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 responsible for completing the first two cohorts of an active Phase I trial evaluating RG3039 in healthy volunteers, as well as provide certain technology transfer services to Pfizer, which will assume full responsibility for the SMA program, including any registration trials 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.
"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," said Walter C. Herlihy, Ph.D., president and chief executive officer of Repligen.
"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."