AYVAKYT showed statistically significant improvements across primary and secondary endpoints in ISM1
Primary endpoint
AYVAKYT + BSC demonstrated a statistically significant reduction in TSS vs placebo + BSC (P=0.003) at Week 241
MEAN CHANGE IN ISM-SAF TSS AT WEEK 24 AND 481
*For AYVAKYT-treated patients who rolled over into the open-label extension of PIONEER (Part 3).
**Placebo patients were allowed to move into an open-label ongoing AYVAKYT crossover arm, with TSS data available from Week 24 onwards.
AYVAKYT demonstrated a deepening reduction in mean TSS sustained over time1
Patients in the AYVAKYT + BSC arm continued to report improvements in TSS over time for at least 48 weeks1
Key secondary endpoints
AYVAKYT + BSC demonstrated statistically significant clinical improvements vs placebo + BSC, using ≥30% and ≥50% TSS thresholds1
PROPORTION OF PATIENTS ACHIEVING ≥30% REDUCTION IN ISM-SAF TSS THROUGH 24 WEEKS1,2
PROPORTION OF PATIENTS ACHIEVING ≥50% REDUCTION IN ISM-SAF TSS THROUGH 24 WEEKS1,2
Figures adapted from Gotlib et al. 2023.
A 30% reduction in TSS was selected as a valid and conservative threshold for clinically important improvement in symptoms3
Key secondary endpoints
AYVAKYT + BSC demonstrated significant improvements in objective measures of mast cell burden at Week 241
†P<0.0001.
TT analysis: For patients with high-dose steroid use within 7 days before Week 24, or greater than 14 consecutive days at any point from baseline to Week 24, the Week 24 score was set to missing.
