UPDATE: Pfizer and Repligen to Collaborate on SMA Therapy Development

The biotechnology firm Repligen today announced it has entered into an agreement with the global pharmaceutical company Pfizer to advance Repligen’s spinal muscular atrophy (SMA) program, which includes RG3039, an experimental drug for SMA whose development MDA has supported.

"We believe this collaboration with Pfizer, a leading pharmaceutical company with specialized efforts in orphan and genetic diseases, has the potential to accelerate the development of therapies for SMA," said Repligen's CEO Walter Herlihy in a Jan. 3, 2013, company press release.

RG3039 is designed to increase levels of the SMN (survival of motor neuron) protein, which is deficient in SMA, by interfering with an enzyme called DcpS (scavenger decapping enzyme). The drug is now being tested in healthy volunteers in a phase 1b trial.

A $1.4 million grant from MDA is helping to support the phase 1b trial and supported a phase 1a trial and laboratory research required for advancing RG3039 to human testing. Families of SMA also has provided substantial support for development of this drug.

"We’re extremely pleased that Pfizer has given its vote of confidence to Repligen’s spinal muscular atrophy program, including RG3039," said Valerie Cwik, MDA's executive vice president of research and medical director. "This is the goal of translational research programs such as MDA’s — providing seed grants that allow small, innovative companies like Repligen to bring a drug candidate to a stage where a large pharmaceutical firm like Pfizer will develop it further and, we hope, ultimately bring it to market."

The U.S. Food and Drug Administration (FDA) has granted fast track and orphan drug designations to RG3039. A fast track designation allows for faster review of drugs that treat serious diseases and fill an unmet need, while orphan drug status provides economic incentives to companies developing treatments for rare diseases.

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