Repligen, a life sciences company based in Waltham, Mass., has entered into an exclusive worldwide licensing agreement with global pharmaceutical company Pfizer 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. Repligen will be 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 CEO 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."

Repligen will be 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 E.U.

"This licensing deal demonstrates the innovative collaborations that Families of SMA has successfully implemented between non-profit, biotech and big pharma," stated Jill Jarecki, Ph.D., research director, Families of SMA. "These partnerships are critical for the development of new treatments for an orphan disease such as SMA. We are extremely pleased to see Pfizer taking the lead on the development and commercialization of the SMA program, following Repligen's development work and FSMA's original investment."

Families of SMA, a patient organization dedicated to funding research to advance therapies for 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.