Isis Reports Positive Phase 2 Study Results For Antisense Drug
Isis Pharmaceuticals Inc. announced positive final results from the Phase II study evaluating its antisense drug ISIS-APOCIIIRx in patients with high to severely high triglycerides on stable doses of fibrates.
The placebo-controlled, double-blind, randomized, 13-week Phase II study was designed to assess the safety and activity of ISIS-APOCIIIRx at 200 mg and 300 mg doses. The drug produced statistically significant mean percent reductions from baseline in triglycerides and apoC-III and apoC-III-associated VLDL in both dose cohorts. Patients treated with the drug also demonstrated rapid, prolonged and statistically significant mean percent increase from baseline in HDL-cholesterol in both dose cohorts. In addition, patients achieved average reductions of up to 71% in apolipoprotein C-III (apoC-III) and up to 64% in triglycerides, and average increases of up to 52% in high-density lipoprotein cholesterol (HDL-C), the 'good' cholesterol.
Richard Geary, SVP of development at Isis, said, “We have shown that ISIS-APOCIIIRx has lowered triglycerides equally well in patients with high to severely high triglycerides as well as in patients with high triglycerides and type 2 diabetes. We observed significant reductions in apoC-III and triglycerides and increased HDL-C both when ISIS-APOCIIIRx is administered as a single agent and in combination with fibrates. We also observed significant reductions in the number of VLDL particles and increases in the number of HDL particles. These data are consistent with a mechanism of reducing ApoC-III and exactly what we would expect given the significant reductions of triglycerides and other lipid parameters in patients treated with ISIS-APOCIIIRx we have observed in our Phase 2 clinical trials. We look forward to advancing ISIS-APOCIIIRx into Phase 3 clinical studies this year.”
ISIS-APOCIIIRx is an antisense drug designed to treat patients with severely high triglycerides either as a single agent or in combination with other triglyceride-lowering agents. The drug targets apoC-III, a protein produced in the liver that plays a central role in the regulation of serum triglycerides. Patients with elevated levels of apoC-III have high triglycerides associated with multiple metabolic abnormalities, such as insulin resistance and/or metabolic syndrome. Those with severely elevated levels of triglycerides are at risk of many serious health conditions, including Type 2 diabetes and pancreatitis, which can require hospitalization.
Results from the study were presented at the recent American College of Cardiology meeting in Washington, D.C.