Ionis Pharmaceuticals has announced that the Phase 3 Balance study of olezarsen in people with familial chylomicronemia syndrome (FCS) met its primary efficacy endpoint.
Olezarsen is an investigational LIgand Conjugated Antisense (LICA) medicine. It is indicated to restrain the body’s production of apoC-III, a protein that controls blood’s triglyceride (TG) metabolism.
The global, multicentre, randomised, double-blind, placebo-controlled Balance study recruited 66 patients aged18 and older with confirmed FCS.
The patients were randomised in a 1:1:1 ratio to get olezarsen 80mg or 50mg or placebo through subcutaneous injection once every four weeks for 53 weeks.
In the study, the results showed a statistically significant reduction in TG levels with the olezarsen 80mg monthly dose at six months compared to placebo, with TG reduction continued to improve at 12 months.
Additionally, olezarsen 80mg showed a 100% reduction in acute pancreatitis events compared to placebo, which was the key secondary endpoint. Olezarsen 80mg treatment caused a >75% decrease in apoC-III.
The drug showed a dose-dependent impact, with pancreatitis significantly reduced at both 80mg and 50mg trial doses.
At six months, the primary aim of reducing triglycerides was not statistically significantly achieved with the lower 50mg dose, the US-based biotechnology company said.
Overall, the safety and tolerability profile of the medicine was found favourable in the study.
Ionis CEO Brett Monia said: “Today is a proud moment in our company’s evolution, as olezarsen is poised to be a long-awaited advance for FCS patients and Ionis’ first potential independent commercial launch.
“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 SHTG where we have ongoing pivotal trials.”
In addition to EU regulatory filings, Ionis intends to submit a New Drug Application to the US Food and Drug Administration (FDA) in the early 2024 timeframe. If approved, Olezarsen would be the first medication for FCS.
The medicine received Fast Track designation from the FDA this year for the treatment of FCS.
