Biogen Idec and Isis Pharmaceuticals are teaming up again, this time to develop an antisense drug for myotonic dystrophy type 1.
DM1, also known as Steinert disease, is the most common form of muscular dystrophy in adults and is caused by a defect in the dystrophia myotonica-protein kinase gene. It is estimated to affect 150,000 patients in the USA, Europe and Japan and is passed from parent to child with each subsequent generation experiencing more severe disease earlier in life.
Cashwise, Isis will get an upfront fee of $12 million and is responsible for the discovery of a lead antisense drug candidate targeting DMPK for the treatment of DM1. It is also eligible to receive up to $59 million in milestone payments associated with clinical development, while has the option to license the drug through the completion of the Phase II trial. After that, Isis could therefore receive up to another $200 million and will receive double-digit royalties.
Steven Holtzman, head of corporate development at Biogen, noted that "the unmet need is great and there are currently no therapies to slow or stop progression of the disease". DM1 has "an identifiable genetic cause, the program fits with our mission to bring innovative therapies to patients with serious neurologic diseases, and Isis’ antisense compound has the potential to make a real difference", he added.
The two companies already have a collaboration in place which was unveiled in January focusing on spinal muscular atrophy.