Perlmutter Cancer Center

The purpose of this study is to evaluate the efficacy and safety of asciminib as 2L therapy for CML-CP patients who had experienced treatment failure or intolerance to 1 prior ATP-binding site TKI. Following the safety and efficacy preliminary data from the ALLG CML13 ASCEND-CML study of asciminib 40 mg BID as 1L treatment, this study will additionally explore the efficacy and safety of asciminib dose escalation in 1L CML-CP patients. Asciminib has demonstrated activity and tolerability in dosages of up to 200 mg BID. (Hughes et al 2019) and so it is of high scientific interest to evaluate whether CML patients whose BCR-ABL1IS levels are above 1% and 0.1% at 6 months and at 12 months, respectively, could potentially benefit from an asciminib dose escalation approach. Based on extensive pharmacokinetic (PK) modeling, a dose escalation from 80 mg QD asciminib to 200 mg QD and/or 200 mg BID asciminib was chosen. Together the data will likely provide valuable guidance for clinical practice, as well as for the use of asciminib in CML-CP patients in the 1L and 2L settings.