SY-2101 is a novel oral form of arsenic trioxide (ATO) that we are developing for acute promyelocytic leukemia (APL), a subtype of acute myeloid leukemia (AML) that is caused by a fusion of the RARA and PML genes. We are currently evaluating SY-2101 in a dose confirmation study, which will inform Phase 3 development.
An intravenously administered formulation of ATO is part of a combination regimen that cures more than 80% of APL patients, but it is extremely burdensome on patients, requiring up to 140 lengthy infusions over the course of nearly a year of treatment. We believe that an oral form of ATO that offers similar efficacy as IV ATO would dramatically reduce the treatment burden on patients and improve access.
Based on preliminary data available as of July 2022 in our Phase 1 dose confirmation trial, SY-2101 at 15 mg achieved comparable PK (AUC and Cmax) exposures to IV ATO administered at 0.15 mg/kg. Additionally, SY-2101 showed high oral bioavailability of ~80% and this preliminary data continues to support a favorable tolerability and safety profile.
