Ionis Reveals Encouraging Findings from Phase 3 Balance Trial of Olezarsen in Familial Chylomicronemia Syndrome

Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) has unveiled comprehensive findings from the Phase 3 Balance study of olezarsen, its leading independent investigational medication, targeted at adults grappling with familial chylomicronemia syndrome (FCS). The study revealed that the monthly administration of olezarsen at an 80 mg dose effectively achieved the primary endpoint, showcasing a significant reduction in triglyceride (TG) levels among patients with genetically validated FCS over a six-month period. This reduction in TG levels was maintained over time, with a substantial placebo-adjusted decrease of 59% observed at the 12-month mark. Furthermore, olezarsen demonstrated consistent and noteworthy decreases in serum apolipoprotein C-III (apoC-III) levels.

Remarkably, the incidence of acute pancreatitis (AP) events, a critical concern for individuals with FCS, was notably reduced during the 12-month treatment period compared to placebo. Patients treated with olezarsen also experienced a significant decrease in all-cause hospitalizations, emphasizing the potential of this medication to alleviate the burden of FCS-related complications.

The study further highlighted the favorable safety and tolerability profile of olezarsen, with no serious treatment-related adverse events reported. Notably, olezarsen-treated patients exhibited a lower number of treatment-emergent adverse events (TEAEs) compared to the placebo group. These findings underscore the potential of olezarsen as a breakthrough therapy for adults grappling with FCS, a rare and life-threatening condition for which approved treatments are currently unavailable in the United States.

Dr. Erik Stroes, a principal investigator of the Balance study, emphasized the pressing need for effective therapies in managing FCS-related complications, citing olezarsen as a promising solution that could significantly enhance the quality of life for affected individuals.

The study results were presented at the 2024 American College of Cardiology (ACC) Annual Meeting in Atlanta, Georgia, and simultaneously published in The New England Journal of Medicine (NEJM), further validating the significance of these findings within the medical community.

Looking ahead, Ionis is actively pursuing regulatory approval for olezarsen based on these compelling data, with the aim of addressing the unmet medical needs of individuals living with FCS. The company remains committed to advancing the development of olezarsen and anticipates a successful outcome in ongoing Phase 3 CORE studies evaluating its efficacy in a broader population of patients with severe hypertriglyceridemia.

In addition to the Balance study data, a late-breaking abstract titled “Efficacy and Safety of Olezarsen in Patients with Hypertriglyceridemia and High Cardiovascular Risk: Primary Results of the Bridge-TIMI 73a Trial” was also presented at the ACC meeting and published in NEJM.

To delve deeper into the detailed results of the Balance study, Ionis will host a webcast on Monday, April 8 at 10:00 am ET, offering interested parties an opportunity to gain insights into the potential of olezarsen as a groundbreaking therapy for FCS. A webcast replay will be available for a limited time following the event.

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