Asciminib monotherapy in patients with BCR::ABL1 T315I-mutated chronic-phase chronic myeloid leukemia: phase 1 trial final results

Abstract In chronic myeloid leukemia in chronic phase (CML-CP), BCR::ABL1 T315I commonly leads to treatment resistance, worse patient outcomes, and limited subsequent treatment options. By targeting the ABL1 myristoyl pocket, asciminib maintains activity against BCR::ABL1 T315I . We report final long-term safety, tolerability, and efficacy results with asciminib in 48 patients with T315I-mutated CML-CP who received asciminib 200 mg twice daily in the phase 1, nonrandomized trial (NCT02081378). After a median exposure of 3.5 years, 52.1% of patients continued to receive asciminib via posttrial access. Of 45 evaluable patients, 24 (53.3%) achieved major molecular response (MMR); 20 of 24 maintained or deepened their response by the cutoff. The Kaplan-Meier estimated proportion of patients maintaining their first MMR for at least 144 weeks (2.8 years) was 86% (95% CI: 71.9–100.0%). The safety profile showed no new or worsening safety signals. With 1.4 years’ additional exposure since the previous analysis, the incidence of grade ≥3 adverse events (AEs) (60.4%) did not increase. Four patients (8.3%) discontinued due to AEs. The exposure-adjusted incidence rate of first all-grade AOEs was 4.4 cases per 100 patient-years. With up to approximately 6 years of exposure, this final analysis confirms asciminib as a treatment option for patients with T315I-mutated CML-CP.

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Journal
Leukemia
Published
2026-07-16
DOI
https://doi.org/10.1038/s41375-026-02972-9
Primary Topic
Chronic Myeloid Leukemia Treatments
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article
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article

Asciminib monotherapy in patients with BCR::ABL1 T315I-mutated chronic-phase chronic myeloid leukemia: phase 1 trial final results

K Sasaki, Michael W. Deininger, Moshe Talpaz, Jörge E. Cortes et al.
Leukemia
Chronic Myeloid Leukemia Treatments
article

Asciminib monotherapy in patients with BCR::ABL1 T315I-mutated chronic-phase chronic myeloid leukemia: phase 1 trial final results

K Sasaki, Michael W. Deininger, Moshe Talpaz, Jörge E. Cortes, Michael J. Mauro, Jeroen J. W. M. Janssen, S. S. Kapoor, Yeow Tee Goh, Nithya Agrawal, D Réa, Andreas Hochhaus, Andrea Damon, Valle Gomez Garcia de Soria, Timothy P. Hughes, Matthias Hoch, Daniel J. DeAngelo, Gessamí Sánchez-Ollé, Massimo Breccia, Michael C. Heinrich, Dong‐Wook Kim, Fabian Lang, Sara Quenet, Mahon Fx, Philipp le Coutre
article en

Abstract

Abstract In chronic myeloid leukemia in chronic phase (CML-CP), BCR::ABL1 T315I commonly leads to treatment resistance, worse patient outcomes, and limited subsequent treatment options. By targeting the ABL1 myristoyl pocket, asciminib maintains activity against BCR::ABL1 T315I . We report final long-term safety, tolerability, and efficacy results with asciminib in 48 patients with T315I-mutated CML-CP who received asciminib 200 mg twice daily in the phase 1, nonrandomized trial (NCT02081378). After a median exposure of 3.5 years, 52.1% of patients continued to receive asciminib via posttrial access. Of 45 evaluable patients, 24 (53.3%) achieved major molecular response (MMR); 20 of 24 maintained or deepened their response by the cutoff. The Kaplan-Meier estimated proportion of patients maintaining their first MMR for at least 144 weeks (2.8 years) was 86% (95% CI: 71.9–100.0%). The safety profile showed no new or worsening safety signals. With 1.4 years’ additional exposure since the previous analysis, the incidence of grade ≥3 adverse events (AEs) (60.4%) did not increase. Four patients (8.3%) discontinued due to AEs. The exposure-adjusted incidence rate of first all-grade AOEs was 4.4 cases per 100 patient-years. With up to approximately 6 years of exposure, this final analysis confirms asciminib as a treatment option for patients with T315I-mutated CML-CP.

Leukemia
Goethe University Frankfurt (DE), Novartis (Switzerland) (CH), Memorial Sloan Kettering Cancer Center (US), The University of Texas MD Anderson Cancer Center (US), Radboud University Nijmegen (NL), Singapore General Hospital (SG), University of Michigan (US), Policlinico Umberto I (IT), Radboud University Medical Center (NL), Portland VA Medical Center (US), University of Alabama at Birmingham (US), Augusta University Health (US), The Catholic University of Korea Uijeongbu St. Mary's Hospital (KR), Versiti Blood Center of Wisconsin (US), Dana-Farber Cancer Institute (US), Novartis (United States) (US), Hospital Universitario de La Princesa (ES), University Hospital Frankfurt (DE), Institut Bergonié (FR), Jena University Hospital (DE), South Australian Health and Medical Research Institute (AU), Hôpital Saint-Louis (FR), VA Portland Health Care System (US), OHSU Knight Cancer Institute, Institute of Science Tokyo (JP), Eulji University Uijeongbu Eulji Medical Center (KR), The University of Adelaide (AU), Charité - Universitätsmedizin Berlin (DE)
Amgen, Bristol-Myers Squibb, Pfizer, AstraZeneca, Sanofi, Alexion Pharmaceuticals, Celgene, Novartis Pharmaceuticals Corporation, Incyte, Astellas Pharma, Servier, Sun Pharma, EUSA Pharma, Sanofi Genzyme, Daiichi Sankyo Europe, Cilag
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Openalex Percentile: Top 9%
Chronic Myeloid Leukemia Treatments
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