Asciminib monotherapy in patients with chronic myeloid leukemia in chronic phase without BCR::ABL1T315I treated with at least 2 prior TKIs: Phase 1 final results

Asciminib is the first approved BCR::ABL1 inhibitor that Specifically Targets the ABL Myristoyl Pocket (STAMP). The present final analysis of the phase 1, open-label, nonrandomized trial (NCT02081378) assessed the long-term safety, tolerability, and antileukemic activity of asciminib in 115 patients with chronic myeloid leukemia in chronic phase without the BCR::ABL1<sup>T315I</sup> mutation who received asciminib 10-200 mg twice daily (BID) or 80-200 mg once daily (cutoff: March 14, 2023). Median exposure duration was 5.9 (range, 0-8.4) years; 60.9% of patients continued receiving asciminib through post-trial access. Grade ≥3 adverse events (AEs) occurred in 88 patients (76.5%). AEs led to treatment discontinuation, dose adjustment/interruption, or additional therapy in 15 (13.0%), 74 (64.3%), and 106 (92.2%) patients, respectively. Most first-ever AEs, particularly hematologic AEs, presented within the first year and no new safety signals emerged. Of 56 patients who achieved major molecular response, 50 maintained the response by cutoff; the Kaplan-Meier-estimated probability of maintaining this response for ≥432 weeks ( ≈ 8.3 years) was 88% (95% confidence interval, 78.2-97.0%). The recommended dose for expansion was determined at 40 mg BID. With up to 8.4 years of treatment, asciminib continued to demonstrate long-term safety and efficacy in this population.

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Publication Details

Journal
Leukemia
Published
2025-04-09
DOI
https://doi.org/10.1038/s41375-025-02578-7
Citations
9
Primary Topic
Chronic Myeloid Leukemia Treatments
Type
article
Field-Weighted Citation Impact
15.93

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article

Asciminib monotherapy in patients with chronic myeloid leukemia in chronic phase without BCR::ABL1T315I treated with at least 2 prior TKIs: Phase 1 final results

Moshe Talpaz, Jörge E. Cortes, Michael J. Mauro, S. S. Kapoor et al.
9 citations
Leukemia
Chronic Myeloid Leukemia Treatments
15.93
article

Asciminib monotherapy in patients with chronic myeloid leukemia in chronic phase without BCR::ABL1T315I treated with at least 2 prior TKIs: Phase 1 final results

Moshe Talpaz, Jörge E. Cortes, Michael J. Mauro, S. S. Kapoor, Yeow Tee Goh, Meng Cao, Koji Sasaki, Andreas Hochhaus, Philipp le Coutre, Nathalie Pognan, Valle Gomez Garcia de Soria, Timothy P. Hughes, Delphine Réa, Oliver G. Ottmann, Matthias Hoch, Daniel J. DeAngelo, Gessamí Sánchez-Ollé, Hironobu Minami, Massimo Breccia, Michael C. Heinrich, Dong‐Wook Kim, Fabian Lang
article en
9 citations

Abstract

Asciminib is the first approved BCR::ABL1 inhibitor that Specifically Targets the ABL Myristoyl Pocket (STAMP). The present final analysis of the phase 1, open-label, nonrandomized trial (NCT02081378) assessed the long-term safety, tolerability, and antileukemic activity of asciminib in 115 patients with chronic myeloid leukemia in chronic phase without the BCR::ABL1<sup>T315I</sup> mutation who received asciminib 10-200 mg twice daily (BID) or 80-200 mg once daily (cutoff: March 14, 2023). Median exposure duration was 5.9 (range, 0-8.4) years; 60.9% of patients continued receiving asciminib through post-trial access. Grade ≥3 adverse events (AEs) occurred in 88 patients (76.5%). AEs led to treatment discontinuation, dose adjustment/interruption, or additional therapy in 15 (13.0%), 74 (64.3%), and 106 (92.2%) patients, respectively. Most first-ever AEs, particularly hematologic AEs, presented within the first year and no new safety signals emerged. Of 56 patients who achieved major molecular response, 50 maintained the response by cutoff; the Kaplan-Meier-estimated probability of maintaining this response for ≥432 weeks ( ≈ 8.3 years) was 88% (95% confidence interval, 78.2-97.0%). The recommended dose for expansion was determined at 40 mg BID. With up to 8.4 years of treatment, asciminib continued to demonstrate long-term safety and efficacy in this population.

LeukemiaVol. 39(5)
Goethe University Frankfurt (DE), Novartis (Switzerland) (CH), Memorial Sloan Kettering Cancer Center (US), The University of Texas MD Anderson Cancer Center (US), Oregon Health & Science University (US), Singapore General Hospital (SG), University of Michigan (US), Policlinico Umberto I (IT), Portland VA Medical Center (US), Augusta University Health (US), Kobe University Hospital (JP), Novartis (France) (FR), The Catholic University of Korea Uijeongbu St. Mary's Hospital (KR), Dana-Farber Cancer Institute (US), Novartis (United States) (US), Hospital Universitario de La Princesa (ES), University Hospital Frankfurt (DE), Jena University Hospital (DE), South Australian Health and Medical Research Institute (AU), Hôpital Saint-Louis (FR), VA Portland Health Care System (US), The University of Adelaide (AU), Charité - Universitätsmedizin Berlin (DE), Cardiff University (GB), Sapienza University of Rome (IT)
Novartis, Novartis Pharmaceuticals Corporation
Good health and well-being
Openalex Percentile: Top 1%
Chronic Myeloid Leukemia Treatments
15.93
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