Current Advances

ISIS-SMN_Rx Phase 1 Study in Patients With Spinal Muscular Atrophy Presented at American Academy of Neurology

Dr. Claudia Chiriboga to present Phase 1 data on Wednesday, March 20 at 4:13 pm PT

Isis to host an investor event and live webcast on Thursday, March 21 at 7:30 am PT

ISIS-SMN_Rx is designed to treat all types of childhood SMA by altering the splicing of a closely related gene (SMN2) to increase production of fully functional SMN protein. ISIS-SMN_Rx is currently being evaluated in a Phase 1b/2a multiple-dose, dose-escalation study in children with Type 2 or Type 3 SMA. Data from the completed Phase 1 single dose, dose-escalation study will be presented at the upcoming American Academy of Neurology meeting in San Diego on March 20 in a presentation titled ‘Results of an open-label, escalating dose study to assess the safety, tolerability, and dose range finding of a single intrathecal dose of ISIS-SMN_Rx in patients with spinal muscular atrophy’.

Interested parties may attend the Isis hosted investor event on the following day, March 21, at 7:30 am PT by contacting info@isisph.com. A live webcast of the event will also be available at www.isispharm.com.

SMA 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 SMA. 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 1 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 2 have greater amounts of SMN protein but still have a shortened lifespan and are never able to stand independently. Children with Type 3 have a normal lifespan but accumulate life-long physical disabilities as they grow.

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 (NASDAQ:BIIB) 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 trial.

Click here for CHIRIBOGA PRESENTATION Slides »

Click here for ISIS INVESTOR EVENT Slides »