RG7800 shows proof of mechanism in Phase I

An abstract released ahead of the American Academy of Neurology meeting said RG7800 from Roche (SIX:ROG; OTCQX:RHHBY) demonstrated proof of mechanism in a Phase I trial by showing a dose-dependent effect on splicing of survival of motor neuron 2 centromeric ( SMN2) in healthy volunteers.

Roche began a Phase Ib/IIa trial of RG7800 in November to treat spinal muscular atrophy (SMA). Roche licensed the compound from PTC Therapeutics Inc. (NASDAQ:PTCT) in 2011. ...