Repligen reports positive Phase I spinal muscular atrophy study data

The blinded, ascending, single dose study demonstrated that RG3039 was well tolerated at all doses administered and showed evidence of a dose-related drug response resulting in 90% inhibition of the target enzyme.

The outcomes may help to establish appropriate RG3039 dosing regimens for future studies, including potential efficacy studies in SMA patients, the company said.

Repligen president and chief executive officer Walter Herlihy said: "The safety and PK outcomes from our Phase 1 study of RG3039 are encouraging, and we look forward to initiating the next steps for this drug candidate in alignment with guidance from the U.S. Food and Drug Administration."

RG3039 is the clinical-stage drug candidate targeting the core genetic deficit in SMA in order to treat the biochemical deficits caused by decreased levels of the survival motor neuron (SMN) protein.