Aptose Biosciences (APTO) Corporate presentation summary

Disease background and unmet need

• Acute myeloid leukemia (AML) is a highly aggressive and deadly cancer with current frontline therapy (VEN + AZA) offering limited survival and frequent drug resistance.

• There is a critical need for improved first-line therapies that achieve deeper, more durable responses and extend survival in newly diagnosed AML patients.

Therapeutic innovation and clinical rationale

• Adding a third agent, tuspetinib (TUS), to VEN + AZA aims to boost efficacy, broaden activity, and minimize resistance, especially in FLT3 wildtype and high-risk genetic subgroups.

• TUS has a favorable safety profile, broad activity, and is effective in patients who failed prior targeted therapies.

• Unlike gilteritinib, which is limited to FLT3-mutated AML and adds toxicity, TUS is active in both FLT3-mutated and wildtype AML with cleaner safety.

Clinical trial progress and results

• The Phase 1/2 TUSCANY trial evaluates TUS + VEN + AZA as frontline therapy in newly diagnosed AML, with dose escalation from 40mg to 80mg TUS.

• Early data show complete remissions, including MRD-negative responses, in both FLT3 wildtype and TP53-mutated/complex karyotype AML.

• No prolonged myelosuppression, dose-limiting toxicities, or need for dose reductions in standard-of-care drugs observed at 40mg.

• Enrollment at 80mg is ongoing, with further data releases planned at major hematology conferences in 2025.