Aptose Announces Dosing of First Patient with 120 mg of Tuspetinib in Phase 1/2 Tuscany Trial of Frontline Triple Drug Therapy after Dose Escalation Decision by Safety Review Committee

Core Viewpoint - Aptose Biosciences Inc. is advancing its clinical-stage precision oncology therapy, tuspetinib (TUS), in a Phase 1/2 trial (TUSCANY) for newly diagnosed acute myeloid leukemia (AML) patients, with recent approval to escalate dosing based on positive safety and efficacy data [1][2][3]. Group 1: Clinical Trial Progress - The Cohort Safety Review Committee has approved the escalation of the TUS dose from 80 mg to 120 mg due to favorable safety and efficacy data [1][3]. - The first patient has been dosed at the 120 mg level, and enrollment for this cohort is ongoing [1][7]. - No significant safety concerns or dose-limiting toxicities have been reported in the trial, including no prolonged myelosuppression in subjects [2][7]. Group 2: Efficacy and Safety Data - Data from the initial cohorts (40 mg and 80 mg) show promising clinical safety and antileukemic activity, even in difficult-to-treat AML populations [3][4]. - The TUS+VEN+AZA triplet therapy has achieved complete remissions (CRs) and minimal residual disease (MRD) negativity with favorable safety profiles [7]. Group 3: Future Presentations and Data - Updated data will be presented at the European Hematology Association Congress (EHA 2025) in Milan, Italy, including safety, CRs, MRD, and pharmacokinetic findings [2][4]. - The TUSCANY trial is being conducted at 10 leading U.S. clinical sites, with an anticipated enrollment of 18-24 patients by mid-late 2025 [5].