Isis Pharma Begins Phase 3 Trial Of ISIS-APOCIII Rx In Patients With FCS

(RTTNews) - Drugmaker Isis Pharmaceuticals, Inc. (ISIS) said it has begun a Phase 3 study assessing ISIS-APOCIIIRx in patients with familial chylomicronemia syndrome or FCS, which is a rare orphan disease, characterized by extremely high triglyceride levels, affecting an estimated 3,000 to 5,000 patients worldwide.

The Phase 3 trial of ISIS-APOCIIIRx is a randomized, double-blind, placebo-controlled, six month study in approximately 50 patients diagnosed with FCS. This would evaluate the efficacy and safety of a 300 mg once weekly dose of ISIS-APOCIIIRx. The primary endpoint of the study is percent change in fasting triglycerides from baseline after three months of dosing.

The Phase 2 data strongly support advancing ISIS-APOCIIIRx into Phase 3 studies in patients with severely elevated triglycerides like FCS. In a Phase 2 study, FCS patients treated with ISIS-APOCIIIRx experienced decreases in triglycerides of up to more than 1,500 mg/dL. Also, Isis has evaluated ISIS-APOCIIIRx in a broad Phase 2 program.

ISIS-APOCIIIRx is designed to target apoC-III, a protein produced in the liver that plays a central role in the regulation of serum triglycerides. ApoC-III is a genetically validated target for lowering triglycerides.