Knopp Neurosciences Receives FDA Fast Track Designation for KNS-760704 in ALS
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PITTSBURGH--(Business Wire)-- 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. About Knopp Neurosciences Inc. Knopp Neurosciences is a drug discovery and development company focused on delivering breakthrough treatments for neurological disorders through innovation, experience, and partnership. The company`s lead product candidate is KNS-760704, an orally bioavailable small molecule in development for the treatment of ALS. Knopp`s leadership includes experienced neuroscience drug development and discovery executives formerly associated with major pharmaceutical companies. Knopp`s financing has been led by Saturn Capital Inc. of Boston as placement agent and Saturn Partners II as lead funder. This press release contains "forward-looking statements," including statements relating to Knopp`s planned regulatory filings and clinical development programs for KNS-760704. All forward-looking statements are based on management`s current assumptions and expectations and involve risks, uncertainties and other important factors, specifically including the uncertainties inherent in clinical trials and product development programs, the availability of funding to support continued research and studies, the availability or potential availability of alternative therapies or treatments, the availability of patent protection for the discoveries and strategic alliances, as well as additional factors that may cause Knopp`s actual results to differ from our expectations. There can be no assurance that KNS-760704 will be successfully developed or manufactured or that final results of clinical studies will be supportive of regulatory approvals required to market the products. Knopp undertakes no obligation to update or revise any such forward-looking statements, whether as a result of new information, future events or otherwise. Knopp Neurosciences Inc. Tom Petzinger, 412-488-1776 Executive Vice President, Business Development and Public Affairs firstname.lastname@example.org Copyright Business Wire 2009