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ISIS Pharmaceuticals Advances Phase 2 Study Of ISIS-SMN Rx In Infants With Spinal Muscular Atrophy

CARLSBAD, Calif., Oct. 3, 2013 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today that it dosed the first infant in the 12 mg dose group in the Phase 2 study evaluating ISIS-SMN Rx in infants with Type I spinal muscular atrophy (SMA).  The Phase 2 study is designed to allow Isis to define the optimal dose for the larger Phase 2/3 study in infants and to provide safety and tolerability data.  Isis plans to complete this study and initiate the larger Phase 2/3 pivotal study early next year.  Isis earned a $2 million milestone payment from Biogen Idec associated with the advancement of this study. 


The Phase 2 study of ISIS-SMN Rx is an open-label, multiple-dose, dose-escalation pilot study, which will include eight infants who have been diagnosed with Type I SMA.  To meet enrollment criteria, infants must be between the ages of three weeks and seven months, live in close proximity to a study site and pass screening evaluations conducted at study sites.  The study is being conducted at centers in the United States and Canada.  For further study information, please visit and search for ISIS-SMN Rx

"We continue to be very encouraged with the progress we are making with ISIS-SMN Rx, and we are on track to begin our Phase 3 program early next year.   We look forward to sharing the results from our multiple dose studies in infants with Type I SMA and in children with Type II and Type III SMA later this year or early next year," said C. Frank Bennett, Ph.D., senior vice president of research at Isis.   

ABOUT ISIS-SMN Rx ISIS-SMN Rx is designed to alter the splicing of a closely related gene (SMN2) to increase production of fully functional SMN protein.  The United States Food and Drug Administration granted orphan drug status and fast track designation to ISIS-SMN Rx for the treatment of patients with SMA.  Isis is currently in collaboration with Biogen Idec to develop and potentially commercialize the investigational compound, ISIS-SMN Rx, to treat all types of SMA.  Under the terms of the January 2012 agreement, Isis is responsible for global development and Biogen Idec has the option to license the compound until completion of the first successful Phase 2/3 study. 

Isis acknowledges support from the following organizations for ISIS-SMN Rx: Muscular Dystrophy Association, SMA Foundation, Families of SMA and intellectual property licensed from Cold Spring Harbor Laboratory and the University of Massachusetts Medical School.

ABOUT SMASMA is a severe genetic disease that affects approximately 30,000-35,000 patients in the United States, Europe and Japan. SMA is caused by a loss of, or defect in, the survival motor neuron 1 (SMN1) gene leading to a decrease in the survival motor neuron (SMN) protein. SMN is critical to the health and survival of nerve cells in the spinal cord responsible for neuromuscular growth and function. One in 50 people, the equivalent of about 6 million people in the United States, are carriers of a defective SMN1 gene, which is unable to produce fully functional SMN protein. Carriers experience no symptoms and do not develop the disease. However, when both parents are carriers, there is a one in four chance that their child will have SMA. The severity of SMA correlates with the amount of SMN protein. Infants with Type I SMA, the most severe form of the disease, produce very little SMN protein and have a life expectancy of less than two years. Children with Type II have greater amounts of SMN protein but still have a shortened lifespan and are never able to stand independently. Children with Type III have a normal lifespan but accumulate life-long physical disabilities as they grow.

ABOUT ISIS PHARMACEUTICALS, INC.Isis is exploiting its leadership position in antisense technology to discover and develop novel drugs for its product pipeline and for its partners.  Isis' broad pipeline consists of 30 drugs to treat a wide variety of diseases with an emphasis on cardiovascular, metabolic, severe and rare diseases, including neurological disorders, and cancer.  Isis' partner, Genzyme, is commercializing Isis' lead product, KYNAMRO™, in the United States for the treatment of patients with HoFH.  Isis' patents provide strong and extensive protection for its drugs and technology.  Additional information about Isis is available at

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