A phase 1/1b study of the all-oral regimen CC-486 and venetoclax in acute myeloid leukemia.
Blood AdvSep 30, 2026
Maria L Amaya, Diana Abbott, Christine McMahon et al.
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In 30 seconds
This phase 1/1b study evaluated the all-oral regimen of CC-486 and venetoclax in patients with relapsed, refractory, and newly diagnosed acute myeloid leukemia (AML). The recommended dose of CC-486 was determined to be 300 mg daily on days 1 to 14. The study reported a 97% incidence of grade ≥3 neutropenia.
Key findings
- The recommended dose of CC-486 was established at 300 mg daily on days 1 to 14.
- Grade ≥3 neutropenia occurred in 97% of patients, while grade ≥3 thrombocytopenia occurred in 66%.
- Responses were observed in all phases of the study, with 6 patients bridged to hematopoietic stem cell transplant.
Why it matters
The findings suggest that the oral combination of CC-486 and venetoclax may provide a viable treatment option for patients with AML, particularly those who are unfit for intensive chemotherapy. Understanding the safety and efficacy of this regimen is crucial for improving patient outcomes.