Isis Pharmaceuticals ISIS-APOCIIIRx begins phase 3 study in CFS patients

Isis Pharmaceuticals ISIS-APOCIIIRx begins phase 3 study in CFS patients -

Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced the initiation of a Phase 3 evaluating ISIS-ApoCIII _Rx in patients of family chylomicronemia syndrome (FCS). FCS is a rare orphan disease characterized by extremely high levels of triglycerides, which affects about 3,000 to 5,000 patients worldwide. The phase 3 study of ISIS-ApoCIII _Rx is a double-blind, placebo-controlled, randomized study of six months in 50 patients diagnosed with FCS. The study will evaluate the efficacy and safety of a 300 mg once weekly doses of ISIS-ApoCIII _Rx . The primary endpoint of the study was the percent change in fasting triglycerides from baseline after three months of treatment.

"FCS is a rare and serious genetic disease that is often associated with higher triglyceride levels to 00 mg / dL. Because of their extremely high triglyceride levels, CFS patients are at significant risk many serious health problems, including frequent bouts of pancreatitis, which may require hospitalization and may be life threatening. current treatment options do not reduce enough to reduce the risk of serious illness triglycerides in patients with FCS, "said Sotirios Tsimikas, MD, professor of medicine and director of vascular medicine at the University of California, San Diego and Vice President of clinical development and Chief cardiovascular franchise Isis. "ISIS-ApoCIII _Rx provides a unique therapeutic approach in that it is designed to reduce triglyceride levels by reducing apoC-III, an important regulator of the clearance of triglycerides. Data from Phase 2 strongly support advancing ISIS-ApoCIII _Rx in phase 3 studies in patients with highly elevated triglycerides as FCS. "

in a phase 2 study, patients treated with FCS ISIS-ApoCIII _Rx experienced decreases in triglyceride up to more than 1500 mg / dl. Isis also evaluated ISIS-ApoCIII _Rx in a broad Phase 2 program in these studies in patients with triglyceride levels to extremely high very high, patients treated with ISIS-ApoCIII _Rx achieved substantial reduction in triglycerides (average percentage reductions of up to 71%) and APOC-III (mean of the percentage discounts up to 88%) and increased HDL cholesterol ( the average percentage increases of up to 78%).

"Our goal is to bring ISIS-ApoCIII _Rx on the market for patients with severely elevated triglycerides. These patients are at increased health risk because they can not reduce their triglycerides to safe levels with currently available drugs. We are pleased with the data from our Phase 2 program, in which the triglyceride substantial decrease was achieved when ISIS-ApoCIII _Rx was administered as monotherapy or in combination with fibrates in patients with a wide range of incoming triglycerides, including CFS patients, "said Richard Geary, Ph.D., senior Vice President of development at Isis." Our extensive experience in the development of drugs to treat lipid disorders, including good working relationships we have established with physicians and centers that treat many patients FCS should support the rapid progress of this program. We currently expect advance ISIS-ApoCIII _Rx without seeking a partner. "

ISIS-ApoCIII _Rx is designed to target apoC-III, a protein produced in the liver which plays a central role in the regulation of serum triglycerides. ApoC-III is a genetically validated target for lowering triglycerides. Independent studies have shown a link between the lower activity apoC-III, which results in reduced levels of triglycerides and cardiovascular disease.

In addition to developing ISIS-ApoCIII _Rx for patients with FCS, Isis also developing ISIS-ApoCIII _Rx for patients with highly elevated triglycerides, higher to 880 mg / dL, a condition that affects about 50,000 patients in the United States and Europe. These patients not only have a high risk of pancreatitis, but also have a higher risk of type 2 diabetes and cardiovascular disease. Current treatments do not sufficiently reduce triglyceride levels in many of these patients to reduce these health risks. Isis plans to launch the Phase 3 program in patients with highly elevated triglycerides in 2014.