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Biogen and Isis to research muscle weakness condition

Deal to develop myotonic dystrophy treatment could be worth up to $271m for Isis

Biogen Idec is collaborating with Isis Pharmaceuticals to develop a new treatment for a rare muscle disorder after signing a deal that could be worth $271m to Isis.

US-based Biogen will pay an initial upfront fee of $12m under an agreement that will initially see Isis lead efforts into the discovery of a novel antisense drug for the treatment of myotonic dystrophy type 1 (DM1), also known as Steinert disease.

Isis' responsibility over any compound's development will continue through to completion of phase 2 clinical trials, with Biogen providing advice on the clinical trial design and regulatory strategy.

Depending on how development progresses, Isis will then be eligible to receive up to $59m in milestone payments prior to licensing, with another $200m available if Biogen chooses to license the drug following the completion of phase II trials. Isis will also receive royalties on the drug's sales if it makes it to market.

The condition at the heart of the collaboration, DM1, is a genetic disease that is the most common form of muscular dystrophy in adults.

It is caused by a genetic defect in the dystrophia myotonica-protein kinase (DMPK) gene that can lead to a halt in the production of proteins needed for normal cellular function.

The condition affects 150,000 people in the US, Europe and Japan, with multiple sufferers often drawn from the same family. Symptoms of the condition include muscle wasting, weakness and disabling muscle spasms.

“The unmet need [in DM1] is great, and there are currently no therapies to slow or stop progression of the disease,” said Steven Holtzman, executive VP of corporate development at Biogen.

“Myotonic dystrophy has an identifiable genetic cause, the programme 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.”

The collaboration is Biogen's second with Isis following a deal worth up to $299m to develop and commercialise an investigational compound for the treatment of spinal muscular atrophy (SMA).

This partnership also uses Isis' antisense technology, which involves the use of nucleic acids to target RNA to stop the production of harmful proteins in genetic conditions.

3rd July 2012

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