On March 26, 2020 Tolero Pharmaceuticals, Inc., a clinical-stage company focused on developing novel therapeutics for hematological and oncological diseases, reported that it has joined The Leukemia & Lymphoma Society (LLS) in the groundbreaking, collaborative Beat AML Master Clinical Trial for newly diagnosed patients 60 years of age or older with acute myeloid leukemia (AML) (Press release, Tolero Pharmaceuticals, MAR 26, 2020, View Source;lymphoma-societys-groundbreaking-beat-aml-master-clinical-trial-for-patients-with-acute-myeloid-leukemia-301030080.html [SID1234555888]). Tolero’s investigational agent, dubermatinib (TP-0903), an AXL receptor tyrosine kinase (RTK) inhibitor has been selected for a new arm in the trial for patients with TP53 mutations and/or complex karyotype.

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AML is one of the deadliest blood cancers and the second most diagnosed type of leukemia in the U.S.1 Although there have been recent advances in the treatment of AML, for patients with certain types of mutations, prognosis and responsiveness to therapy remains poor.2 The Beat AML Master Clinical Trial aims to leverage the expertise of functional genomic technologies and pharmaceutical collaborators, using a personalized medicine approach to accelerate research findings and ultimately improve outcomes for AML patients. Patients with TP53 mutations have few effective treatment options as their response rate to chemotherapy is poor and long-term survival after stem cell transplant is rare.3 The incidence of TP53 mutations in AML has been reported to be between 5 and 19 percent of patients.4,5,6,7,8

"Tolero Pharmaceuticals is proud to have been selected to join The Leukemia & Lymphoma Society in its efforts to apply a precision medicine approach toward fighting acute myeloid leukemia," said David J. Bearss, Ph.D., Chief Executive Officer of Tolero Pharmaceuticals, Inc. "The Beat AML Master Clinical Trial provides us a unique opportunity to contribute to the advancement of science in AML and evaluate the potential of dubermatinib in a patient group which has a poor prognosis and limited treatment options."

In the Phase 1b/2 study, dubermatinib in combination with decitabine will be evaluated in patients 60 years or older with newly diagnosed AML who have TP53 mutations and/or complex karyotype. The primary objectives of the study are to determine the safety and maximum tolerated dose of dubermatinib in combination with decitabine and evaluate the composite complete response rate. Secondary objectives of the study include overall survival and proportion of patients transitioning to allogeneic stem cell transplantation.

"We believe the Beat AML Master Clinical Trial is changing the treatment paradigm for AML as well as for clinical trials more broadly across cancer types," said Amy Burd, Ph.D., Vice President of Research Strategy, The Leukemia & Lymphoma Society. "Through this trial, we have taken a tailored approach to treatment based on patients’ unique genomic profile, with the ultimate goal of identifying safe and effective targeted therapies for patients who previously had limited options. LLS is pleased that Tolero Pharmaceuticals is joining this unprecedented collaboration of top cancer centers and scientists, several top pharmaceutical companies, the U.S. Food and Drug Administration and a leading-edge genomics company to bring novel targeted therapies to patients faster."

The trial is being conducted at several leading cancer centers across the United States.

About dubermatinib (TP-0903)

Dubermatinib is an investigational oral AXL receptor tyrosine kinase (RTK) inhibitor under evaluation in a Phase 1/2 study in patients with chronic lymphocytic leukemia or small lymphocytic lymphoma (NCT03572634) and a Phase 1a/b study in patients with advanced solid tumors (NCT02729298). Tolero is exploring parallel clinical development paths for dubermatinib in both solid and hematologic malignancies.

About AXL Kinase

AXL belongs to the TAM (Tyro3, AXL and Mer) family of receptor tyrosine kinases and is overexpressed in many human cancers.9 It plays a key role in tumor cell proliferation, survival, metastasis, cellular adhesion and avoidance of the immune response. The overexpression of AXL is associated with a poor patient prognosis and drug resistance.10