained, with olezarsen 80 mg achieving a placebo-adjusted 59% reduction in TGs. ApoC-III placebo-adjusted reductions were robust and sustained at six and 12 months. In the 50 mg group, olezarsen reduced TG levels, however this difference was not statistically significant at six months compared to placebo. Reductions from six to 12 months were improved, with olezarsen 50 mg achieving a placebo-adjusted 44% reduction in TGs. Olezarsen-treated patients had markedly fewer AP events during the 12-month period, compared to placebo. Eleven episodes of AP occurred in the placebo group versus one episode in the 80 mg olezarsen group and one episode in the 50 mg group. Furthermore, there was a substantially greater time to the first event with olezarsen compared to placebo. Olezarsen-treated patients experienced a placebo-adjusted 84% reduction in all-cause hospitalizations between baseline and 12 months. A favorable safety and tolerability profile was observed, with a higher number of treatment-emergent adverse events (TEAEs) in the placebo group. There were no serious TEAEs related to olezarsen.