KEY TAKEAWAYS
- The phase I/II NEXI-001 trial aimed to evaluate the safety and efficacy of NEXI-001 in pts with relapsed AML after allogeneic HCT.
- Pts with relapsed AML after allogeneic HCT were divided into 3 cohorts and received different doses of NEXI-001.
- The study found NEXI-001 has shown promise in treating relapsed AML. More patients are being enrolled in the trial to assess its efficacy.
NEXI-001 is a donor-derived T-cell therapy that targets Acute Myeloid Leukemia (AML) blasts and leukemic stem cells. Researchers aimed to evaluate the safety and efficacy of NEXI-001 in patients (pts) with relapsed AML after allogeneic hematopoietic cell transplantation (HCT).
The study included AML patients who relapsed after allogeneic HCT with limited treatment options and a poor prognosis (2-year survival rate < 10%). About 11 pts have completed the 4-week dose-limiting toxicity (DLT) period in the dose-escalation stage. T-cell infusions were administered at varying doses across three cohorts; Cohort 1 (50M to 100M T cells), Cohort 2 (200M T cells), and Cohort 3 (200M T cells on Days 1, 8, and 15).
The study observed NEXI-001 antigen-specific T-cells persist and multiply in the blood and bone marrow for at least three months without severe side effects. Adverse events (AE) included mild cytokine release syndrome and gastrointestinal issues. Pts in each cohort showed positive clinical activity, such as improved blood cell counts, reduced need for transfusions, and decreased leukemia cells. One patient achieved complete remission after receiving NEXI-001 cells and remained in remission after six months. Another patient with relapsed disease experienced a significant decrease in pleural effusion size and remained asymptomatic with no detectable leukemia cells in blood or bone marrow after NEXI-001 treatment. A PET scan showed no signs of disease in the chest.
The study found NEXI-001 has shown promise in treating relapsed AML. More patients are being enrolled in the trial to assess its efficacy.
Source: https://ascopubs.org/doi/abs/10.1200/JCO.2023.41.16_suppl.7043
Clinical Trial: https://classic.clinicaltrials.gov/ct2/show/NCT04284228
Monzr M. Al Malki, Sumithira Vasu, Dipenkumar Modi, Suzanne D Afonso-Smith, Sojung Kim, Emily Lu, Guido Marcucci, Mathias Oelke, Robert D. Knight, and Juan Carlos Varela. DOI: 10.1200/JCO.2023.41.16_suppl.7043 Journal of Clinical Oncology 41, no. 16_suppl (June 01, 2023) 7043-7043.
