Biogen Idec and Isis Pharmaceuticals have entered into an exclusive, worldwide option and collaboration under which the companies will develop and commercialize Isis’ antisense investigational drug, ISIS-SMNRx, for the treatment of spinal muscular atrophy (SMA). SMA is a genetic neuromuscular disease characterized by muscle atrophy and weakness, and it is the most common genetic cause of infant mortality. Isis’ ISIS-SMNRx is designed to compensate for the underlying genetic defect that causes SMA.
Isis will receive a $29 million upfront payment and is eligible to receive as much as $45 million in milestone payments associated with the clinical development of ISIS-SMNRx prior to licensing. Biogen Idec has the option to license ISIS-SMNRx until completion of the first successful Phase II/III trial. Isis could receive as much as another $225 million in a license fee and regulatory milestone payments, as well as royalties on sales. Isis will be responsible for global development through the completion of Phase II/III trials, with Biogen Idec providing advice on the clinical trial design and regulatory strategy. If Biogen Idec exercises its option, it will assume global development, regulatory and commercialization responsibilities.
“SMA is a heartbreaking disease — it can kill children before their second birthday and there are currently no therapies to treat the disease,” said George A. Scangos, Ph.D., chief executive officer of Biogen Idec. “The unmet need could not be any greater, the program fits with our mission to bring innovative therapies to patients with serious neurologic diseases, and Isis’ antisense compound has the potential to be a highly effective, first-to-market therapy for this deadly disease.”
“Biogen Idec’s expertise in the global development and commercialization of innovative new therapies for neurologic diseases is a great strategic fit to advance ISIS-SMNRx,” said Stanley T. Crooke, M.D., Ph.D., Isis’ chairman and chief executive officer. “We believe that, together with Biogen Idec, we will be able to expeditiously develop this investigational drug in hopes of bringing to market an effective and desperately needed treatment to improve the lives of children with SMA.”