Pfizer and Repligen To Collaborate on SMA Therapy Development

Repligen’s spinal muscular atrophy program, including development of the experimental drug RG3039, now has the support of a major pharmaceutical company

Pfizer will now work with Repligen to advance potential treatments for SMA.
Article Highlights:
  • Pfizer will collaborate with Repligen to advance development of therapies for spinal muscular atrophy; the Repligen-Pfizer SMA program includes the MDA-supported drug RG3039, as well as other compounds and technologies.
  • RG3039, which is designed to raise levels of the deficient SMN protein in patients' cells, is now being tested in healthy volunteers.
by Margaret Wahl on January 4, 2013 - 8:48am

Update (Feb. 2, 2015): Pfizer announced in January 2015 that it will not continue its collaboration with Religen for development of RG3039. The reasons are not clear, but industry observers suspect that the change of plan is related to an announcement in summer 2014 that the compound failed to increase production of the SMN protein. See Pfizer Bails on a $70M Rare Disease Deal with Repligen.

Update (Sept. 4, 2013): In a Sept. 4, 2013, press release, Repligen Corp. announced that it has received a $1 million payment from Pfizer for the development of compounds — including RG3039 — to treat SMA. The payment represents reaching the “clinical milestone” of having successfully completed the first two dose groups of a phase 1b trial of RG3039 in healthy volunteers.

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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