Aptose Reports Early Data Demonstrating Tuspetinib Improves Standard of Care Treatment Across Diverse Populations of Newly Diagnosed AML in Phase 1/2 TUSCANY Trial

• Addition of Tuspetinib (TUS) to Venetoclax (VEN) and Azacitidine (AZA) is being developed as safe and mutation agnostic frontline therapy for AML
• Addition of TUS to VEN+AZA improves response rates; 100% CR/CRh at 80 mg and 120 mg
• Addition of TUS to VEN+AZA improves MRD-negativity rates; 78% among responders
• 100% CR/CRh in FLT3 wildtype AML, representing 70% of AML population
• 100% CR/CRh and MRD-negativity rates in TP53, RAS and FLT3-ITD mutated AML
• Broad spectrum activity and excellent safety profile continue at 120 mg dose to date

Excerpt from the Press Release:

SAN DIEGO and TORONTO, Aug. 18, 2025 (GLOBE NEWSWIRE) — Aptose Biosciences Inc. (“Aptose” or the “Company”) (OTC: APTOF, TSX: APS), a clinical-stage precision oncology company developing the tuspetinib (TUS)-based triple drug frontline therapy to treat patients with newly diagnosed AML, today provided a data update from the Phase 1/2 TUSCANY trial in newly diagnosed AML. The trial was initiated in December 2024, and the growing body of positive data includes the recently completed third cohort of 120 mg TUS in the TUS+VEN+AZA triplet therapy. Data to date from ten (10) patients across all three cohorts, 40 mg, 80 mg or 120 mg TUS dose in TUS+VEN+AZA, support the use of TUS with standard of care treatment across all AML populations, including those carrying mutations that are the most difficult to treat and those with mutated and unmutated (wildtype) FLT3 genes.

The TUS+VEN+AZA triplet is being developed as a safe and well-tolerated mutation agnostic frontline therapy to treat large, mutationally diverse populations of newly diagnosed AML patients who are ineligible to receive induction chemotherapy. At the 120 mg TUS dose level in combination with VEN+AZA, as with the prior reported 40 mg and 80 mg TUS dose cohorts, no significant safety concerns or dose limiting toxicities (DLTs) have been observed in the TUSCANY trial, including no prolonged myelosuppression in Cycle 1 of subjects in remission, no reports of drug-related QTc prolongation or differentiation syndrome (DS), and no treatment-related deaths.

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