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<p>In chronic myeloid leukemia in chronic phase (CML-CP), <i>BCR::ABL1</i><sup>T315I</sup> commonly leads to treatment resistance, worse patient outcomes, and limited subsequent treatment options. By targeting the ABL1 myristoyl pocket, asciminib maintains activity against <i>BCR::ABL1</i><sup>T315I</sup>. 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.</p>

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

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年7月16日

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  • Jorge E. Cortes,
  • Delphine Rea,
  • Michael J. Mauro,
  • Andreas Hochhaus,
  • Dong-Wook Kim,
  • Koji Sasaki,
  • Fabian Lang,
  • Michael C. Heinrich,
  • Massimo Breccia,
  • Michael Deininger,
  • Yeow-Tee Goh,
  • Jeroen J. W. M. Janssen,
  • Moshe Talpaz,
  • Valle Gómez García de Soria,
  • Philipp le Coutre,
  • Francois-Xavier Mahon,
  • Daniel J. DeAngelo,
  • Andrea Damon,
  • Shruti Kapoor,
  • Gessami Sanchez-Olle,
  • Matthias Hoch,
  • Nithya Agrawal,
  • Sara Quenet,
  • Timothy P. Hughes

摘要

In chronic myeloid leukemia in chronic phase (CML-CP), BCR::ABL1T315I commonly leads to treatment resistance, worse patient outcomes, and limited subsequent treatment options. By targeting the ABL1 myristoyl pocket, asciminib maintains activity against BCR::ABL1T315I. 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.