Bemcentinib receives FDA approval for Fast Track Designation for the treatment for relapsed or refractory AML

On the 22^nd of October 2019, Bemcentinib (BGB324), an oral selective inhibitor of Axl kinase (AXL),  received fast track designation from the U.S. Food & Drug Administration (FDA) for the treatment of elderly patients with relapsed/refractory acute myeloid leukemia (AML).¹ 

Clinical trials

The FDA designation was based on data from a phase Ib/II study where safety, efficacy and signs of anti-leukemic activity were evaluated following bemcentinib plus cytarabine or decitabine in patients with AML who were unfit for intensive chemotherapy. The phase Ib portion of the study involved a dose-escalation phase in patients with AML given bemcentinib as a single agent (NCT02488408).^2,3

AML global portal created a brief summary of the preliminary phase II data from this trial presented at the 2018 European Hematology Association (EHA) Annual Congress. The final results of this study were presented at the EHA 2019 congress and published in the Journal of Clinical Oncology.

Study design⁴

The phase I trial aimed to find the maximum tolerated dose (MTD) or recommended dose for the phase II portion. Oral administration of bemcentinib alone in a 3+3 dose escalation was given over a 21-day period.

In the phase II open-label trial patients were given a daily dose of 200 mg bemcentinib with low-dose cytarabine in 21-day cycles.

Results^2,3

Sixteen patients with AML were enrolled in the combination part of the phase II study.  Of these patients, 14 were evaluable for response in the preliminary analysis and this data was presented at EHA 2019.

• 44% had achieved rapid CR with incomplete hematologic recovery (Cri)
• 43% of patients did respond to treatment
• 29% of patients had complete responses (CRs)
• 14% of patients had partial responses
• 83% of responses occurred among elderly patients with AML (>75 years)
• Treatment-related adverse events (TRAEs) was seen in 15% of patients. The most common TRAEs were anemia, neutropenia, and diarrhea. Nonetheless, the treatment was considered tolerable

Bemcentinib is now eligible for accelerated approval and priority review, this approval could be considered an important validation of bemcentinib use in an area of significant unmet medical need.