Phase Ib Clinical Trial Of RG3039 Initiated By Repligen

27 September 2012


Repligen Corporation has initiated a Phase Ib clinical trial of RG3039, a drug with potential to treat spinal muscular atrophy (SMA). RG3039 is a small molecule drug with US Orphan Drug and Fast Track status that was recently shown to be well tolerated by healthy volunteers in Phase Ia clinical testing of its safety andpharmacokinetic profile.

RG3039 was licensed by Repligen in 2009 from Cure SMA, and is able to boost SMN protein levels by increasing its production from the SMN 2 gene. When administered to SMA model mice, RG3039 improves lifespan and various disease symptoms including motor neuron survival.

In this new Phase Ib trial, which is an extension of the phase Ia trial, researchers at the Jasper Clinic in Kalamazoo, Michigan aim to study further the safety and pharmacokinetics of the drug. RG3039 will be tested with the help of 32 healthy adult volunteers over a 28 day period.

It is hoped that the information gained from these Phase I trials will aid in the future design of clinical trials of RG3039 in SMA patients.

Further Information

Article on the Phase Ib trial on the fSMA website

Article about the Phase Ia trial on the Cure SMA website

Article on the initial licensing of RG3039 by Repligen

Cure SMA website

Repligen Corporation website