Ionis Reports Positive Results From Phase 3 Study Of Olezarsen In Familial Chylomicronemia Syndrome

(RTTNews) - Ionis Pharmaceuticals Inc. (IONS) announced positive results for the Phase 3 Balance study of olezarsen in people with familial chylomicronemia syndrome.

Familial chylomicronemia syndrome or FCS is a rare, debilitating genetic disease that can lead to acute, potentially fatal pancreatitis attacks.

In Tuesday pre-market trade, IONS was trading at $44.10 up $1.44 or 3.38%.

The company noted that the trial met its primary efficacy endpoint with a statistically significant reduction in triglyceride (TG) levels with the olezarsen 80 mg monthly dose at six months compared to placebo; triglyceride lowering continued to improve at 12 months.

In addition, olezarsen 80 mg showed a 100 percent reduction in acute pancreatitis events compared to placebo (0 events for olezarsen versus 11 events for placebo), a key secondary endpoint.

Treatment with olezarsen 80 mg resulted in a more than 75% reduction in apoC-III, a protein produced in the liver that regulates triglyceride metabolism in the blood. In addition to the 80 mg monthly dose, the study also evaluated a lower 50 mg monthly dose. Olezarsen demonstrated a dose-dependent effect, with both study doses showing a substantial reduction in pancreatitis. The lower 50 mg dose did not reach statistical significance at six months on the primary endpoint of triglyceride lowering.

Ionis said it plans to file a New Drug Application in early 2024 with the U.S. Food and Drug Administration in addition to EU regulatory filings. The FDA granted olezarsen Fast Track designation for the treatment of FCS in 2023.

For More Such Health News, visit rttnews.com