ISIS-APO(a)Rx and ISIS-APOCIIIRx Data Presented in Two Platform Presentations:
Lipoprotein (a) - Lost Cause or New Hope
Saturday, March 14, 2015
Beyond LDL: APOC3 and Other Emerging Genetic Targets
Monday, March 16, 2015
In two invited presentations at the 2015 American College of Cardiology Annual Meeting, held in San Diego, CA, Dr. Sotirios Tsimikas, M.D., professor of medicine and director of vascular medicine at the University of California, San Diego and vice president of clinical development and leader of the cardiovascular franchise at Isis, highlighted data from ISIS-APO(a)Rx and ISIS-APOCIIIRx.
In the first of these presentations, Dr. Tsimikas described the role of Lp(a) in cardiovascular disease and the efforts Isis is making to develop a drug to reduce the levels of this cardiovascular risk factor. Isis is developing ISIS-APO(a)Rx, an antisense drug designed to reduce apolipoprotein(a), one of the major components of Lp(a). In the presentation, Dr. Tsimikas presented data showing dose-dependent reductions in Lp(a) levels in subjects treated with ISIS-APO(a)Rx. Isis is developing ISIS-APO(a)Rx to treat patients with high Lp(a) level s who have either coronary heart disease or aortic stenosis and is currently evaluating ISIS-APO(a)Rx in a Phase 2 study in patients with high levels of Lp(a). Data from this study is planned for late 2015 or early 2016.
The second presentation discussed other newly identified targets for lowering blood lipids, including apoC-III, a key regulator of blood triglyceride levels. ISIS-APOCIIIRx is a drug designed to treat patients with severely high triglyceride levels. In Phase 2 studies, patients treated with ISIS-APOCIIIRx achieved statistically significant reductions in triglycerides, apoC-III and non-HDL-cholesterol, and statistically significant increase in HDL-cholesterol. In all clinical studies, ISIS-APOCIIIRx had a safety and tolerability profile supportive of continued development. Isis is currently evaluating ISIS-APOCIIIRx in a Phase 3 study in patients with an ultra-orphan genetic condition called familial chylomicronemia syndrome, and also plans to initiate a second Phase 3 study later in 2015 in patients with another ultra-orphan genetic condition called partial lipodystrophy. Patients in both of these diseases are characterized by high triglyceride levels and increased risk of pancreatitis, as well as other symptoms and health risks. Data from both studies are planned for 2016/2017.
Both ISIS-APO(a)Rx and ISIS-APOCIIIRx are being developed by Akcea Therapeutics, a wholly owned subsidiary of Isis Pharmaceuticals. Akcea Therapeutics is led by a high caliber operational and commercial team that is responsible for the development and commercialization of these two drugs, as well as ISIS-ANGPTL3Rx and their more potent follow on compounds.