Proven efficacy of AYVAKYT demonstrated with 60% ORR across evaluable Advanced SM patients1
Proven efficacy of AYVAKYT
ORR of AYVAKYT was evaluated across all Advanced SM patients, (n=47) evaluable according to the mIWG-MRT-ECNM criteria, who received at least one prior systemic therapy and a starting dose of 200 mg1–4*
*A clinical improvement is defined as having a response duration of ≥12 weeks and fulfilment of one or more of the non-haematological and/or haematological response criteria.3,4
CR=complete response; CRh=complete remission with partial haematological recovery; mlWG-MRT-ECNM=modified International Working Group-Myeloproliferative Neoplasms Research and Treatment and European Competence Network on Mastocytosis; PR=partial response
Efficacy of AYVAKYT has been proven across all subtypes of Advanced SM (ASM, SM-AHN, MCL)1
Efficacy was demonstrated across all Advanced SM patients (ASM, SM-AHN, MCL), n=47, who received at least one prior systemic therapy and a starting dose of 200 mg:1
Of particular note is the strong efficacy in SM-AHN, as this is the most frequently occurring subtype in Advanced SM seen in clinical practice5
AYVAKYT demonstrated rapid responses that deepen over time1
In patients who received at least one prior systemic therapy:1
**Estimated from Kaplan-Meier analysis. DOR (months), median (95% confidence interval).
Since SM-AHN represents over 70% of the population in this analysis, the high response rates suggest a clinically important effect of AYVAKYT across all subtypes, regardless of prior systemic therapy6
AYVAKYT demonstrated reductions in objective mast cell measures1,2
Among Advanced SM patients treated with AYVAKYT at a starting dose of 200 mg once daily following prior systemic therapy:1
88.1%
≥50% reduction in serum tryptase level [n=67]†
83.1%
≥50% reduction in bone marrow mast cells [n=65]
60.0%
≥35% reduction in spleen volume from baseline [n=65]
68.7%
≥50% reduction in KIT D816V MAF in blood [n=67]
†To achieve a CR and CRh, serum tryptase had to be <20 ng/mL if the baseline value was ≥40 ng/mL.3
CR=complete response; CRh=complete remission with partial haematological recovery; DOR=duration of response; MAF=mutant allele function
Duration of response observed with AYVAKYT2
Interim results from the ongoing phase 2 PATHFINDER trial for Advanced SM patients who received at least one prior systemic therapy and a starting dose of 200 mg:2
Figures adapted from Gotlib J et al. 2021
‡Median PFS in the interim analysis efficacy population and median OS in the safety population (secondary endpoints) were not yet statistically assessable at the time of data cut-off.
AYVAKYT reduction in patient-reported symptoms following initiation of treatment, and improved quality of life across 5 domains2
Patient-reported symptoms, measured by AdvSM-SAF total symptom score (TSS)2§
Figures adapted from Gotlib J et al. 2021
- Following AYVAKYT treatment initiation, mean symptom scores were lower than the baseline at cycles 3 and 11 for all SM symptoms, including fatigue, abdominal pain, spots, itching, flushing, nausea, diarrhoea and vomiting
- Patient-reported symptoms improved rapidly following treatment initiation, dropping by 7.1 points from baseline at cycle 3 (n=51) and by 9.8 points from baseline at cycle 11 (n=22; P <0.001)
§TSS is the sum of eight possible common symptoms (each scored 0 to 10, where 0 represents no symptoms and 10 is the worst imaginable).
AdvSM-SAF=Advanced SM-Symptom Assessment Form; BL=baseline; CI=confidence interval; C3=cycle 3; C11=cycle 11
Quality of Life (QoL), assessed by EORTC-QLQ-CV302‖
Figures adapted from Gotlib J et al. 2021
- Following AYVAKYT treatment initiation, patients reported noteworthy improvements in physical (strenuous activity), role (work or household jobs), emotional (irritability, feeling tense and depression), cognitive (memory and concentration) and social (family life and social activities) functioning domains
The QoL data demonstrates the meaningful benefits that AYVAKYT can offer to patients, beyond clinical efficacy alone
‖EORTC-QLQ-CV30 ranges from 0 to 10, where 0 represents the lowest QoL and 100 represents the highest QoL. This is the same for all domains (physical, role, emotional, cognitive, social).
BL=baseline; CI=confidence interval; C3=cycle 3; C11=cycle 11; EORTC-QLQ-CV30=European Organization for Research and Treatment of Cancer Core QoL Questionnaire C30
