Biogen Idec, Knopp Neurosciences enter license agreement for KNS-760704 ALS drug candidate

Biogen Idec (NASDAQ: BIIB) and Knopp Neurosciences today announced they have entered into an exclusive, worldwide license agreement under which Biogen Idec will develop and commercialize KNS-760704 (dexpramipexole) for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, and potentially other indications.

“We are very pleased to enter into this agreement with Biogen Idec, a world leader in neurology drug development and commercialization”

KNS-760704 is a novel oral neuroprotective therapy under development. In a Phase 2 study of ALS patients conducted by Knopp, the compound achieved its primary endpoint evaluating safety and tolerability and showed favorable dose-related effects in preserving motor function and extending survival. KNS-760704 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. Biogen Idec expects to initiate a Phase 3 program of the compound in the first half of 2011.

Under the terms of the agreement, Biogen Idec will lead the development of KNS-760704 for ALS and its potential commercialization in global markets, with Knopp providing development support and conducting certain U.S. commercialization activities under the direction of Biogen Idec. As part of the transaction, Biogen Idec will purchase $60 million of Knopp stock, provide an up-front payment of $20 million and additional payments of up to $265 million based on the achievement of development, regulatory, and sales milestones. Biogen Idec will also pay tiered, double-digit royalties to Knopp on worldwide sales.

"We are very pleased to enter into this agreement with Biogen Idec, a world leader in neurology drug development and commercialization," said Michael Bozik, M.D., president and CEO of Knopp, a privately held, Pittsburgh-based biopharmaceutical company. "Biogen Idec's proven track record of delivering innovative medicines for diseases with high unmet needs makes it the ideal partner to advance KNS-760704 in ALS."

"We are excited to begin this new collaboration with Knopp on KNS-760704, a potential treatment for ALS with promising Phase 2 data," said George Scangos, Ph.D., CEO of Biogen Idec. "ALS is a devastating disorder and, with only one approved therapy, there is a tremendous need to provide more therapeutic options for patients. This disease is a natural fit for Biogen Idec, given our global capabilities in neurology, and we look forward to beginning a Phase 3 program."

Knopp has completed a two-part Phase 2 study that assessed the effects of KNS-760704 in the same subjects over two randomized, double-blind treatment periods separated by a one-month washout. These Phase 2 data were presented at the 20^th International Symposium on ALS/MND in Berlin in December 2009. In Part 1 of the study, 102 subjects received daily doses of 50 mg, 150 mg, or 300 mg of KNS-760704 or placebo for 12 weeks. KNS-760704 showed a dose-dependent trend in slowing the rate of disease progression, as measured by the difference in slopes of the ALS Functional Rating Scale-Revised (ALSFRS-R) across treatment groups, with the greatest benefit observed in the 300 mg dose group.

In Part 2 of the study, 92 subjects were re-randomized to receive daily doses of 50 mg or 300 mg of KNS-760704 for 24 weeks. In addition to results again suggesting a dose-dependent trend in slowing the rate of disease progression as measured by the ALSFRS-R, there was also a trend toward a survival benefit in the 300 mg group compared with the 50 mg group. In an exploratory test comparing subject rankings on the basis of mortality and functional outcomes, subjects in the 300 mg group had a significantly improved outcome compared with the 50 mg group.

KNS-760704 was generally well tolerated. There were no per-treatment group differences in the frequency of adverse events or serious adverse events reported in the Phase 2 trial. The most common adverse events reported more frequently than in placebo subjects were fall, nausea, and joint pain. Some cases of neutropenia were observed, which resolved when therapy was discontinued and generally did not recur when therapy was reinitiated.

Clearview Projects of Princeton, N.J., and Wolpert Associates Inc. of Summit, N.J., acted as transaction advisors to Knopp. K&L Gates LLP acted as Knopp's corporate/licensing counsel and Pepper Hamilton LLP as its patent counsel.