Isis Pharmaceuticals (ISIS) has started a Phase III clinical trial of ISIS-APOCIII to treat patients with familial chylomicronemia syndrome (FCS), a rare orphan disease.
Around 50 patients diagnosed with FCS will be enrolled in the randomised, double-blind, placebo-controlled, six month Phase III trial.
The trial will evaluate the efficacy and safety of a 300mg once weekly dose of ISIS-APOCIII and the primary endpoint is percent change in fasting triglycerides from baseline after three months of dosing.
Isis clinical development vice president and University of California professor of medicine and director of vascular medicine Sotirios Tsimikas: "FCS is a rare and very serious genetic disorder that is often associated with triglyceride levels higher than 2,000mg/dL.
"Current treatment options do not reduce triglyceride levels enough to reduce the risk of serious illness in patients with FCS.
"ISIS-APOCIII provides a unique therapeutic approach, in that it is designed to reduce triglyceride levels by reducing apoC-III, an important regulator of triglyceride clearance.
"The Phase II data strongly support advancing ISIS-APOCIII into Phase III studies in patients with severely elevated triglycerides like FCS."
ISIS-APOCIII is designed to target apoC-III, a protein produced in the liver that plays a major role in the regulation of serum triglycerides.
FCS patients treated with ISIS-APOCIII in a Phase II trial experienced decreases in triglycerides of up to more than 1,500mg/dL.
The company has also assessed ISIS-APOCIII in a broad Phase II programme, which showed that patients treated with ISIS-APOCIII achieved substantial lowering of triglycerides and apoC-III and increasing of HDL-cholesterol.
Isis development senior vice president Richard Geary said: "Our focus is to bring ISIS-APOCIIIRx to the market for patients with severely elevated triglycerides.
"These patients are at significant health risk because they cannot reduce their triglycerides to safe levels with currently available medicines.
"We are pleased with the data from our Phase II programme in which substantial triglyceride lowering was achieved when ISIS-APOCIIIRx was dosed as a single agent or in combination with fibrates in patients with a wide range of incoming triglycerides, including FCS patients."
The company also developing ISIS-APOCIII for patients with severely elevated triglycerides, greater than 880mg/dL, a condition that affects an estimated 50,000 patients in the US and Europe.