Isis Begins Phase III Study Of Rare Disease Antisense Drug
Isis Pharmaceuticals announced that it has begun the Phase III study of ISIS-APOCIIIRx in patients with the rare disease, familial chylomicronemia syndrome (FCS). The 6-month, placebo controlled, randomized, double blind Phase III study will investigate the safety and efficacy of a 300 mg once weekly dose of ISIS-APOCIIIRx in 50 patients with FCS. The trial’s primary endpoint is percent change in fasting triglycerides from baseline following three months of dosing with ISIS-APOCIIIRx.
ISIS-APOCIIIRx is an antisense drug under development to treat patients with severely high triglycerides, either as monotherapy or as part of a combination with other triglyceride-lowering agents. The drug targets apoC-III, a protein produced in the liver that is a major factor in the regulation of serum triglycerides. Research shows that lowering apoC-III levels can result in lower triglyceride levels, as well as significantly reduce cardiovascular disease.
Earlier this year Isis also announced that the European Committee for Orphan Medicinal Products (COMP) positively recommended ISIS-APOCIIIRx for Orphan Drug Designation as treatment for FCS.
Familial chylomicronemia syndrome is a rare and life-threatening disorder linked with triglyceride levels higher than 2,000 mg/dL. Extremely high levels of triglyceride put patients at risk of various conditions, including frequent episodes of pancreatitis. FCS affects approximately 3,000 to 5,000 people worldwide.
Sotirios Tsimikas, professor of medicine and director of vascular medicine at the University of California, San Diego, said, “SIS-APOCIIIRx 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 2 data strongly supports advancing ISIS-APOCIIIRx into Phase 3 studies in patients with severely elevated triglycerides like FCS.”
Aside from FCS, Isis is also developing the drug as treatment for patients with spiked levels of triglycerides (greater than 880 mg/dL), a condition that afflicts around 50,000 patients in the U.S. and EU combined. Isis said it plans to initiate the Phase III trial for the second indication later this year.