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 is being developed as a frontline treatment for diverse populations of newly diagnosed AML patients who are ineligible for induction chemotherapy [1][4][5] Group 1: Clinical Trial Results - The TUSCANY trial has reported complete remissions in Cycle 1 for patients with TP53 mutations and FLT3 wildtype AML [1][3][6] - Early data from the trial indicates favorable safety and antileukemic activity, with no dose-limiting toxicities observed in patients receiving the lowest dose of TUS (40 mg) [2][6] - The triplet therapy has demonstrated the ability to treat patients with traditionally difficult-to-treat mutations, improving patient outcomes from the outset [3][4] Group 2: Trial Design and Administration - The TUSCANY Phase 1/2 study is designed to test various doses and schedules of TUS in combination with standard dosing of azacitidine (AZA) and venetoclax (VEN) [5] - TUS will be administered as a once-daily oral agent in 28-day cycles, starting at a dose of 40 mg, with planned dose escalations after safety reviews [5] - The trial aims to enroll 18-24 patients by mid-late 2025, with data to be released as it becomes available [5] Group 3: 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 clinical-stage oral kinase inhibitor, tuspetinib (TUS), has shown activity as both a monotherapy and in combination therapy for relapsed or refractory AML [8]