Pfizer RG3039 Program for Spinal Muscular Atrophy Reaches Development Milestone

Originally published on September 4, 2013.

Reaching the first milestone was triggered by completion of specific program activities and coincides with the successful completion of all transition obligations to Pfizer by Repligen. Click here for the Repligen press release.

Repligen Corporation announced today that it has achieved a milestone from Pfizer, Inc. under the terms of the companies’ exclusive worldwide licensing agreement for the development of compounds to treat spinal muscular atrophy (SMA). 

RG3039 is orally bioavailable, brain-penetrant small molecule that has been shown to be an inhibitor of the mRNA decapping enzyme, DcpS. The pharmacological characterization of RG3039, reported recently in new studies in Human Molecule Genetics, demonstrates RG3039 can extend survival, improve function, and impact neuromuscular pathology in three SMA mouse models of varying disease severity. 

Repligen originally in-licensed the SMA program from Cure SMA a patient organization dedicated to supporting research to advance therapies for SMA. Cure SMA 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. 

Repligen licensed RG3039 in 2009 from Cure SMA for clinical development. In January 2013, Pfizer licensed the program, marking a significant advance for the SMA community by securing the commitment of one of the worlds largest pharmaceutical companies to develop potential treatments for SMA. The Muscular Dystrophy Association also provided critical support to Repligen’s research and clinical efforts.  The lead compound is currently being tested in Phase 1b human clinical trials.

“There is a critical need to expedite potential treatment solutions for rare diseases such as spinal muscular atrophy, where patients have such limited options,” said Jose Carlos Gutierrez-Ramos, Senior Vice President, Pfizer BioTherapeutics R&D. “This partnership will combine our expert capabilities in advancing molecules for genetic diseases with Repligen’s leading SMA program.”

The Cure SMA research model is to fund early stage drug discovery programs for SMA and then partner with companies for later stages and accelerated clinical development.  At the earliest stages of drug development, programs have less than a 1% chance of success. This inherent risk along with small patient populations has traditionally hindered industry from working on orphan diseases.

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