ISIS-APOCIIIRx: Final Phase II data

Final data from 26 patients with high to severely high triglyceride levels (225-2,000 mg/dL) on stable doses of fibrates in cohort 1 of a double-blind Phase II trial showed that once-weekly 200 and 300 mg subcutaneous doses of ISIS-APOCIIIRx for 13 weeks each led to significant mean percent reductions from baseline vs. placebo in both APOCIII (p<=0.01 for low-dose and p<=0.001 for high-dose) and triglyceride (p<=0.05 for both) levels - the co-primary endpoints - that were sustained to at least 6 weeks after the last dose. Additionally, ISIS-APOCIIIRx led to a sustained, significant increase from baseline at post-treatment week 6 in mean percent HDL-C (p<=0.01 for both) and a sustained, significant reduction from baseline at post-treatment week 6 in mean triglyceride (mg/dL) levels (p<=0.001 for low-dose and p<=0.05 for high-dose) vs. placebo. Data were presented at the American College of Cardiology meeting in Washington, D.C.

In July 2013, Isis reported data from cohort 1 showing that both doses of ISIS-APOCIIIRx met the co-primary endpoints of reducing APOCIII and triglyceride levels from baseline to week 13 vs. placebo (see BioCentury, July 29, 2013). In September 2013, the company reported data from 28 evaluable patients with very high to severely high triglyceride levels (440-2,000 mg/dL) who were not on a triglyceride-lowering therapy in cohort 2 of the trial showing that both doses of ISIS-APOCIIIRx as monotherapy met the same co-primary endpoints vs. placebo. The company also previously reported data from 3 patients with genetic confirmation of familial chylomicronemia syndrome and baseline triglyceride levels of >1,400 mg/dL in cohort 3 of the trial (see BioCentury, Sept. 16, 2013 & Sept. 30, 2013). ...