I think it's possible the DMC can simply recommend 2 x 2mg/kg and for the trial to move directly to stage 2. If this is the case then the stage 1 results will be used to establish 2 x 2mg/kg as the minimum effective dose, so won't be a complete waste. There will be a slight setback since the durability study will be delayed. The second option will be for PAR to go back to the FDA and get the IND amended to include 2 x 2mg/kg and possibly start from stage 1 again. It's obviously complicated since none of the P2 trials for iPPS have been conducted in the USA, but given the presumed lack of AE's in the stage 1 trial then the DMC should be confident enough to allow the 2 x 2mg/kg dose to be used.
All IMO, dyor
PAR Price at posting:
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