Repligen Corporation (NASDAQ:RGEN) announced today that it has entered into an exclusive worldwide licensing agreement with Pfizer Inc. to advance Repligen's spinal muscular atrophy (SMA) program, originally in-licensed from Families of SMA (FSMA). The SMA program includes RG3039, a small molecule drug candidate in clinical development for SMA, as well as backup compounds and enabling technologies. Under the terms of the agreement, Repligen is entitled to receive up to $70 million from Pfizer, commencing with an upfront 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 Chief Executive Officer of Repligen. "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."
"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 Vice President, Pfizer BioTherapeutics R&D. "This partnership will combine our expert capabilities in advancing molecules for genetic diseases with Repligen's leading SMA program."