Aptose Biosciences (APTO) Bloom Burton & Co. Healthcare Investor Conference 2025 summary

Company and Therapy Overview

• Clinical-stage precision oncology company developing Tuspetinib as part of a triple therapy for newly diagnosed AML patients.

• Tuspetinib is a safe, mutation-agnostic frontline therapy designed to address AML's genetic heterogeneity.

• AML is aggressive with poor survival rates, especially in older adults, highlighting the need for improved therapies.

• Current standard of care (VEN-AZA) is insufficient for many patients, particularly those with adverse mutations.

• Tuspetinib is positioned as an ideal third drug due to its safety, convenience, and broad activity across AML genotypes.

Clinical Trial Progress and Results

• TUSCANY trial initiated, testing Tuspetinib with VEN-AZA in newly diagnosed AML patients at leading U.S. sites.

• High response rates, complete remissions, and MRD-negativity achieved at both 40mg and 80mg doses, including in high-risk mutations like TP53, IDH, NPM1, and FLT3.

• No dose-limiting toxicities or significant safety concerns observed in trial cohorts.

• Patients achieving complete remission are also becoming transfusion independent.

• The triplet regimen is mutation agnostic and maintains a strong safety profile, distinguishing it from other third-drug combinations.

Strategic Milestones and Future Plans

• Orphan drug and fast-track status granted; initial trials required in relapsed/refractory AML before frontline use.

• Multiple clinical milestones achieved, including positive safety reviews and dose escalation approval in the TUSCANY trial.

• Dose selection for pivotal studies expected this year, with pivotal trials and pharma partnerships planned for 2025–2026.

• Data updates and presentations scheduled at major hematology conferences (EHA, ASH) throughout 2025 and 2026.

• National Cancer Institute selected Tuspetinib for the MATCH/MyeloMATCH program, expanding its clinical evaluation.