Repligen and Pfizer entered into a licensing agreement to advance Repligen’s spinal muscular atrophy (SMA) program, originally in-licensed from Families of SMA, a patient organization dedicated to funding research to advance therapies for SMA. The SMA program includes RG3039, a small molecule drug candidate in clinical development for SMA, as well as backup compounds and enabling technologies.
Repligen is entitled to receive up to $70 million from Pfizer, commencing with an up-front payment of $5 million and total potential future milestone payments of up to $65 million as well as royalties on any future sales of SMA compounds developed under the agreement. SMA is an orphan neurodegenerative genetic disease that presents early in life.
“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 Walter C. Herlihy, Ph.D., president and CEO of Repligen. “We believe this collaboration with Pfizer . . . has the potential to accelerate the development of therapies for SMA.”
Under the agreement, Repligen is responsible for completing the first two cohorts of an active Phase I trial evaluating RG3039 in healthy volunteers, which it anticipates will occur during the first quarter of 2013. Repligen will also provide certain technology transfer services to Pfizer, who will then assume full responsibility for the SMA program moving forward including the conduct of any registration trials necessary for product approval. Repligen has previously received U.S. Orphan Drug and Fast Track designations for RG3039 for the treatment of SMA, as well as Orphan Medicinal Product designation in the EU.
Families of SMA funded and directed the preclinical development of RG3039 with an investment of more than $13 million. This was the first drug discovery program ever conducted specifically for SMA. Repligen’s research and clinical efforts, including the current Phase Ib trial, have been partially supported by a $1.4 million grant from the Muscular Dystrophy Association.