Weston, Mass.-based Biogen Idec and Isis Pharmaceuticals of Carlsbad, Calif., have entered into an exclusive, worldwide option and collaboration agreement to develop and commercialize a novel antisense drug for the treatment of myotonic dystrophy type 1 (DM1), also known as Steinert disease.

"Genetic testing can aid in identifying people at risk of developing DM1, but there are no treatments available to delay the onset of symptoms or slow down the progression,” said Charles A. Thornton, M.D., School of Medicine at University of Rochester Medical Center. “Because the disease-causing substance in DM1 is a toxic RNA, the disease is a good target for Isis' antisense approach to selectively target the toxic RNA within the cell, removing it and restoring normal cell function."

Isis will receive an upfront payment of $12 million and is responsible for the discovery of a lead antisense drug candidate targeting DMPK for the treatment of DM1. Isis is eligible to receive up to $59 million in milestone payments associated with the clinical development of the DMPK-targeting drug prior to licensing. Biogen Idec has the option to license the drug from Isis up through the completion of the phase II trial. Isis could receive up to another $200 million in a license fee and regulatory milestone payments. In addition, Isis will receive double-digit royalties on sales of the drug. Isis will be responsible for global development of the drug through the completion of phase II clinical 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.

The collaboration follows on a worldwide option and collaboration agreement between Biogen Idec and Isis, which was announced in January, to develop and commercialize Isis' antisense investigational drug, ISIS-SMNRx, for the treatment of spinal muscular atrophy (SMA).

"This collaboration allows us to expand our pipeline of drugs for rare and severe diseases with Biogen Idec's additional resources and support,” said B. Lynne Parshall, J.D., COO, CFO and secretary, Isis Pharmaceuticals. “It also complements our new alliance with Biogen Idec for our phase I program in spinal muscular atrophy, or SMA. As with SMA, we are using our antisense technology in a unique manner to treat another devastating disease. Biogen Idec is an ideal partner for these programs with its expertise in neurodegenerative disease and global reach to help bring these therapies successfully to patients who have no treatment options."