Biogen Idec has bagged exclusive, worldwide rights to Knopp Neuroscience’s KNS-760704 (dexpramipexole) for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, for up to $345 million. 

KNS-760704 is a novel oral neuroprotective therapy with orphan drug designation in the US, which in Phase II trials was shown to be safe and tolerable, with a favourable dose-related effect in preserving motor function and extending survival in patients with the disease.

As per the terms of the deal, Biogen Idec will take the reigns of the drug’s development and commercialisation in ALS, and potentially other indications, with Knopp providing support and certain US commercialisation activities.

In return for rights to the drug, Biogen will buy $60 million of Knopp stock alongside an up-front payment of $20 million and, subsequently, up to $265 million on the achievement of certain development, regulatory, and sales milestones. In addition, Knopp stands to receive tiered, double-digit royalties on global sales of the drug.

"ALS is a devastating disorder and, with only one approved therapy, there is a tremendous need to provide more therapeutic options for patients,” said Biogen’s chief executive George Scangos, explaining the firm’s interest in the drug.