Efficacy of Asciminib

The U.S. Food and Drug Administration (FDA)grants accelerated approval of Asciminib (Asciminib) for the treatment of chronic phase Philadelphia chromosome-positive chronic myelogenous leukemia< /span> (Ph+CML) patients who have been previously treated with two or more tyrosine kinase inhibitors (TKIs) , and aceminib is approved for the use of T315I mutation Ph+CML chronic phase (CP) patients.

ASCEMBL is a multicenter, randomized, active-controlled, open-label clinical trial evaluating the efficacy of aceminib in patients with Ph+CML CP who have received two or more prior TKIs. A total of 233 patients were randomized (2:1), stratified by major cytogenetic response (MCyR) status, to receive either aximinib 40 mg twice daily or bosutinib 500 mg once daily. Patients continued treatment until unacceptable toxicity or treatment failure. The primary efficacy outcome measure was major molecular response (MMR) at 24 weeks. The incidence of MMR was 25% (95% CI: 19, 33) in patients treated with asiminib and 13% (95% CI: 6.5, 23; p=0.029) (95% CI: 6.5, 23; p=0.029). The median follow-up duration was 20 months, and the median MMR duration has not yet been reached.

CABL001X2101 is a multi-center, open-label clinical trial that is evaluating the efficacy of aceminib in patients with Ph+CML CP with T315I mutation. Efficacy was based on 45 patients with T315I mutations who received aceminib 200 mg twice daily. Patients continued treatment until unacceptable toxicity or treatment failure. The primary efficacy outcome measure was MMR. Forty-two percent (19/45, 95% confidence interval: 28% to 58%) of patients achieved MMR within 24 weeks. 49% (22/45, 95% confidence interval: 34% to 64%) of patients achieved MMR at 96 weeks. The median duration of treatment was 108 weeks.

The most common adverse reactions (≥20%) of aceminib are upper respiratory tract infection, musculoskeletal pain, fatigue, nausea, rash, and diarrhea;The most common laboratory abnormalities are decreased platelet count, increased triglycerides, decreased neutrophil count and hemoglobin, and increased creatine kinase, alanine aminotransferase, lipase, and amylase. For those who have received it twice or moreFor patients with Ph+CML CP treated with TKI, the recommended dose of aceminib is 80 mg once daily or 40 mg twice daily. The recommended dose of aximinib for patients with T315I-mutated Ph+CML CP is 200 mg orally twice daily.