Globenewswire·2025-12-06 13:00Core Insights - Aptose Biosciences Inc. presented clinical data for its lead compound tuspetinib (TUS) in combination with venetoclax (VEN) and azacitidine (AZA) at the 67th American Society of Hematology Annual Meeting, highlighting promising safety and antileukemic activity in treating acute myeloid leukemia (AML) [1][2][3] Group 1: Clinical Data and Efficacy - The TUSCANY trial showed a 100% clinical response rate (CR/CRh) at the higher doses of 80 mg and 120 mg TUS [5][6] - High-quality clinical responses were observed across various genetic subgroups, including FLT3 wildtype and those with adverse mutations such as TP53 and RAS [6][7] - Minimal residual disease (MRD) negativity was achieved in 78% of responding subjects, indicating effective treatment outcomes [6][7] Group 2: Safety and Tolerability - TUS+VEN+AZA therapy demonstrated a favorable safety profile with no dose-limiting toxicities reported across all evaluated TUS dose levels [6][7] - No drug-related deaths or significant adverse events such as differentiation syndrome or QTc prolongation were reported [6][7] - The therapy was well tolerated, with 8 out of 10 evaluable subjects achieving red cell and platelet transfusion independence for over 8 weeks after their best response [6][7] Group 3: Future Directions - The company has commenced treating patients at the highest dose level of 160 mg TUS, with early responses already observed [3][6] - The ongoing TUSCANY Phase 1/2 study aims to further evaluate the efficacy and safety of TUS in combination with standard therapies for newly diagnosed AML patients ineligible for induction chemotherapy [7][8]