Ionis says higher olezarsen dose hits main goal of pivotal familial chylomicronaemia syndrome study

26 Sep 2023
firstwordpharma.com
Clinical ResultPhase 3
Ionis Pharmaceuticals announced Tuesday that the experimental drug olezarsen met the primary endpoint of a Phase III study in people with familial chylomicronaemia syndrome (FCS). The company said that it plans to seek FDA approval of the ligand conjugated antisense (LICA) medicine early next year, as well as submitting a filing in the EU.
Top-line results from the Balance trial showed that the higher monthly dose of olezarsen significantly reduced triglyceride levels at six months compared to placebo, with the lowering continuing to improve at 12 months. Ionis added that the higher dose of olezarsen also showed a 100% reduction in acute pancreatitis events compared to placebo, hitting a key secondary goal of the study, with no events for the experimental drug and 11 for placebo.
The trial enrolled 66 patients with confirmed FCS who received background therapies including statins, fibrates and omega-3 fatty acids. Subjects were randomised to receive placebo or olezarsen at a dose of 80mg or 50mg via subcutaneous injection once every four weeks for 53 weeks. The primary endpoint was the percent change from baseline in fasting triglyceride levels at six months, while secondary goals included triglyceride levels at 12 months and adjudicated acute pancreatitis event rate.
Lower dose not statistically significant
The company said that the higher dose of olezarsen resulted in a >75% reduction in apoC-III, a protein produced in the liver that regulates TG metabolism in the blood. However, Ionis noted that the lower dose of olezarsen evaluated in the Balance study did not reach statistical significance at six months on the primary endpoint of triglyceride lowering, although it was associated with a substantial reduction in pancreatitis.
In the trial, olezarsen - formerly known as IONIS-APOCIII-LRx - demonstrated a favourable safety and tolerability profile, with more adverse events in the placebo group, primarily due to pancreatitis. Ionis said that it will present the data at a future medical congress.
CEO Brett Monia remarked “we believe olezarsen has the potential to become the new standard of care for patients with FCS and we are excited about its potential in the broader population of patients with [severe hypertriglyceridaemia] where we have ongoing pivotal trials.” Ionis kicked off the late-stage CORE study in 2021 of olezarsen in people with severe hypertriglyceridaemia.
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