Knopp NeuroSciences Forms New Parent Company, Knopp Biosciences LLC to Expand Operations and Mission

Knopp NeuroSciences Forms New Parent Company, Knopp Biosciences LLC to Expand Operations and Mission

PITTSBURGH--(BUSINESS WIRE)-- Knopp Neurosciences Inc. announced today that it has formed a new parent entity called Knopp Biosciences LLC to expand operations with the goal of becoming a more broadly integrated biopharmaceutical company.

The corporate restructuring follows an August 2010 agreement under which Biogen Idec (NASDAQ:BIIB - News) secured exclusive global rights to develop and commercialize KNS-760704 (dexpramipexole) from Knopp Neurosciences ("KNS"). KNS remains the licensor of assets associated with KNS-760704 but has distributed its other assets and operations to Knopp Biosciences, with a corresponding shareholder recapitalization. The transferred assets and operations include the discovery research activities of KNS, which are directed to pharmacological mediation of mitochondrial disease mechanisms.

"Knopp Biosciences establishes a platform for corporate growth while signaling the potential to apply our discovery research in therapeutic areas beyond neuroscience," said Michael Bozik, M.D., President and CEO. "While expanding our discovery research capabilities, Knopp Biosciences will take steps toward becoming a more integrated company with both development and commercialization activities." He also noted that Knopp Biosciences will support the role of KNS in the licensing arrangement with Biogen Idec, which, as previously reported, expects to initiate Phase 3 development of KNS-760704 in amyotrophic lateral sclerosis (ALS) in the first half of 2011.

Knopp Biosciences remains on Pittsburgh's South Side at the street address of KNS. Knopp Biosciences may be found on the web at

About Knopp Biosciences LLC

Knopp Biosciences LLC, based in Pittsburgh, PA, USA, is a drug discovery and development company focused on delivering breakthrough treatments for unmet medical needs 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 under a license agreement with Biogen Idec. Knopp's leadership includes experienced neuroscience drug development and discovery executives formerly associated with major pharmaceutical companies.

About KNS-760704 (dexpramipexole)

KNS-760704 is the chirally pure form of dexpramipexole, a low molecular weight benzothiazole shown to improve mitochondrial function and to confer significant cellular protection in neurons under stress. KNS-760704 is highly orally bioavailable, water soluble, renally excreted, and only moderately protein bound. KNS-760704 was originally identified as a candidate therapy for ALS by James Bennett, M.D., Ph.D., then of the University of Virginia. 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, as well as Fast Track designation from the FDA. In August 2010, Biogen Idec secured an exclusive, worldwide license agreement to develop and commercialize KNS-760704 for the treatment of ALS and potentially other indications.

About ALS

Amyotrophic lateral sclerosis, also known as Lou Gehrig's disease and motor neuron disease, is a universally and rapidly 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.

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.