European Commission Awards Orphan Designation to Knopp Neurosciences' KNS-760704 in ALS
PITTSBURGH--(BUSINESS WIRE)--Knopp Neurosciences Inc. ("Knopp") today announced that the European Commission ("EC") has designated KNS-760704 as an orphan medicinal product for the treatment of Amyotrophic Lateral Sclerosis.
The EC decision, based on a favorable opinion from the European Medicines Agency (EMEA), follows the designation of KNS-760704 as an orphan drug for the treatment of ALS by the U.S. Food and Drug Administration in 2007. KNS-760704 is currently in Phase 2 clinical trials for ALS, a universally fatal neurodegenerative disease with limited treatment options.
"We are very pleased with the European Commission's designation of KNS-760704 as an orphan medicinal product and the recognition of its potential to be of significant benefit for patients with ALS," said Michael Bozik, M.D., president and CEO of Knopp. "Orphan medicinal product designation will significantly facilitate our efforts to develop a safe and effective treatment for patients suffering from this relentless disease."
To stimulate the research and development of orphan drugs, the European Union ("EU") has established a centralized procedure for the designation of orphan medicinal products and provides incentives for the development of medicinal products for rare disorders. Companies with an orphan designation for a medicinal product benefit from incentives that include fee reductions, a 10-year market exclusivity period following authorization for designated products, scientific advice to optimize development, and direct access to the EMEA centralized procedures for marketing authorization.
Knopp plans to initiate Phase 3 development of KNS-760704 in ALS in late 2009.
KNS-760704 is a low molecular weight benzothiazole shown to improve mitochondrial function and 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. Phase 2 studies of KNS-760704 in ALS are ongoing. The compound has received orphan drug designation from the U.S. Food and Drug Administration and the European Commission for the treatment of patients with 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. 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