Aptose’s Tuspetinib Exceeds Expectations When Combined with Standard of Care Treatment Across Diverse Populations of Newly Diagnosed AML

Core Insights - The TUSCANY trial is evaluating the safety and efficacy of a triplet therapy combining tuspetinib (TUS) with venetoclax (VEN) and azacitidine (AZA) for newly diagnosed acute myeloid leukemia (AML) patients who are ineligible for induction chemotherapy [1][8] - Initial results from 10 patients show promising clinical safety and antileukemic activity, with a high rate of complete remissions (CR) and minimal safety concerns [2][3] Summary by Sections Trial Overview - The TUSCANY Phase 1/2 trial is designed to test various doses of TUS in combination with standard dosing of AZA and VEN for AML patients [9] - The trial is being conducted at 10 leading U.S. clinical sites, with an expected enrollment of 18-24 patients by the end of 2025 [9] Safety and Efficacy - No significant safety concerns or dose-limiting toxicities (DLTs) have been observed in the TUSCANY trial, including no prolonged myelosuppression or treatment-related deaths [2][6] - The addition of TUS to VEN+AZA achieved CR/CRh responses in 100% of patients treated at higher dose levels of 80 mg and 120 mg, exceeding the expected 66% response rate from VEN+AZA alone [5][6] Patient Responses - Out of 10 patients, 9 achieved complete remissions, with 7 demonstrating minimal residual disease (MRD) negativity [3][14] - The therapy has shown effectiveness across diverse genetic subtypes, including those with unmutated FLT3, FLT3-ITD, and TP53 mutations [3][14] Current Developments - Dosing has progressed to the 160 mg TUS level, with ongoing assessments of safety and efficacy [2][5][14] - The triplet therapy is being developed as a mutation-agnostic frontline treatment for AML, targeting a broad range of patient populations [2][8]