Abstract
Ewing Sarcoma (ES) is a rare pediatric cancer with poor prognosis and high unmet need. Chimeric oncoproteins encoded by fusions of the EWS gene and one of five different ETS transcription factors are dominant drivers of the disease. TK216 was designed to bind ETS proteins directly, disrupt protein interactions, inhibit transcription factor function and cause apoptotic cell death. Notably, TK216 plus vincristine (VCR) was shown to exert synergistic activity (Zollner 2017). Here, we report the results of the phase I trial of TK216 in ES.
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