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Pfizer acquires spinal muscular atrophy research programme

Gains rights to drug candidate RG3039 in deal with Repligen that could be worth up to $70m

Pfizer headquartersPfizer has licensed the rights to a biologic research programme that aims to develop new treatments for the muscle wasting condition spinal muscular atrophy (SMA).

Although the deal could be worth up to $70m to Repligen based on milestone payments, Pfizer has been cautious in its upfront payment, and the US company will initially receive just $5m.

The main candidate involved in the programme is RG3039, which will undergo phase I trials carried out by Repligen in the first quarter of 2013.

Once these have been completed, Pfizer will then assume full responsibility for the SMA programme, which also includes backup compounds and enabling technologies.

According to Repligen, SMA is the leading cause of infant mortality and the second most common inherited neuromuscular disease, with a defect in a person's SMN1 gene leading to low levels of a protein necessary to protect motor neurons and keep muscles from weakening.

Despite its gravity, the condition is still relatively rare, and Pfizer will benefit from the incentives provided by the US Food and Drug Administration and the European Medicines Agency, both of which have designated orphan status for the drug.

In addition, RG3039 has also been given fast-track status in the US, further speeding up its approval process if it first proves a success in clinical trials.

“There is a critical need to expedite potential treatment solutions for rare diseases such as spinal muscular atrophy, where patients have such limited options,” said Jose Carlos Gutierrez-Ramos, senior VP at Pfizer BioTherapeutics R&D.

The partnership was welcomed by support group and research funder Families of SMA, who provided initial funding for the SMA programme before it was in-licensed by Repligen.

The organisation's research director Jill Jarecki said: “This licensing deal demonstrates the innovative collaborations that Families of SMA has successfully implemented between non-profit, biotech and big pharma.

“These partnerships are critical for the development of new treatments for an orphan disease such as SMA.”

4th January 2013

From: Research

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