Table 1.

Response rates to avapritinib in the EXPLORER and PATHFINDER studies

CharacteristicPhase 1 EXPLORERPhase 2 Interim PATHFINDER
Response evaluable, n 53 32 
AdvSM subtype, n   
 ASM 
 SM-AHN 37 26 
 MCL 13 
ORR by modified IWG criteria, % (CR + CRh + PR + CI) 75 75 
Best response, %   
 CR 15 
 CRh 21 19 
 PR 34 31 
 CI 25 
 SD 23 13 
 PD 
 NE 
ORR by subtype, %   
 ASM 100 100 
 SM-AHN 76 81 
 MCL 69 25 
ORR by any prior therapy, %   
 Yes 69 74 
 No 86 78 
ORR by midostaurin history, %   
 Prior midostaurin 59 82 
 Midostaurin naive 83 67 
ORR by S/A/R comutation status, %   
 Yes 74 71 
 No 77 80 
CharacteristicPhase 1 EXPLORERPhase 2 Interim PATHFINDER
Response evaluable, n 53 32 
AdvSM subtype, n   
 ASM 
 SM-AHN 37 26 
 MCL 13 
ORR by modified IWG criteria, % (CR + CRh + PR + CI) 75 75 
Best response, %   
 CR 15 
 CRh 21 19 
 PR 34 31 
 CI 25 
 SD 23 13 
 PD 
 NE 
ORR by subtype, %   
 ASM 100 100 
 SM-AHN 76 81 
 MCL 69 25 
ORR by any prior therapy, %   
 Yes 69 74 
 No 86 78 
ORR by midostaurin history, %   
 Prior midostaurin 59 82 
 Midostaurin naive 83 67 
ORR by S/A/R comutation status, %   
 Yes 74 71 
 No 77 80 

CI, clinical improvement; CRh, complete remission with partial hematologic recovery; NE, not evaluable; PD, progressive disease; S/A/R, SRSF2, ASXL1, RUNX1; SD, stable disease.

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