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

Core Insights - Aptose Biosciences is presenting promising data from the TUSCANY trial, which evaluates the safety and efficacy of the tuspetinib-based triplet therapy (TUS+VEN+AZA) for newly diagnosed AML patients ineligible for induction chemotherapy [1][2][3] Group 1: Clinical Trial Data - The TUSCANY trial has shown that the addition of tuspetinib to the standard treatment of venetoclax and azacitidine has resulted in complete remission (CR) or complete remission with incomplete hematologic recovery (CRh) in 100% of patients treated at higher doses of 80 mg and 120 mg [5][6] - Overall, 90% of patients in the trial have achieved CR/CRh responses, with 88% of FLT3 wildtype AML patients responding positively [6][8] - The therapy has demonstrated activity across diverse genetic subtypes, including those with unmutated FLT3, FLT3-ITD, NPM1c, biallelic TP53, RAS, and myelodysplasia-related mutations [3][14] Group 2: Safety Profile - The TUS+VEN+AZA combination has been well tolerated, with no significant safety concerns or dose-limiting toxicities reported, including no prolonged myelosuppression, differentiation syndrome, QTc prolongation, or treatment-related deaths [2][6] - Dosing has commenced at the 160 mg level, indicating ongoing escalation in the trial [5][14] Group 3: Trial Design and Objectives - The TUSCANY trial is a Phase 1/2 study being conducted at 10 leading U.S. clinical sites, aiming to establish a safe and effective frontline therapy for a broad range of newly diagnosed AML patients [9][11] - The trial is designed to test various doses and schedules of tuspetinib in combination with standard dosing of azacitidine and venetoclax, with an anticipated enrollment of 18-24 patients by the end of 2025 [9][10]