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Pfizer snags orphan drug in $70M licensing deal with Repligen

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About the same time Pfizer ($PFE) was outlicensing a chronic migraine drug to the upstart Labrys, another one of its deal teams was bringing in an orphan drug program from Waltham, MA-based Repligen. The biotech today announced that it grabbed a small $5 million up front for its Phase I drug for spinal muscular atrophy (SMA) along with $65 million in promised milestones for a successful development effort at the giant pharma company.

The therapy for the orphan neurodegenerative genetic disease caught the attention of Jose Carlos Gutierrez-Ramos, senior vice president, Pfizer BioTherapeutics R&D. Once Repligen is finished with its Phase I work in the next few months, Pfizer will take over the research work on the treatment, which Repligen had inlicensed from Families of SMA.

"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 Gutierrez-Ramos. "This partnership will combine our expert capabilities in advancing molecules for genetic diseases with Repligen's leading SMA program."

"This agreement is consistent with the strategic decision we announced in August 2012 to focus Repligen's internal efforts on the growth of our bioprocessing business, while seeking external partners for our therapeutic development programs," said Repligen CEO Walter C. Herlihy. "We believe this collaboration with Pfizer, a leading pharmaceutical company with specialized efforts in orphan and genetic diseases, has the potential to accelerate the development of therapies for SMA."

- here's the press release

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