Repligen Corporation, a life sciences company, entered into an exclusive worldwide licensing agreement with Pfizer Inc. to advance Repligen’s spinal muscular atrophy (SMA) program. The program was 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 responsible for completing the first two cohorts of an active Phase I trial evaluating RG3039 in healthy volunteers, which it expects to complete during the first quarter of 2013. Repligen will also provide certain technology transfer services to Pfizer, which will assume full responsibility for advancing the SMA program, including the conduct of any registration trials necessary for product approval. Repligen previously received US Orphan Drug and Fast Track designations for RG3039 for the treatment of SMA, as well as Orphan Medicinal Product designation in the EU.
