Ionis Pharmaceuticals has achieved a significant milestone in their phase 3 clinical trial focused on therapies that reduce lipids. This achievement positions the company to seek approval, marking the commencement of its independent commercial ventures. The trial exhibited a remarkable reduction in triglyceride levels with the administration of a high dose, alongside an impressive 100% decline in acute pancreatitis events compared to a placebo.
Ionis Pharmaceuticals meticulously crafted the trial to evaluate the effectiveness of their ligand-conjugated antisense drug, olezarsen, in patients afflicted by familial chylomicronemia syndrome (FCS), a rare genetic disorder resulting in exceedingly elevated triglyceride levels in the bloodstream. A prior attempt for FDA approval for a different treatment targeting this condition, volanesorsen, faced rejection in 2019. However, buoyed by promising data from the current trial, Ionis is confident about securing FDA approval for olezarsen.
The optimistic outlook is grounded in crucial trial data emerging from a phase 3 trial encompassing 66 adults, randomly assigned to receive either a placebo or one of two monthly subcutaneous doses of olezarsen. The trial spanned six months, during which Ionis observed a statistically significant drop in triglyceride levels among those receiving the higher 80-mg dose. The lower 50-mg dose did not manifest significant improvements over the placebo concerning triglyceride level reduction.
Brett Monia, CEO of Ionic highlighted the criticality of this primary endpoint outcome, emphasizing its implications for the commercial viability of olezarsen in treating FCS. He stressed that a significant reduction in triglyceride levels within this patient population will be instrumental in driving market success.
Importantly, the trial revealed that patients administered the 80-mg dose experienced no acute pancreatitis events, a stark contrast to the 11 such events recorded in the placebo group. The 50-mg dose also demonstrated a considerable reduction in pancreatitis incidents.
Furthermore, the 80-mg dose showcased a reduction of more than 75% in apoC-III, the target protein, and exhibited a favorable safety and tolerability profile as per Ionis. Although specific safety and tolerability data are yet to be revealed, Ionis asserted that the majority of adverse events linked to olezarsen were mild, with no instances of hepatic or renal toxicity, or clinically significant reductions in platelet count. The company also confirmed one death unrelated to the drug.
Ionis Pharmaceuticals is gearing up to apply for FDA approval early in the upcoming year. This strategic move aligns with the company’s fast-track program, propelling them towards the delivery of the inaugural treatment for FCS in the U.S. Simultaneously, Ionis is conducting phase 3 trials targeting severe hypertriglyceridemia, a condition affecting a considerably larger patient population, reinforcing their belief in olezarsen’s potential to achieve substantial sales.