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Biogen agrees $299m deal for Isis' muscle weakness drug

Investigational orphan drug ISIS-SMNRx is a potential treatment for spinal muscular atrophy

Biogen Idec has agreed a deal with Isis Pharmaceutical to develop and commercialise an early-stage compound for the treatment of spinal muscular atrophy (SMA).

Isis, a US company specialising in antisense drug development, will receive an upfront payment of $29m, but with further R&D and regulatory milestones this could push the potential total worth of the agreement to $299m.

The compound, ISIS-SMNRx, has been developed to treat all types of childhood SMA, a genetic neuromuscular disease that causes muscle atrophy and weakness.

It was granted orphan drug status and fast-track designation by the US Food and Drug Administration (FDA) due to the unmet clinical need in its therapy area.

A child is born with SMA for every one out of 10,000 births worldwide, according to Isis, and in its most severe form the condition can significantly shorten the lifespan of a child. It is also the most common genetic cause of death in children.

ISIS-SMNRx attempts to affect the genetic causes of the condition to produce greater amounts of the survival motor neuron (SMN) protein, which is deficient in people with SMA.

The protein helps with the health and survival of nerve cells in the spinal cord responsible for neuromuscular growth and function.

The first phase I trial involving use of the compound in children with SMA was initiated in December, 2011.

The study is designed to assess the safety, tolerability and the pharmacokinetic profile of the drug in children between the ages of two and 14 who are medically stable.

Isis will be responsible for global development of the treatment until the completion of phase II/III registration clinical trials, after which Biogen will take over global development, regulatory and commercialisation responsibilities.

Biogen has the option to license ISIS-SMNRx, until completion of the first successful phase II/III trial.

5th January 2012

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