Biogen Idec (BIIB) and Isis Pharmaceuticals have entered into an exclusive, worldwide option and collaboration agreement, pursuant to which the companies will develop and commercialize a novel antisense drug for the treatment of myotonic dystrophy type 1 (DM1).
DM1, which is also known as Steinert disease and the most common form of muscular dystrophy in adults, is a genetic neuromuscular disease characterized by progressive muscle atrophy, weakness and disabling muscle spasms.
Isis, which is developing its DM1 antisense program to correct the underlying genetic defect that causes DM1, is responsible for the discovery of a lead antisense drug candidate targeting DMPK for the treatment of DM1.
As part of the agreement, Isis will receive an upfront payment of $12m and is eligible to receive up to $59m in milestone payments associated with the clinical development of the DMPK-targeting drug prior to licensing.
Isis will also receive double-digit royalties on sales of the drug and up to another $200m in a license fee and regulatory milestone payments, while Biogen Idec will provide advice on the clinical trial design and regulatory strategy.
Biogen Idec will assume global development, regulatory and commercialization responsibilities, if it exercises its option.
Biogen Idec executive vice president of Corporate Development Steven Holtzman said Myotonic dystrophy has an identifiable genetic cause, the program fits with the company's mission to bring innovative therapies to patients with serious neurologic diseases, and Isis' antisense compound has the potential to make a real difference.
"This collaboration, which is our second with Isis, reflects the tremendous respect we have for their scientific leadership and expertise in antisense technology," Holtzman said.
Isis chief operating officer, chief financial officer and secretary B. Lynne Parshall said this collaboration allows the company to expand its pipeline of drugs for rare and severe diseases with Biogen Idec's additional resources and support.
"It also complements our new alliance with Biogen Idec for our Phase 1 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," Parshall said.
The new agreement follows a January agreement between Biogen Idec and Isis to develop and commercialize Isis' antisense investigational drug (ISIS-SMNRx) for the treatment of spinal muscular atrophy (SMA).