CARLSBAD, Calif., March 5 /PRNewswire-FirstCall/ -- Isis Pharmaceuticals, Inc. announced today that it has initiated a Phase 1 study of ISIS-SOD1Rx in patients with an inherited, aggressive form of Lou Gehrig's disease also known as familial amyotrophic lateral sclerosis (ALS). Approximately 20 percent of all familial ALS cases are caused by a mutant form of superoxide dismutase, or SOD1. ISIS-SOD1Rx is an antisense drug designed to inhibit the production of SOD1. The ALS Association and the Muscular Dystrophy Association are providing funding for the development of ISIS-SOD1Rx.
"It is evident that certain cases of familial ALS are related to mutant forms of SOD1. Therefore, the selective inhibition of SOD1 production could provide a way to improve the outcomes of these patients with ALS," said Timothy Miller, M.D., Ph.D., Assistant Professor of Neurology at Washington University School of Medicine and Director of the Christopher Wells Hobler Laboratory for ALS Research at the Hope Center for Neurological Disorders. "This is the first study to administer an inhibitor of SOD1 directly into the central nervous system. Having been involved with Isis in the research and now the clinical development of ISIS-SOD1Rx, I am looking forward to co-chairing this study with Dr. Cudkowicz and the Northeast ALS Consortium."
The Phase 1 study of ISIS-SOD1Rx is a placebo-controlled, dose-escalation study designed to assess the safety, tolerability and pharmacokinetic profile of ISIS-SOD1Rx in patients with familial ALS that is caused by mutations within the SOD1 gene. The study consists of four cohorts with eight patients each. In this study, ISIS-SOD1Rx will be administered intrathecally using an external pump to deliver the drug directly into the spinal fluid during a single, 12-hour infusion. The study will be conducted in multiple centers within the United States.
"This is a watershed moment," said R. Rodney Howell, M.D., Chairman of the MDA Board of Directors. "More than 30 people with familial ALS caused by mutations in the SOD1 gene soon will receive infusions of a SOD1 inhibitor directly into their central nervous systems." Dr. Howell added that "MDA has invested more than $270 million fighting ALS, and it's exciting to see an excellent dose-escalation study get underway to assess the safety of ISIS-SOD1Rx."
ABOUT ISIS PHARMACEUTICALS, INC.
Isis is exploiting its expertise in RNA to discover and develop novel drugs for its product pipeline and for its partners. The Company has successfully commercialized the world's first antisense drug and has 22 drugs in development. Isis' drug development programs are focused on treating cardiovascular, metabolic and severe neurodegenerative diseases and cancer. Isis' partners are developing antisense drugs invented by Isis to treat a wide variety of diseases. Isis and Alnylam Pharmaceuticals are joint owners of Regulus Therapeutics Inc., a company focused on the discovery, development and commercialization of microRNA therapeutics. Isis also has made significant innovations beyond human therapeutics resulting in products that other companies, including Abbott, are commercializing. As an innovator in RNA-based drug discovery and development, Isis is the owner or exclusive licensee of over 1,600 issued patents worldwide. Additional information about Isis is available at www.isispharm.com.
In this press release, unless the context requires otherwise, "Isis," "Company," "we," "our," and "us" refers to Isis Pharmaceuticals and its subsidiaries, including Regulus Therapeutics Inc.
CONTACT: Kristina Lemonidis, Director, Investor Relations,
+1-760-603-2490; or Amy Blackley, Ph.D., Assistant Director, Corporate
Communications, +1-760-603-2772, both of Isis Pharmaceuticals, Inc.
Web site: http://www.isispharm.com/
