Aptose Launches TUSCANY Phase 1/2 Study for New AML Patients on Tuspetinib Triplet Therapy

SAN DIEGO and TORONTO, Nov. 20, 2024 – Aptose Biosciences Inc. has announced the initiation of the TUSCANY study, a new clinical trial involving the combination of tuspetinib (TUS), azacitidine (AZA), and venetoclax (VEN) for newly diagnosed acute myeloid leukemia (AML) patients. This trial, which is being conducted at multiple clinical sites across the United States, focuses on patients who are not suitable candidates for intensive chemotherapy.

Tuspetinib, an oral medication taken once daily, targets several kinases such as SYK, FLT3, mutated KIT, JAK1/2, and RSK2. This agent is noted for its favorable safety profile and ability to treat broader AML populations compared to other treatments that often target narrower subpopulations and carry significant toxicity concerns. In previous Phase 1/2 APTIVATE trials, tuspetinib demonstrated significant activity as a single agent and in combination with venetoclax among a heavily pre-treated AML population. This included patients with prior therapies involving venetoclax, FLT3 inhibitors, and hematopoietic stem cell transplantation, as well as those with adverse genetic mutations such as TP53 and RAS, regardless of FLT3 gene status.

The launch of the TUSCANY study is a pivotal moment for Aptose. The evolving landscape of AML treatment increasingly favors combination therapies. William G. Rice, Chairman, President, and Chief Executive Officer of Aptose, expressed optimism about tuspetinib’s potential to enhance frontline treatment for a wide range of AML patients without the severe toxicities associated with some existing treatments. He acknowledged the dedication of the investigators and the clinical team in bringing the TUSCANY triplet study to fruition. The study aims to determine whether adding tuspetinib to the standard treatment regimen can improve outcomes for AML patients.

The TUSCANY Phase 1/2 trial will explore different dosages and schedules of tuspetinib in combination with azacitidine and venetoclax. The medication will be given in 28-day cycles, starting with a 40mg dose and escalating based on safety reviews. The trial plans to enroll 18 to 24 patients across 12 sites in the US by mid to late 2025.

Aptose Biosciences is a clinical-stage biotechnology company focused on developing precision medicines for unmet needs in oncology, particularly hematologic malignancies. Their pipeline includes small molecule cancer therapeutics that aim to provide single-agent efficacy and enhance the effectiveness of other anti-cancer therapies without overlapping toxicities. Currently, Aptose has two clinical-stage oral kinase inhibitors under development: tuspetinib and luxeptinib (CG-806). Luxeptinib, another oral kinase inhibitor, is in Phase 1 a/b development for patients with relapsed or refractory hematologic malignancies.

Tuspetinib has shown promise as a monotherapy and in combination with other treatments for relapsed or refractory AML, and is now being developed as part of a triplet therapy for newly diagnosed AML patients. The TUSCANY study represents a significant step forward in this effort, with the potential to bring a new, effective treatment option to a broader range of AML patients. Further updates on the study's execution are anticipated during ASH 2024.