Blue­print met all its end­points in bid for ex­pand­ed Ay­vak­it la­bel — but stock trends low­er any­way

Blue­print Med­i­cines an­nounced this morn­ing that the sec­ond part of its study on Ay­vak­it in non-ad­vanced sys­temic mas­to­cy­to­sis (SM) — a rare dis­ease in which a type of white blood cells known as mast cells builds up — met all end­points, but the bio­phar­ma left key ques­tions unan­swered.

In 212 pa­tients, with 141 in the treat­ment arm and 71 in the con­trol arm, pa­tients who got Ay­vak­it saw an av­er­age 15.6-point de­crease in their symp­tom scores com­pared to a 9.2-point de­crease in the place­bo arm at 24 weeks. In an ex­ten­sion study, those on Ay­vak­it saw their symp­tom scores drop by 20.2 points by week 48.

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