Isis Pharmaceuticals Initiates Phase 1 Clinical Study in Patients with Spinal Muscular Atrophy.

Isis Pharmaceuticals, Inc. announced today that it has initiated a Phase 1 study of ISIS-SMNRx in patients with spinal muscular atrophy (SMA). Isis is developing ISIS-SMNRx as a potential treatment for all types of SMA.
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Dec. 19, 2011 - PRLog -- The Phase 1 study of ISIS-SMNRx is a single-dose, dose-escalation study designed to assess the safety, tolerability and pharmacokinetic profile of the drug in children with SMA between the ages of 2-14 who are medically stable.  In this study, ISIS-SMNRx will be administered intrathecally as a single injection directly into the spinal fluid.  Intrathecal administration of an antisense drug, ISIS-SOD1Rx, has been shown to be safe and well tolerated in an ongoing Phase 1 study in patients with amyotrophic lateral sclerosis.

“SMA represents a serious unmet medical need with no currently available treatments.   ISIS-SMNRx is our first drug to intervene in the splicing of RNA to increase the production of a normal protein, SMN.  Together with Dr. Krainer’s lab, we have validated the antisense approach to treating this disease and are now advancing this program into clinical studies,” said C. Frank Bennett, Ph.D., Senior Vice President of Research at Isis.  “We are committed to quickly developing this drug and are finalizing what we believe will be a rapid development path for this drug in all types of SMA.  Once we evaluate ISIS-SMNRx as a single-dose in children with SMA, we will move to multiple-doses in our Phase 1 studies and eventually evaluate the drug in Phase 2 studies in children with SMA, including infants with Type I SMA.”

“Our strategy to treat SMA relies on a simple, powerful antisense method that boosts SMN protein levels by fixing a genetic RNA splicing glitch.  Working with Isis, we have successfully redirected splicing to increase functional SMN production.  We have thoroughly validated this approach in multiple animal models, observing marked improvement in modifying the disease course in both mild and severe models of SMA,” said Adrian Krainer, Ph.D., Professor of Molecular Genetics at Cold Spring Harbor Laboratory in Long Island, NY.  “We look forward to translating this important discovery into an effective treatment for this serious disease.” Dr. Krainer is a member of the FSMA Scientific Advisory Board.

“We are very pleased to see the great milestone of a disease-modifying drug treatment advancing into clinical trials in SMA patients,” said Kenneth Hobby, President of Families of SMA.  “Our community has worked for a long time to reach the goal of moving specific therapies for SMA from the bench and into the clinic.  This has been made possible by close interactions between basic researchers, families, clinicians, and industry.  Families of SMA applauds ISIS for investing in and leading drug developments efforts for this devastating, orphan disease.”

Isis Pharmaceuticals exclusively licensed certain intellectual property from the University of Massachusetts to develop this new drug for Spinal Muscular Atrophy.  Families of SMA provided over $500,000 in funding support for the University of Massachusetts' research program responsible for creating this intellectual property.  

“SMA is a terrible disease and the leading genetic cause of infant mortality.  The ability of our drugs to specifically target RNA and drive the production of SMN may be able to compensate for the underlying genetic defect and offer some therapeutic benefit to patients with SMA.  We are grateful for the support provided by Families of SMA in funding early research on SMA, which has significantly expanded the basic science of SMA and provided a roadmap to developing the first targeted therapy to treat SMA,” said Frank C. Bennett, Ph.D., Senior Vice President of Research at Isis Pharmaceuticals.

The United States Food and Drug Administration granted Orphan Drug Designation with Fast Track Status to ISIS-SMNRx for the treatment of patients with SMA.  For more information on the Phase 1 study of ISIS-SMNRx please visit:

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Families of Spinal Muscular Atrophy is dedicated to creating a treatment and cure by:
- Funding and advancing a comprehensive research program
- Supporting SMA families through networking, information and services
- Improving care for all SMA patients
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