Second collaboration between firms this year will focus on myotonic dystrophy type 1.

Biogen Idec is paying Isis Pharmaceuticals $12 million up front as part of an exclusive worldwide option and collaboration agreement focused on the development of an antisense drug for treating myotonic dystrophy type 1 (DM1, or Steinert disease). The deal is the second between the two firms this year. In January they inked an exclusive worldwide option and collaboration agreement focused on the development of Isis’ antisense investigational drug, ISIS-SMNRx, for the treatment of spinal muscular atrophy (SMA).

Under terms of the DM1 alliance, Isis will be responsible for the discovery of a lead antisense drug targeting the causative dystrophia myotonica-protein kinase (DMPK) gene, and carry out global development of the drug through to the completion of Phase II studies. Biogen Idec will provide support on clinical trial design and regulatory issues.

Biogen Idec retains an ongoing option to license the drug from Isis up until the end of Phase II development. Isis could receive $59 million from its partner in clinical development-related milestone payments, plus up to another $200 million in license fees and regulatory milestone payments. If Biogen Idec exercises its license to the candidate, the firm will take over all global development, regulatory, and commercialization activities and responsibilities.

“This collaboration allows us to expand our pipeline of drugs for rare and severe diseases with Biogen Idec’s additional resources and support,” remarks B. Lunne Parshall, Isis COO and CFO. “It also complements our new alliance with Biogen Idec for our Phase I program in spinal muscular atrophy.”

Isis is exploiting its antisense drug discovery platform to develop a pipeline of in-house and partnered drug candidates in a range of therapeutic areas including cardiovascular, metabolic, and inflammatory diseases, and severe and rare disorders. 

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