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Tolero Pharmaceuticals Drug Added to Beat AML Umbrella Trial

This article has been updated with additional information on the future study plans for dubermatinib.

NEW YORK – Tolero Pharmaceuticals' investigational agent dubermatinib (TP-0903) will be used in a new arm in the Beat AML Master Clinical Trial for acute myeloid leukemia patients with TP53 mutations or complex karyotype.

The clinical-stage drug company announced on Thursday that it joined The Leukemia & Lymphoma Society's Beat AML umbrella trial. The study is employing a rapid precision medicine approach to match older, previously untreated patients with acute myeloid leukemia to receive first-line treatments based on their molecular profile. 

TP53 mutations occur in 5 percent to 19 percent of AML patients. This subgroup tends to respond poorly to standard treatment options like chemotherapy and not survive long after stem cell transplants. Tolero is also investigating the AXL inhibitor dubermatinib as a pan-tumor agent in a Phase I/II study of small lymphocytic lymphoma and chronic lymphocytic leukemia and a Phase Ia/b study in patients with advanced solid tumors.

In the Phase Ib/II study arm that is part of Beat AML, dubermatinib will be administered in combination with decitabine to patients 60 years or older with newly diagnosed AML harboring TP53 mutations and/or complex karyotype.

The primary objectives of the study are to evaluate the safety and maximum tolerated dose of the drug combination and gauge the complete response rate. Secondary objectives include overall survival and the proportion of patients who can be transitioned to allogeneic stem cell transplantation.