Isis Pharmaceuticals, Inc. (Nasdaq: ISIS) announced today that it has added two new drugs to its development pipeline, ISIS-FXIRx and ISIS-SMNRx.
ISIS-FXIRx is designed to treat clotting disorders without increased risk of bleeding. High levels of Factor XI are linked to myocardial infarction, stroke and venous thrombosis. In preclinical studies, ISIS-FXIRx has demonstrated potent antithrombotic activity and a superior safety profile (lower risk of bleeding) compared with standard anti-clotting agents, including low molecular weight heparin, warfarin and Factor Xa inhibitors. Isis is developing ISIS-FXIRx as part of its strategy to discover and develop antisense drugs against a range of liver-derived coagulation factors for the treatment of thromboembolic, inflammatory and oncology-related diseases. Isis' scientist, Robert MacLeod, Ph.D., and collaborators will be presenting data on ISIS-FXIRx in a poster titled "Pharmacological Characterization and Structure Activity Relationship of FXI Antisense Oligonucleotides in Cynomolgus Monkeys" at the upcoming American Society of Hematology meeting on Sunday, December 6.
"Using our efficient antisense drug discovery platform, we were able to selectively target and inhibit the production of every clotting factor produced in the liver. This extensive screening effort allowed us to evaluate the relative profiles of inhibitors of each clotting factor and to select ISIS-FXIRx, which we believe will provide the optimal therapeutic activity without causing bleeding, a major side effect of marketed anti-clotting drugs," said Stanley T. Crooke, M.D., Ph.D., Chairman and Chief Executive Officer of Isis. "With ISIS-FXIRx entering development, we are continuing to broaden our cardiovascular franchise beyond regulation of lipids and inflammatory processes into thrombosis."
ISIS-SMNRx is designed to treat spinal muscular atrophy (SMA), a neuromuscular disorder and the leading genetic cause of infant mortality. The incidence of the disease is 1 in 6,000-10,000 births, and most infants born with severe SMA die within 2 years according to the National Institutes of Health's National Institute of Neurological Disorders and Stroke. Isis is developing ISIS-SMNRx as part of its strategy to discover and develop antisense drugs against neurodegenerative diseases. ISIS' SMA program is part of its collaboration in neurodegenerative disease with Genzyme, pursuant to which Genzyme has an exclusive option to license ISIS-SMNRx from ISIS.