Core Insights - Aptose Biosciences Inc. is advancing its clinical-stage precision oncology drug, tuspetinib (TUS), in a Phase 1/2 trial named TUSCANY for newly diagnosed acute myeloid leukemia (AML) patients [1][9] Group 1: Trial Overview - The TUSCANY trial began in December 2024 and has shown positive data from three cohorts with varying doses of TUS (40 mg, 80 mg, and 120 mg) combined with venetoclax (VEN) and azacitidine (AZA) [1][2] - The trial aims to provide a safe, mutation-agnostic frontline therapy for AML patients who cannot receive induction chemotherapy [2][9] Group 2: Efficacy Data - The combination therapy of TUS+VEN+AZA has demonstrated a complete response (CR) or complete response with partial hematological recovery (CRh) rate of 90% (9 out of 10 patients) compared to 65% for VEN+AZA [4] - In specific subpopulations, such as those with NPM1, FLT3-ITD, and TP53 mutations, the CR/CRh rates reached 100% [4][5] - The minimal residual disease (MRD) negativity rate among all subjects treated with TUS+VEN+AZA was 70% (7 out of 10), significantly higher than the 23.4% rate for VEN+AZA [4][5] Group 3: Safety Profile - The TUSCANY trial reported no significant safety concerns or dose-limiting toxicities (DLTs) at the 120 mg TUS dose level, with no treatment-related deaths or prolonged myelosuppression observed [2][5] - Nine out of ten patients remain on study, indicating a favorable safety and tolerability profile [2][6] Group 4: Future Directions - Following a safety review, the trial is advancing to a 160 mg TUS dose level, with expectations of continued positive outcomes [2][3] - The company anticipates that the ongoing data collection will further support the efficacy and safety of the TUS+VEN+AZA combination therapy [6][10]