Repligen Announces Successful Phase I Clinical Trial Of RG3039 For SMA

15 May 2012

Repligen Corporation has recently announced the positive outcome of initial Phase I clinical testing of the safety and pharmokinetic profile of RG3039, a drug with potential to treat spinal muscular atrophy (SMA). RG3039 is an orally administered, small molecule drug that has been granted US Orphan Drug and Fast Track designations by the US Food and Drug Administration.

Licensed by Repligen in 2009 from Families of SMA, RG3039 has been shown to increase SMN protein expression from the SMN2 gene in cells derived from SMA patients. When given to severe SMA mice, RG3039 improved lifespan, body weight, mobility, and motor neuron survival.

In the blinded, ascending, single dose trial (meaning that the drug was administered without patients knowing whether they were receiving the drug or a placebo, and that a range of single doses were given), RG3039 was tested on healthy volunteers and shown to be well tolerated without causing any serious side effects. RG3039 was able to successfully inhibit its target protein with approximately 90% efficiency.

This Phase I clinical trial has provided useful information on RG3039 dosage for future studies in SMA patients.

Further Information

Repligen press release on the trial 

Article about the trial on the Cure SMA website 

Article on the initial licensing of RG3039 by Repligen 

Cure SMA website 

Repligen Corporation website