Home > Drug List > Asciminib > Therapeutic efficacy of Asciminib

Therapeutic efficacy of Asciminib

1.Ph+ CML-CP, Previously Treated With Two or More TKIs 

The efficacy of Asciminib in the treatment of patients with Ph+ CML in chronic phase (Ph+ CML-CP), previously treated with two or more tyrosine kinase inhibitors was evaluated in the multi-center, randomized, active-controlled, and open-label study Asciminib (NCT 03106779). 

In this study, a total of 233 patients were randomized in a 2:1 ratio and stratified according to major cytogenetic response (MCyR) status to receive either Asciminib 40 mg twice daily (N = 157) or bosutinib 500 mg once daily (N = 76). Patients continued treatment until unacceptable toxicity or treatment failure occurred. 

Patients were 52% female and 48% male with a median age of 52 years (range, 19 to 83 years). Of the 233 patients, 19% were 65 years or older, while 2.6% were 75 years or older. Patients were White (75%), Asian (14%), and Black or African American (4.3%). Of the 233 patients, 81% and 18% had Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1, respectively. Patients who had previously received 2, 3, 4, or 5 or more prior lines of TKIs were 48%, 31%, 15%, and 6%, respectively. The median duration of treatment was 67 weeks (range, 0.1 to 162 weeks) for patients receiving Asciminib and 30 weeks (range, 1 to 149 weeks) for patients receiving bosutinib. 

The main efficacy outcomes from Asciminib are summarized in Table 8. 

The MMR rate at 48 weeks was 29% (95% CI: 22, 37) in patients receiving Asciminib and 13% (95% CI: 6.5, 23) in patients receiving bosutinib. With a median duration of follow-up of 20 months (range: 1 day to 36 months), the median duration of response had not yet been reached for patients with MMR at any time. 

2.Ph+ CML-CP with the T315I mutation 

The efficacy of Asciminib in the treatment of patients with Ph+ CML-CP with the T315I mutation was evaluated in a multi-center open-label study CABL001X2101 (NCT02081378). Testing for T315I mutation utilized a qualitative p210 BCR-ABL mutation test using Sanger Sequencing. 

Efficacy was based on 45 patients with Ph+ CML-CP with the T315I mutation who received Asciminib at a dose of 200mg twice daily. Patients continued treatment until unacceptable toxicity or treatment failure occurred. 

Of the 45 patients, 80% were male and 20% female; 31% were 65 years or older, while 9% were 75 years or older with a median age of 54 years (range, 26 to 86 years). The patients were White (47%), Asian (27%), and Black or African American (2.2%), and 24% were unreported or unknown. Seventy-three percent and 27% of patients had ECOG performance status 0 and 1, respectively. Patients who had previously received 1, 2, 3, 4, and 5 or more TKIs were 18%, 31%, 36%, 13%, and 2.2%, respectively. 

MMR was achieved by 24 weeks in 42% (19/45, 95% CI: 28% to 58%) of the 45 patients treated with Asciminib. MMR was achieved by 96 weeks in 49% (22/45, 95% CI: 34% to 64%) of the 45 patients treated with Asciminib. The median duration of treatment was 108 weeks (range, 2 to 215 weeks). 

from FDA,2021.10