Squaring off against SMA

WALTHAM, Mass.—Repligen Corp. has kicked off 2013 with theestablishment of an exclusive worldwide licensing agreement with Pfizer Inc.for the advancement of Repligen's spinal muscular atrophy (SMA) program, which was originally in-licensed from Families of SMA. The program includes RG3039, asmall-molecule drug candidate in clinical development for the treatment of SMA, in addition to backup compounds and related technologies.

"There is a critical need to expedite potential treatmentsolutions for rare diseases such as spinal muscular atrophy, where patientshave such limited options," Jose Carlos Gutierrez-Ramos, senior vice presidentof Pfizer BioTherapeutics R&D, said in a press release regarding the deal. "This partnershipwill combine our expert capabilities in advancing molecules for geneticdiseases with Repligen's leading SMA program."

Per the terms of the agreement, Repligen is entitled toreceive an upfront payment of $5 million as well as total potential milestonepayments of up to $65 million. Repligen will also be eligible for royalties onany future sales of SMA compounds that result from the agreement. Repligen willbe responsible for completing the first two cohorts of an active Phase I trialevaluating RG3039 in healthy volunteers. Repligen will provide certaintechnology transfer services to Pfizer, who will then take over fullresponsibility for moving the program forward, including conducting anynecessary registration trials in the pursuit of product approval.

"This agreement is consistent with the strategic decision weannounced in August 2012 to focus Repligen's internal efforts on the growth ofour bioprocessing business, while seeking external partners for our therapeuticdevelopment programs," Walter C. Herlihy, Ph.D., president and CEO of Repligen,said in a statement. "We believe this collaboration with Pfizer, a leadingpharmaceutical company with specialized efforts in orphan and genetic diseases,has the potential to accelerate the development of therapies for SMA."

SMA is an orphan neurodegenerative genetic disease thatusually presents in early on in childhood or adolescence. The disease causesmuscle damage and increasing muscle weakness, and after Duchenne musculardystrophy, is the second leading cause of neuromuscular disease, withapproximately four out of every 100,000 people presenting with the disease.Repligen has received Orphan Drug and Fast Track designations for RG3039 fromthe U.S. Food and Drug Administration for the treatment of SMA, as well asOrphan Medical Product designation in the European Union.

"This licensing deal demonstrates the innovativecollaborations that Families of SMA has successfully implemented betweennon-profit, biotech and big pharma," said Jill Jarecki, Ph.D., researchdirector for Families of SMA, in a press release. "These partnerships arecritical for the development of new treatments for an orphan disease such asSMA. We are extremely pleased to see Pfizer taking the lead on the developmentand commercialization of the SMA program, following Repligen's development workand FSMA's original investment."

Families of SMA funded and directed preclinical developmentof RG3039 with more than $13 million in investment, and Repligen's research andclinical work, including the current Phase Ib trial, have been partiallysupported by a grant from the Muscular Dystrophy Association of $1.4 million.