Aptose's Frontline Triple Drug Therapy with Tuspetinib Achieves Notable Responses in Newly Diagnosed AML Patients in the Phase 1/2 TUSCANY Trial

Core Insights - The TUS+VEN+AZA triplet therapy has shown promising early results in achieving complete remission in newly diagnosed acute myeloid leukemia (AML) patients, particularly those with TP53 mutations [1][3][4] - The therapy demonstrates a favorable safety profile, with no need for dose adjustments of venetoclax (VEN) and azacitidine (AZA) during treatment [1][3][6] - The TUSCANY trial aims to establish this triplet as a frontline therapy for diverse AML populations, including those traditionally difficult to treat [4][5][8] Company Overview - Aptose Biosciences is a clinical-stage biotechnology company focused on developing precision medicines for oncology, particularly in hematology [8] - The company's lead product, tuspetinib (TUS), is being evaluated as a frontline triplet therapy for newly diagnosed AML patients [8] Trial Details - The TUSCANY Phase 1/2 trial is designed to test various doses of TUS in combination with standard doses of AZA and VEN for AML patients ineligible for induction chemotherapy [5] - The initial cohort received a starting dose of 40 mg of TUS, with plans for dose escalations based on safety reviews [5] - Enrollment is ongoing, with an expected 18-24 patients by mid to late 2025 [5] Early Results - Four newly diagnosed AML patients have been treated with the 40 mg dose of TUS, with three patients achieving complete remissions by the end of Cycle 1 [6] - Notably, a patient with biallelic TP53 mutations achieved complete remission, highlighting the potential of the therapy in challenging cases [6] - Pharmacokinetic analyses indicate that TUS levels remain stable and unaffected by the addition of AZA, suggesting predictability in dosing [6]