. 2021 Mar 18;14:44. doi: 10.1186/s13045-021-01055-9

Table 5.

Efficacy and safety results from the asciminib monotherapy cohorts of the phase I dose-finding study

Cohort	Efficacy	Safety
Cohort without T315I mutations [81]	37% (37/99) and 48% (44/91) of all evaluable patients achieved or maintained MMR by 6 and 12 months, respectively 77% (85/110) and 70% (77/110) of all evaluable patients achieved or maintained MCyR and CCyR, respectively	Study drug–related AEs of any grade were reported in 100% (150/150) of patients, and grade 3/4 AEs were reported in 60% (90/150) of patients The most common AEs of any grade were fatigue (29.3%), headache (28.0%), and increased lipase (26.7%)
Cohort with T315I mutations [81]	21% (4/19) and 24% (4/17) of all evaluable patients achieved MMR by 6 and 12 months, respectively 55% (11/20) and 41% (9/22) of all evaluable patients achieved MCyR and CCyR, respectively
Cohort with baseline BCR-ABL1^IS ≤ 1% [77]	87.5% of patients remained on therapy at data cutoff 75% (18/24) of these patients were in MMR at data cutoff MR⁴ and MR^4.5 were achieved by > 40% of evaluable patients who were not in MR⁴ or MR^4.5 at baseline 12.5% discontinued due to AEs	Grade 3/4 AEs (in > 10% of patients), regardless of study drug, were increased lipase (27.1%) and hypertension (12.5%)

Cohort

Efficacy

Safety

Cohort without T315I mutations [81]

37% (37/99) and 48% (44/91) of all evaluable patients achieved or maintained MMR by 6 and 12 months, respectively

77% (85/110) and 70% (77/110) of all evaluable patients achieved or maintained MCyR and CCyR, respectively

Study drug–related AEs of any grade were reported in 100% (150/150) of patients, and grade 3/4 AEs were reported in 60% (90/150) of patients

The most common AEs of any grade were fatigue (29.3%), headache (28.0%), and increased lipase (26.7%)

Cohort with T315I mutations [81]

21% (4/19) and 24% (4/17) of all evaluable patients achieved MMR by 6 and 12 months, respectively

55% (11/20) and 41% (9/22) of all evaluable patients achieved MCyR and CCyR, respectively

Cohort with baseline BCR-ABL1^IS ≤ 1% [77]

87.5% of patients remained on therapy at data cutoff

75% (18/24) of these patients were in MMR at data cutoff

MR⁴ and MR^4.5 were achieved by > 40% of evaluable patients who were not in MR⁴ or MR^4.5 at baseline

12.5% discontinued due to AEs

Grade 3/4 AEs (in > 10% of patients), regardless of study drug, were increased lipase (27.1%) and hypertension (12.5%)

AE adverse event, CCyR complete cytogenetic response, MCyR major cytogenetic response, MMR major molecular response, MR⁴ 4.0-log molecular response (BCR-ABL1^IS ≤ 0.01%), MR^4.5 4.5-log molecular response (BCR-ABL1^IS ≤ 0.0032%)