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Aptose Reports Early Data Demonstrating Tuspetinib Improves Standard of Care Treatment for Newly Diagnosed AML

Aptose Biosciences Inc. (APTOF) | Aug. 18, 2025

By Laura Young

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Aptose provided an update on the Phase 1/2 TUSCANY trial for newly diagnosed AML.

The addition of Tuspetinib to Venetoclax and Azacitidine shows improved response rates and MRD-negativity rates.

Positive results were observed across diverse AML populations, including difficult-to-treat mutations.

Excellent safety profile was maintained at the 120 mg dose level.

The data supports the use of Tuspetinib with standard care treatment in AML patients.

Improved Response Rates

Tuspetinib added to Venetoclax and Azacitidine resulted in 100% CR/CRh at 80 mg and 120 mg dose levels.

Enhanced MRD-Negativity

78% MRD-negativity rates among responders were achieved with Tuspetinib+Venetoclax+Azacitidine.

Broad Spectrum Activity

Positive outcomes in FLT3 wildtype AML and TP53, RAS, and FLT3-ITD mutated AML cases.

Safety and Tolerability

No significant safety concerns or dose-limiting toxicities observed with the combination therapy.

Future Treatment Potential

Expectation of extended patient survival with continued long-term treatment and no loss of MRD-negativity.

  • The data highlights the success of the TUS+VEN+AZA triplet therapy in improving response rates and MRD-negativity.
  • The addition of Tuspetinib demonstrated excellent safety and well-tolerated outcomes across diverse AML populations.
  • The potential to address specific mutations like TP53, RAS, and FLT3-ITD could lead to longer overall survival.
  • The promising outcomes support the progression of TUS+VEN+AZA as a mutation agnostic frontline therapy for AML patients.

The early data from the TUSCANY trial reinforces the efficacy and safety of Tuspetinib in combination with standard care treatment for newly diagnosed AML patients. The positive results across different mutation profiles indicate a promising future for this triple drug therapy.