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Aptose Biosciences Begins Expansion Trial of Tuspetinib in TP53, FLT3-Mutant AML

NEW YORK – Aptose Biosciences on Monday began treating biomarker-defined acute myeloid leukemia patients with myeloid kinase inhibitor tuspetinib in the Phase I/II APTIVATE expansion trial.

The San Diego-based firm began treating patients with the 120 mg monotherapy dose of tuspetinib in the APTIVATE expansion study. The expansion trial will enrich for patients with relapsed or refractory AML harboring TP53 or FLT3 mutations. The study will also include a combination arm exploring tuspetinib with AbbVie/Genentech's Venclexta (venetoclax) in this population.

"Tuspetinib has demonstrated noteworthy safety and mutation-agnostic potency across a spectrum of AML patients with a diversity of adverse mutations, further distinguishing it from competing compounds and targeting a much larger AML population," Aptose CEO William Rice said in a statement. "This breadth of activity along with its significant safety profile has allowed us to define a precise clinical and commercial plan for tuspetinib in multiple lines of therapy, including its use in doublet and triplet combinations, as well as maintenance therapy."

The APTIVATE trial includes several mutationally defined cohorts of AML patients. In data presented in December, responses were seen in AML patients with mutations in NPM1, MLL, TP53, NRAS, KRAS, DNMT3A, RUNX1, wild-type FLT3, ITD- or TKD-mutated FLT3, and other genes.

Tuspetinib was designed to simultaneously target SYK, JAK1/2, FLT3, and other kinases operative in AML, according to Aptose. In May, Aptose received fast track designation from the US Food and Drug Administration for tuspetinib in patients with relapsed or refractory AML with a FLT3 mutation.