Isis Pharmaceuticals Receives $7M Funding To Advance Clinical Trials Of ISIS-SMN Rx Spinal Muscular Atrophy Drug

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by Charles Moore |

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Carlsbad, California-based Isis Pharmaceuticals, Inc. has received a isispharnlogo$7 million milestone payment from Biogen Idec, to advance an ongoing open-label extension clinical study of the drug ISIS-SMNRx in children with spinal muscular atrophy (SMA).

The study of ISIS-SMNRx is open to children with SMA who have completed dosing in previous Isis studies. In this particular study, juvenile subjects male and female aged 2 to 15 years of age with SMA will be administered a 12 mg dose of ISIS -SMNRx at six month intervals for the study’s duration.

The trial will be conducted at several locations across the U.S. including Boston Children’s Hospital in Boston, Massachusetts; Columbia University Medical Center in New York; UT Southwestern Medical Center at the Children’s Medical Center in Dallas, Texas; and the University of Utah School of Medicine in Salt Lake City, Utah.

More information about details of the clinical trial can be found can be found on Isis’s Website here and at (search for ISIS-SMNRx).

ISIS-SMNRx is designed as a pharmaceutical agent whose action is to alter the splicing of a closely related gene (SMN2) in order to increase the body’s production of fully functional SMN protein. Orphan drug status and fast track authorization have been granted to ISIS-SMNRx by the FDA for treatment of patients afflicted with SMA.

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biogenideclogoIsis is collaborating with Biogen Idec on development and potential commercialization the of the compound ISIS-SMNRx being investigated in the clinical trial for treating all SMA types. Under terms of a January 2012 agreement, Isis is responsible for handling global development of ISIS-SMNRx, with Biogen Idec having the option of licensing the compound through to first successful completion of the Phase 2/3 trial or of two Phase 2/3 studies. Isis is conducting two Phase 3 trials under FDA authorization for special protocol assessments (SPA), ie: written agreement between the FDA and a drug’s sponsor confirming the adequacy of a clinical trial protocol consistent with current scientific and regulatory requirements for new drug candidate applications.

Isis is also acknowledging support for its ISIS-SMNRx trials from the Muscular Dystrophy Association, the SMA Foundation, Cure SMA, as well as intellectual property licensed from Cold Spring Harbor Laboratory and the University of Massachusetts Medical School.

SMA is a severe and devastating neuromuscular disease, and one of the most common fatal genetic disorders — occurring in 1 in every 6,000 to 10,000 live births, and affecting an estimated 30,000-35,000 patients in the United States, Europe and Japan. SMA is caused by a deficiency or or defect in the survival motor neuron 1 (SMN1) gene that leads a deficiency of 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. Approximately one in 50 people, the rough equivalent of 6 million people in the United States, carry a defective SMN1 gene unable to produce fully functional SMN protein.

Carriers themselves experience no symptoms and don’t develop the disease, but in cases where both parents are carriers, a one in four chance exists of conceiving a child who will develop SMA. The severity of SMA correlates with the amount of SMN protein.

The disease presents with variable degrees of severity and life expectancy, correlating with the amount of SMN protein being produced. Symptoms manifest as progressive muscle weakness that results in respiratory and mobility impairment. The four SMA categories are named for the age of the victim at initial onset of muscle weakness and related symptoms: Type I (Infantile), Type II (Intermediate), Type III (Juvenile) and Type IV (Adult onset).

Life expectancy and severity vary by SMA type. Type I has the grimmest prognosis with a life expectancy raging no greater than 2 years from birth, while Type IV sufferers can have up to normal lifespans but with increasing weakness of proximal muscles of the extremities, that will eventually result in mobility inhibitions. Currently treatment options for SMA patients are limited, and the is a high unmet need for new therapeutic options, both to address symptoms directly and to slow disease progression.

The four collaborations between Biogen Idec and Isis to date have been focused on leveraging antisense technology to advance development of treatments for neurological and neuromuscular disorders. The complimentary alliance harnesses Isis’ expertise in antisense technology in evaluating potential neurological targets and discovery of antisense drugs with Biogen Idec’s capacity to develop therapies for neurological disorders. Fortune 500 firm Biogen Idec heralds itself as the world’s oldest independent biotechnology company with more than $5 billion in revenues. Under terms of the cooperative arrangement, Isis is primarily responsible for drug discovery and early development of antisense therapies.

The agreement also grants Biogen Idec the option to license each antisense program at a particular stage in its development. Current development-stage programs include antisense drugs to treat patients with spinal muscular atrophy (SMA), ISIS-SMNRx, myotonic dystrophy type 1 (DM1), ISIS-DMPKRx, and an undisclosed neurodegenerative disease, ISIS-BIIB3Rx.

For more information on the trial, visit:

Additional information about Isis ca be found at at:

Isis Pharmaceuticals Inc.
Biogen Idec Inc.