Aptose Biosciences (APTO) Study update presentation summary

Precision oncology approach and trial overview

• Developing tuspetinib as a mutation-agnostic therapy for newly diagnosed AML, aiming for broad efficacy and safety.

• TUSCANY trial evaluates tuspetinib in combination with venetoclax and azacitidine (VEN + AZA + TUS) in frontline AML therapy.

• Dose escalation strategy includes 40 mg, 80 mg, 120 mg, and 160 mg tuspetinib, with active enrollment at leading US clinical sites.

Mechanism of action and kinase inhibition

• Tuspetinib directly inhibits key oncogenic kinases: FLT3, SYK, KIT, JAK/STAT, and RAS/MAPK/ERK pathways.

• Demonstrates potent activity across multiple kinase mutations, including FLT3-ITD, D835Y, and KIT D816V.

Clinical efficacy and safety findings

• No dose-limiting toxicities or significant safety concerns observed up to 120 mg; excellent tolerability.

• 100% CR/CRh response rate at 80 mg and 120 mg dose levels; MRD-negativity achieved in 78% of responders.

• Broad efficacy across diverse genetic subgroups, including high-risk TP53-mutated AML.