Biogen Idec is paying about $80 million up front for exclusive, worldwide rights to Knopp Neurosciences’ mid-stage candidate for amyotrophic lateral sclerosis (ALS) called KNS-760704. The firm agreed to pay an additional $265 million in development, regulatory, and sales milestones and will be able to leverage the drug in other indications as well.
The up-front fee is split into a $60 million purchase of Knopp stock and a $20 million cash payment. Biogen Idec will lead further development and potential commercialization of KNS-760704 in global markets. Knopp will provide development support and conduct certain U.S. commercialization activities under Biogen Idec’s direction. The company will receive tiered, double-digit royalties from Biogen Idec on worldwide sales.
Biogen Idec expects to initiate a Phase III program in the first half of 2011. In a Phase II ALS study, KNS-760704 achieved its primary endpoint evaluating safety and tolerability and showed favorable dose-related effects in preserving motor function and extending survival.
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. It 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. Certain rights licensed by Knopp to Biogen Idec include rights licensed from the University of Virginia Patent Foundation. The compound received orphan drug designation from the FDA and the European Commission for the treatment of ALS as well as Fast Track designation from the FDA.