Pfizer Licenses Spinal Muscular Atrophy Program From Repligen
In August, Repligen decided to get out of the drug discovery business and focus on manufacturing biologic drugs, so it wasn't a big surprise today when the company announced that it had licensed its spinal muscular atrophy, or SMA, program to Pfizer .
The SMA program includes a phase 1 drug candidate, RG3039, as well as backup compounds and enabling technologies. Repligen licensed the program from Families of SMA, a charity that offers support to families and researchers developing drugs to treat the disease. SMA is a neurodegenerative genetic disease that affects approximately one in 6,000 babies.
Repligen will get $5 million upfront and potentially another $65 million if future milestones are met. Pfizer also owes Repligen undisclosed royalties on any future sales of SMA drugs developed under the agreement.
Repligen will complete the first two cohorts of healthy volunteers in the ongoing phase I trial of RG3039, and then Pfizer will take over development of the program.
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