Summary

Treatment of MDS patients with parenteral hypomethylating agents (HMA) such as decitabine (DAC) requires frequent visits to the physician. An orally administered HMA would provide significant patient convenience, potentially enhance adherence to treatment, and permit the exploration of extended treatment schedules with lower doses of DAC. Neither DAC nor azacitidine is readily bioavailable in oral form due to rapid clearance by cytidine deaminase (CDA) present in the gut and liver. E7727, a novel CDAi, is orally bioavailable with a large safety margin and reproducible effectiveness in preclinical models. We report here the first in human Phase 1 results of a PK-guided dose escalation trial of ASTX727 (the orally administered combination of DAC
and E7727).

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2015 ASH: Results of FIH P1 PK Guided Dose-Escalation Study of ASTX727 in Subjects with MDS