ISIS: Clinical Trials for Its ALS Drug

| About: Ionis Pharmaceuticals, (IONS)

Isis Pharmaceuticals (ISIS) initiated a Phase 1 study of its antisense drug ISIS-SOD1Rx in patients with an inherited, aggressive form of Lou Gehrig's disease also known as familial amyotrophic lateral sclerosis (ALS). As ISIS-SOD1Rx selectively inhibits the production of SOD1, it is expected to benefit the cases of familial ALS that are related to mutant forms of superoxide dismutase, or SOD1, which constitutes approximately 20% of ALS patients suffering from familial ALS.

In preclinical studies, using the drug ISIS-SOD1Rx, Isis, in collaboration with Timothy Miller, Don Cleveland (University of California, San Diego), and Richard Smith (Center for Neurological Study) were able to lower the production of SOD1 in neurons and surrounding cells, which seemed to have prolonged life in rats that showed many symptoms of ALS. In December 2007, the FDA granted ISIS-SOD1Rx Orphan Drug designation for the treatment of ALS.

Because antisense drugs do not cross the blood-brain barrier, Isis is administering the drug through a small pump directly into the cerebral spinal fluid - a drug delivery procedure known as intrathecal infusion.

Professionals demonstrating enthusiasm about the news include:

Merit Cudkowicz, M.D., Co-Chair of the Northeast ALS Consortium and a Professor of Neurology at the Massachusetts General Hospital of Harvard Medical School, said:

"There is a desperate need for new and more substantive treatments for ALS patients. The mission of the Northeast ALS Consortium is to help advance new therapeutic options for patients with ALS, and I am very pleased to be a co-chair on this important study evaluating ISIS-SOD1Rx in people with familial ALS."

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 said:

"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." Commenting on the drug, Dr Miller said, "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. "

ISIS-SOD1Rx is the firm’s first antisense drug to enter clinical trials to treat a neurodegenerative disease and its first antisense drug to be administered directly into the central nervous system. Based on earlier preclinical studies, the firm expressed its belief that ISIS-SOD1Rx could offer an effective treatment for patients with familial ALS.

Prohost Biotech considers any successful efforts that bring rational evidence-based treatments for ALS into clinical trials to be good news. The disease is deadly and still eluding scientists, researchers and drug developers. There seems to be various etiologies for this disease with abstracts published about some discoveries, which we will tackle later in a future article. As we see with Isis’ drug ISIS-SOD1Rx, it is a fact that no one single targeted drug will help all Lou Gehrig's disease victims.

To learn more about the study click the following link: ISIS Initiates Phase 1 Clinical Trial of ISIS-SOD1Rx in Patients With ALS.

Genzyme has a right of first negotiation to license ISIS-SOD1Rx from Isis.

The ALS Association and the Muscular Dystrophy Association are providing funding for the development of ISIS-SOD1Rx. ISIS is still trading at less than $9, with a market cap of 876.63 M.

Disclosure: No Positions