Dr. Nguyen on ABL001 in Patients With CML

Marie Huong Nguyen, MD

Marie Huong Nguyen, MD, assistant professor, Hematology and Oncology, University of Michigan Health System, discusses the safety and efficacy of ABL001 as a potential treatment for patients with chronic myeloid leukemia (CML).

ABL001 is a potent, allosteric BCR-ABL inhibitor designed to treat patients with CML and Philadelphia chromosome-positive acute lymphoblastic leukemia. In a phase I study examining ABL001 in patients with CML, three dose-limiting toxicities were reported, due to reports of arthralagia (n = 1), lipase elevation (n = 1) and acute coronary syndrome (n = 1). The majority of adverse events reported were grade 1/2, Nguyen adds. There were 5 discontinuations due to disease progression.

With regard to efficacy, 11 TKI-resistant patients who received ABL001 achieved a > 1-log reduction. Five patients who had hematologic relapse also achieved complete hematologic response within 3 months. Nine out of 11 patients achieved complete cytogenetic response, Nguyen adds.

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