Pfizer licenses spinal muscular atrophy programme from Repligen

Pfizer has entered into an exclusive worldwide licensing agreement with Repligen for quinazoline, a new therapy for spinal muscular atrophy (SMA).

Originally created by Families of SMA, a group dedicated to developing a cure for the disease, the quinazoline project represents the first industrial drug programme for SMA ever conducted, with work having commenced in 2000.

Repligen will be responsible for completing the first two cohorts of an active phase I trial evaluating the compound among healthy volunteers, which it anticipates will occur during the first quarter of 2013.

Subsequent technology transfer services will allow Pfizer to assume full responsibility of the SMA programme – which has received orphan drug designations in the US and EU – moving forward.

Jose Carlos Gutierrez-Ramos, senior vice-president at Pfizer BioTherapeutics, said: "There is a critical need to expedite potential treatment solutions for rare diseases such as spinal muscular atrophy, where patients have such limited options."

This comes after Pfizer agreed a new biologic therapy alliance with Halozyme Therapeutics last month.