Aptose Begins Dosing AML Patients with Tuspetinib Triplet in TUSCANY Trial

Aptose Biosciences Inc., a company specializing in precision oncology at the clinical stage, has made a significant stride in treating acute myeloid leukemia (AML). The company recently commenced dosing for the initial group of patients under the TUSCANY Phase 1/2 clinical trial. This trial examines the effectiveness of a combination therapy involving tuspetinib (TUS), venetoclax (VEN), and azacitidine (AZA) administered as a frontline treatment for newly diagnosed AML patients.

The TUSCANY trial aims to assess the potential of the TUS+VEN+AZA triplet therapy to provide a more efficacious initial treatment for AML patients across a wide spectrum of disease profiles, aiming for durability and minimized side effects. Previous trials, particularly the APTIVATE studies, have already indicated that tuspetinib, both as a standalone treatment and in conjunction with venetoclax, is safe and effective across various AML patient groups. These groups include those who have previously undergone treatment with VEN and FLT3 inhibitors and patients with highly aggressive mutations such as TP53 and RAS.

Tuspetinib is an oral medication taken once daily, offering the convenience and potential to address a broad range of mutations within the AML population. This capability allows it to treat not only specific subpopulations but a more extensive set of patients without the need for mutation-specific therapies. Dr. Rafael Bejar, the Chief Medical Officer at Aptose, expressed enthusiasm about the TUSCANY trial's progress, noting that the triplet therapy holds promise for enhancing response rates and prolonging survival in newly diagnosed AML patients. Importantly, it aims to reduce the adverse effects associated with existing treatments, thereby making the therapy accessible to more individuals, including those with challenging disease characteristics.

The TUSCANY Phase 1/2 study is meticulously designed to evaluate different dosages and schedules of tuspetinib alongside standard dosing of azacitidine and venetoclax. The patient group involves those not eligible for induction chemotherapy. The trial protocol includes administering tuspetinib in 28-day cycles, starting at a daily dose of 40 mg. Subsequent dose increases will follow, contingent upon safety reviews at each level. The trial is being conducted across numerous sites in the United States, with a planned enrollment of 18-24 patients expected by mid to late 2025.

Aptose Biosciences is dedicated to developing precision medicines that address unmet medical needs, with a primary focus on hematological conditions. The company's leading clinical-stage drug, tuspetinib, has shown promising results both as a single agent and in combination therapies for patients with relapsed or refractory AML. Aptose is now directing its efforts toward enhancing initial treatment options for newly diagnosed AML patients through the development of the TUS+VEN+AZA triplet therapy.

By focusing on a therapy that is mutation agnostic, the company is working to ensure a broader application of its treatment, potentially transforming the approach to AML treatment and offering hope to a larger group of patients. This initiative underscores Aptose's commitment to advancing patient care and expanding the therapeutic landscape for AML.