Roche, PTC provide first look at pivotal SMA data in adults

Roche and PTC reported the first pivotal data for a spinal muscular atrophy therapy in adults with established disease on Thursday when they unveiled the detailed data supporting their bid for risdiplam to treat SMA patients of a broad age range.

PTC Therapeutics Inc. (NASDAQ:PTCT) closed up $1.56 to $54.94 on Thursday despite falling as much as 12% in early trading as investors digested data that included a miss on a secondary endpoint in the second part of the Phase II/III SUNFISH trial that evaluated Hammersmith Functional Motor Scale-Expanded (HFMSE) scores -- the endpoint on which Spinraza nusinersen was approved for SMA. ...