Akcea has announced positive results from their Phase 3 trial of volanesorsen, a potential treatment for severe hypertriglyceridemia.

Volanesorsen is an antisense drug in development to decrease triglyceride levels by targeting apoliprotein C-III (ApoC-III). It is being developed specifically for familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy (FPL), two rare metabolic diseases.

Patients with FCS have extremely high levels of triglycerides and run the risk of recurrent, potentially fatal pancreatitis. FPL is characterized by an inability to store fat in normal locations resulting in high levels of triglycerides in the bloodstream, abnormal fat distribution around and within organs, and a range of metabolic abnormalities. 

Related Articles

The COMPASS trial included 113 patients with severe hypertriglyceridemia. Seventy-five patients were randomized to volanesorsen and 38 to placebo. After 13 weeks of treatment, the volanesorsen treated group achieved a statistically significant (P<0.0001) mean reduction in triglycerides of 71.2% from baseline. The effect was sustained through the end of the 26 week treatment period.

Seven patients in the trial had FCS; the volanesorsen-treated patients (n=5) achieved a mean reduction in triglycerides of 73% from baseline after 13 weeks of treatment. This effect was sustained through the end of the 26 weeks.

Overall 82% of volanesorsen-treated patients achieved triglyceride levels of ≤500mg/dL after 13 weeks (P<0.0001).

“The success of COMPASS represents an important milestone towards our planned regulatory filings for volanesorsen in the U.S., Europe and Canada in 2017,” said Paula Soteropoulos, president and CEO of Akcea Therapeutics.

Akcea Therapeutics is a wholly-owned subsidiary of Ionis Pharmaceuticals, Inc. The company plans to present additional data from the study at an upcoming medical meeting.

For more information visit Akceatx.com.