TuHURA Biosciences has recently filed an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) to evaluate TBS-2025, a novel VISTA-inhibiting antibody, for the treatment of relapsed or refractory (r/r) Acute Myeloid Leukemia (AML) with mutNPM1 mutations.

The IND submission has been made to the FDA’s Division of Hematologic Malignancies 1 (DHM1). TBS-2025 will be studied in combination with a menin inhibitor in patients who are menin inhibitor naïve. Subject to FDA clearance, the company plans to initiate a Phase II study in early Q2 2026, with preliminary Stage 1 results expected in Q3 2026.

The Phase II trial will use a Simon two-stage design to assess the therapy’s activity and safety profile in patients with mutNPM1 r/r AML. According to the company, leukemogenic mutations such as mutNPM1 are associated with increased expression of VISTA on leukemic cells, which may contribute to reduced response rates and poorer overall survival.

Dr. James Bianco, President and CEO, TuHURA Biosciences, said that although the introduction of menin inhibitors has provided the first approved treatment option for patients with mutNPM1 r/r AML, complete remission (CR/CRh) rates remain below 25 percent and are often short-lived. He noted that combining TBS-2025 with a menin inhibitor could potentially enhance response rates and durability, addressing a significant unmet medical need.

If clinical results are positive, the company intends to seek FDA guidance regarding the potential development of TBS-2025 under the accelerated approval pathway.

TuHURA Biosciences is developing novel immuno-oncology therapies aimed at overcoming resistance to cancer immunotherapy and advancing treatment options for patients with difficult-to-treat malignancies.