Knopp Neurosciences Receives FDA Fast Track Designation For KNS-760704 In ALS

Main Category: Neurology / Neuroscience
Article Date: 02 Sep 2009 - 22:00 PDT

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Knopp Neurosciences Inc. announced that it received Fast Track designation from the U.S. Food and Drug Administration (FDA) for the development of KNS-760704 in amyotrophic lateral sclerosis (ALS).

Knopp has completed the randomized, placebo-controlled portion of its Phase 2 studies of KNS-760704 in 102 ALS patients and expects to initiate Phase 3 studies in the U.S. and Europe in 2010. Subjects who completed the Phase 2 program were offered enrollment in an ongoing, 48-week open-label safety extension in which all participants are receiving the highest dose tested of KNS-760704.

The Fast Track Program was created to facilitate the development and expedite the review of new drugs with the potential to meet unmet needs in serious or life-threatening conditions. ALS is a universally fatal disease of progressive paralysis with limited treatment options.

"Knopp Neurosciences is very pleased to receive Fast Track designation and the recognition that KNS-760704 holds the potential to address unmet needs in ALS," said Michael Bozik, M.D., president and CEO of Knopp. "We are pursuing every available opportunity to accelerate the development of KNS-760704, and will take full advantage of the opportunities under Fast Track to work with the FDA in designing a program to demonstrate the drug's safety and efficacy."

About KNS-760704

KNS-760704 is a low molecular weight benzothiazole shown to improve mitochondrial function and to confer significant cellular protection in neurons under stress. The chirally pure form of the synthetic benzothiazole (6R)-2-amino-4,5,6,7-tetrahydro-6-(propylamino)benzothiazole, KNS-760704 is highly orally bioavailable, water soluble, renally excreted, and only moderately protein bound. In Phase 1 studies, the compound was shown to be safe and well tolerated in healthy human subjects. KNS-760704 has received orphan drug designation from the U.S. Food and Drug Administration and the European Medicines Agency for the treatment of patients with ALS.

About ALS

Amyotrophic lateral sclerosis, also known as Lou Gehrig's disease and Charcot's sclerosis, is a rapid, universally fatal neurodegenerative disorder characterized by progressive muscle weakness and wasting. ALS affects adults in the prime of life and creates a substantial burden for caregivers. U.S. prevalence is approximately 20,000 and the global incidence is approximately two per 100,000. Worldwide prevalence estimates of ALS range from 2 to 9 people per 100,000. Only one drug has been approved for the treatment of ALS. Life expectancy after symptom onset is usually three to five years.

Source
Knopp Neurosciences Inc.