Repligen said today it received its first milestone payment, totaling $1 million, from Pfizer under their nine-month-old, up-to-$70 million exclusive worldwide licensing agreement to develop compounds designed to treat spinal muscular atrophy (SMA).
The first milestone payment followed completion of unspecified program activities, and coincided with the successful completion of all transition obligations by Repligen. Following the $1 million payment, plus the $5 million up-front payment it received when the agreement was announced in January, Repligen is still eligible for up to $64 million in additional milestone payments, plus 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 outlicensed the SMA program and have successfully completed its transition to Pfizer," Walter C. Herlihy, Ph.D., Repligen’s president and CEO, said in a statement.
Repligen originally licensed the SMA program from Families of SMA (FSMA), a patient organization focused on supporting research to advance therapies for the disease. FSMA spent $13 million to fund and direct preclinical development of the program's lead compound, the small molecule drug candidate RG3039, in what Repligen said was the first drug discovery program ever conducted specifically for SMA.
The Muscular Dystrophy Association awarded Repligen a $1.4 million grant supporting the company’s research and clinical efforts, including its carrying out a Phase Ib trial.