RBC Capital views FDA flexibility on another HD drug as positive for PTC’s ‘518

Keeping an Outperform rating on PTC Therapeutics (PTCT), RBC Capital says the firm sees net positive albeit indirect/limited read-throughs from Huntington’s Disease competitor uniQure’s (QURE) alignment with the FDA on its statistical analysis plan for approval, using a 3-year change in cUHDRS compared to a propensity-matched natural history data from ENROLLHD registry as the intermediate clinical endpoint in supporting its potential accelerated approval. While the firm does acknowledge that there are some key differences in between PTC’s ‘518 and uniQure’s HD programs, namely the endpoint of interest for AA and PTC’s program in review under CDER, who has been viewed to be more stringent in recent years, RBC believes the overall flexibility of the Agency for another HD program and no material changes to FDA attendance/participation in its type B meetings bode well for ‘518 ahead of its end-of-phase II meeting.