PTC Therapeutics Phase 2 Data from Spinal Muscular Atrophy Program to be Presented at 20th International Annual Congress of WMS

PTC Therapeutics, Inc.

today announced that clinical data from the company's joint development program with Roche and the SMA Foundation in spinal muscular atrophy (SMA) were presented at the 20th International Congress of the World Muscle Society (WMS) in Brighton, U.K. Results from the first cohort of patients enrolled in the Phase 2 'MOONFISH' trial evaluating oral RG7800, a small molecule modifier of Survival Motor Neuron 2 (SMN2) splicing, were highlighted in a late breaking oral session. The presentation is titled "SMN2 splicing modifier RG7800 increases SMN protein in first study in SMA patients."  "We have now demonstrated, in two independent studies, that treatment with RG7800 shifts SMN2 splicing toward the production of full length SMN mRNA.  Most importantly, in the MOONFISH trial we also observed relevant increases in SMN protein levels in whole blood in patients with SMA," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "This is