FDA lifts partial hold on the phase II trial evaluating MT-401 for posttransplant AML

On January 5, 2021, the U.S. Food and Drug Administration (FDA) lifted the partial clinical hold on a phase II trial (NCT04511130) evaluating MT-401 for patients with acute myeloid leukemia (AML) following allogeneic transplant.

MT-401 is a multi-tumor-associated antigen (MultiTAA)-specific T-cell product able to target a broad range of tumor antigens simultaneously. In April 2020, the product received FDA orphan drug designation in the posttransplant AML setting.

The phase II multicenter trial was designed to determine the safety and efficacy of MT-401 in both the adjuvant and active disease settings. Approximately 120 patients enrolled in the adjuvant group will be randomized 1:1 to receive either MT-401 90 days posttransplant or standard-of-care observation. In the single-arm, active disease group, around 40 patients will receive MT-401. Primary endpoints include relapse-free survival in the adjuvant group, as well as complete remission rates and duration of complete remission in patients with active AML. Further objectives include the following:

• Adjuvant group:
  • overall survival
  • graft-versus-host disease relapse-free survival
• Active AML group:
  • overall response rate
  • duration of response
  • progression-free survival
  • overall survival

This decision will allow the continued enrollment of patients in the second part of the safety lead-in portion and subsequent studies.