This week Ionis Pharmaceuticals, Inc. issued a press release via PRNewswire announcing topline Phase 3 results for its Balance study of olezarsen. The study ( NCT04568434 ) enrolled people with familial (inherited) chylomicronemia syndrome (FCS), many of whom saw a 100% reduction of acute pancreatitis which is inflammation of the pancreas and which often results in severe pain in the abdomen.
The trial participants also saw their triglyceride levels improve for a minimum of 12 months. High triglycerides can increase the risk of heart and vascular disease.
FCS is a rare, and sometimes fatal, inherited lipid disease. It is difficult to diagnose and therefore often goes unnoticed or unmanaged. It is caused by a mutation in lipoprotein lipase.
About the Trial
The study was designed to evaluate olezarsen’s efficacy from the baseline against a placebo on the percent change in fasting triglycerides.
Olezarsen was administered at a monthly dose of 80 mg and showed a significant triglyceride reduction at six months when compared to a placebo. Olezarsen 80mg showed zero acute pancreatitis events at the secondary endpoint compared to eleven secondary events for a placebo.
The olezarsen 80mg dose also caused a 75% reduction in a protein located in the liver that regulates the metabolism of triglycerides in the blood.
Additionally, the study evaluated a 50mg dose each month which did not reach significance at the six-month mark. FCS is a rare, inherited lipid disease that often goes underdiagnosed and unmanaged.
The Future of Olezarsen
Ionis has plans to file an NDA early in 2024 with the FDA and also participate in European regulatory filings. If approved, olezarsen will be the first treatment for FCS in the United States. The company will also participate in European regulatory filings. Approval would mean that olezarsen would be the first treatment that is available in the United States for this genetic disease that leads to fatal pancreatitis attacks.
In 2023, the FDA granted olezarsen Fast Track designation to treat FCS for new drugs that show a potential to remedy unmet medical needs. Onis is prepared to demonstrate the drug’s potential at a future medical conference.
Ionis Senior Vice President, Dr. Sam Tsimikas, stated that approval of the drug represents a very important step in relieving patients of the fear of unexpected abdominal pain and fatal attacks of acute pancreatitis. Dr. Tsimikas further stated that the positive study results gave strength to Ionis’ confidence in olezarsen’s ability to deliver benefits to FCS patients.
About the Trial
The study was designed to evaluate olezarsen’s efficacy from baseline against a placebo on the percent change in fasting triglycerides.
Olezarsen was administered at a monthly dose of 80 mg and showed a significant triglyceride reduction at six months when compared to placebo. Olezarsen 80mg showed zero acute pancreatitis events at the secondary endpoint compared to eleven secondary events for placebo.
The Future of Olezarsen
Ionis has plans to file an NDA early in 2024 with the FDA and also participate in European regulatory filings. If approved, olezarsen will be the first treatment for FCS in the United States. The company will also participate in European regulatory filings.
In 2023, the FDA granted olezarsen Fast Track designation to treat FCS for new drugs that show a potential to remedy unmet medical needs. Ionis is prepared to demonstrate the drug’s potential at a future medical conference.
Ionis Senior Vice President, Dr. Sam Tsimikas, stated that approval of the drug represents a very important step in relieving patients of the fear of unexpected abdominal pain and fatal attacks of acute pancreatitis. Dr. Tsimikas further stated that the positive study results gave strength to Ionis’ confidence in olezrsen’s ability to deliver benefits to FCS patients.
