Repligen Corporation has received a $1 million milestone payment from Pfizer, Inc. under the terms of the companies’ exclusive worldwide licensing agreement for the development of compounds to treat spinal muscular atrophy (SMA). This first milestone payment was triggered by completion of specific programme activities and coincides with the successful completion of all transition obligations by Repligen.

Repligen announced the Agreement in January of this year, at which time it received an upfront payment of $5 million. Repligen remains eligible to receive up to $64 million in additional success-based milestone payments, as well as royalties on any future sales of compounds developed under the Agreement.

“Consistent with our strategic decision last August to focus on  building Repligen’s bioprocessing business while scaling back our investment in therapeutics, we out-licensed the SMA programme and have successfully completed its transition to Pfizer,” said Walter C Herlihy, president and CEO of Repligen. “We believe the Agreement  preserves the potential for  the SMA programme to deliver significant long-term upside for our shareholders.”

Repligen originally in-licensed the SMA program  from Families of SMA (FSMA), a patient organisation dedicated to supporting research to advance therapies for SMA. FSMA funded and directed the preclinical development of the program’s lead compound, RG3039, with an investment of more than $13 million. This was the first drug discovery program ever conducted specifically for SMA. The Muscular Dystrophy Association also provided critical support to Repligen's research and clinical efforts, including the conduct of a phase I b trial.

Spinal muscular atrophy is an autosomal recessive neuromuscular disease in which a defect in the SMN1 (survival motor neuron) gene results in low levels of the protein SMN and leads to progressive damage to motor neurons. It is the leading genetic cause of infant mortality and the second most common inherited neuromuscular disease, with symptoms that  typically emerge before the age of two. SMA is characterized by progressive muscle weakness leading to severe physical disability and often, early loss of life due to respiratory insufficiency.

Repligen Corporation is a life sciences company focused on the development, production and commercialization of high-value consumable products used in the process of manufacturing biological drugs.