Pfizer Licenses Spinal Muscular Atrophy Drug Candidate from Repligen
Pfizer Inc. has entered into a worldwide licensing agreement with Repligen Corporation to advance Repligen's spinal muscular atrophy (SMA) drug program, including the clinical-stage candidate RG3039.
Pfizer Inc. has acquired exclusive worldwide rights from Repligen Corporation to further develop a drug program for spinal muscular atrophy (SMA). The agreement encompasses RG3039, a small molecule drug candidate currently in clinical development, along with backup compounds and related technologies, originally licensed by Repligen from Families of SMA (FSMA).
Under the terms of the deal, Repligen is eligible to receive up to $70 million from Pfizer. This includes an upfront payment of $5 million and potential future milestone payments totaling up to $65 million. Repligen will also receive royalties on any future sales of compounds developed through the agreement.
Repligen will complete the first two cohorts of an active Phase 1 trial evaluating RG3039 in healthy volunteers, anticipated in the first quarter of 2013. Following this, Pfizer will assume full responsibility for the SMA program, including conducting any necessary registration trials for product approval.
Spinal muscular atrophy is a rare, early-onset genetic neurodegenerative disease with a significant unmet medical need. The partnership aims to leverage Pfizer's expertise in genetic diseases and orphan drugs to accelerate the development of potential therapies for SMA patients.