Biogen inks $299m deal for rare genetic disorder drug
pharmafile | January 5, 2012 | News story | Research and Development, Sales and Marketing | Biogen, Isis Pharma
Biogen has signed a new deal with drug discovery firm Isis for its spinal muscular atrophy drug ISIS-SMNRx.
Biogen will pay Isis $29 million upfront and up to $270 million in milestone payments for the drug candidate, which is now in phase I studies.
The drug is a treatment for spinal muscular atrophy (SMA), a genetic neuromuscular disease characterised by muscle atrophy and weakness, and it is the most common genetic cause of children’s death.
Isis will also receive double-digit royalties on sales of the drug, and will be responsible for global development of ISIS-SMNRx through the completion of late-stage registration trials.
If Biogen uses its option within the contract, it will assume global development, regulatory and commercialisation responsibilities.
Stanley Crooke, chief executive of Isis, said: “Biogen Idec’s expertise in the global development and commercialisation of innovative new therapies for neurologic diseases is a great strategic fit to advance ISIS-SMNRx.
“This alliance is consistent with our business strategy to develop antisense drugs to proof-of-concept with a knowledgeable partner that is committed to supporting the rapid development of the drug.
“Given the severity of the unmet need in SMA, our proof-of-concept studies should also serve as the registrational trials for ISIS-SMNRx.”
George A. Scangos, chief executive of Biogen Idec said: “SMA is a heartbreaking disease – it can kill children before their second birthday and there are currently no therapies to treat the disease,”
Scangos said the drug fitted in with Biogen’s strategy, and could be a first-to-market treatment for the deadly disease.
“We have the utmost respect for Isis’ scientific leadership and expertise in antisense technology, and we have crafted a collaboration that brings together our two companies’ strengths toward a common goal.”
SMA
SMA is a severe genetic disease that affects approximately 30,000-35,000 patients in the US, Europe and Japan. One in 50 people, the equivalent of about six million people in the US, are carriers of the SMA gene. Carriers experience no symptoms and do not develop the disease, but when both parents are carriers there is a one in four chance that their child will have SMA.
SMA is caused by a loss of, or defect in, the survival motor neuron 1 (SMN1) gene leading to a decrease in the survival motor neuron (SMN) protein. SMN is critical to the health and survival of nerve cells in the spinal cord responsible for neuromuscular growth and function. The severity of SMA correlates with the amount of SMN protein. Infants with the most severe Type I SMA form of the disease, produce very little SMN protein and have a life expectancy of less than two years. Children with Type II have greater amounts of SMN protein but still have a shortened lifespan and are never able to stand independently. Children with Type III have a normal lifespan but accumulate life-long physical disabilities as they grow.
ISIS-SMNRx is part of the antisense drug technology field that targets RNA molecules and effectively turns a faulty gene on or off.
ISIS-SMNRx is designed to overcome the SMN1 fault by altering the splicing of a closely related gene (SMN2) that leads to the increased production of fully functional SMN protein.
Isis already has numerous other collaborations based on its antisense platform, including its lead candidate, cholesterol treatment Mipomersen, which it is developing with Genzyme and is currently in phase III.
Other companies are also working on treatments for SMA. In December, Roche signed a $490 million deal to partner with PTC Therapeutics on its programme for SMA to bolster its rare diseases portfolio.
Ben Adams
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