FDA Lifts Partial Clinical Hold for Phase 2 Study of MT-401 in Post-Transplant AML

The FDA has lifted the partial clinical hold that was placed on a phase 2 study of MT-401 as treatment of patients with post-transplant acute myeloid leukemia (AML), announced Marker Therapeutics, Inc., developer of the drug, in a press release.

“We are pleased to move forward with our phase 2 AML trial of MT-401, which we believe may provide a safe and effective treatment option for patients with post-transplant AML over the standard of care," said Mythili Koneru, MD, PhD, chief medical officer, Marker Therapeutics, in a statement. "During the partial clinical hold, we continued to open clinical centers to enroll patients in the first half of the safety lead-in of our phase 2 trial. With the FDA's decision, we will now be able to seamlessly enroll patients in the second half of the safety lead-in, as well as the remainder of the trial. We look forward to working with our clinical sites to continue enrolling patients.”

The multicenter study is exploring the safety and efficacy of Marker’s lead multi-tumor-associated antigen (MultiTAA)-specific T-cell product candidate MT-401 as treatment of patients with AML in both the adjuvant and active disease setting following an allogeneic stem cell transplant. The primary objective is to explore the relapse-free survival in the adjuvant group and determine the complete response (CR) rate and durable CR in patients with active disease.

A total of 120 patients were randomized 1:1 to either MT-401, an allogeneic T-cell therapy, at 90 days post-transplant or standard of care observation for the adjuvant group in the study, and about 40 patients with active disease will enroll in the single-arm group of the study who will receive MT-401. The study also includes a safety lead-in portion, which is expected to enroll 6 patients.

The study also aims to explore the overall survival and graft-versus-host disease relapse-free survival in the adjuvant group, as well as overall response rate, duration of response, progression-free survival, and overall survival in the active disease group.

To be included in the study, patients must have had their first hematopoietic stem cell transplant in ≤ CR2, and MRD negative prior to transplant. Patients must also have a Karnofsky/Lansky score of ≥ 60, a life expectancy of ≥ 12 weeks, and adequate blood, liver, and renal function. Patients can enroll in the experimental conditioning regimens if no maintenance therapy post-transplant is planned.

Patients will be excluded from the study should they have clinically-significant or severely symptomatic intercurrent infection, are pregnant/lactating, or have evidence of acute or chronic GVHD ≥ 2 within 1 week prior to receiving the study drug.

MT-401 received an Orphan Drug designation from the FDA in April 2020 for the treatment of patients with AML following allogenic stem cell transplant.

_Reference

_{Marker Therapeutics announces FDA lifted partial clinical hold on phase 2 AML clinical trial. News Release. Marker Therapeutics, Inc. January 5, 2021. Accessed January 6, 2021. https://bit.ly/3pSgaMe}