SY-2101 is a novel oral form of arsenic trioxide (ATO) in development 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 conducting a Phase 1 clinical trial of SY-2101 in newly diagnosed APL patients that is designed to confirm the dose for a future registration-enabling study.
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. The regimen requires up to 140 lengthy infusions over the course of a year. We believe that an oral form of ATO that offers similar efficacy to IV ATO would dramatically reduce the treatment burden on patients and improve access.
SY-2101 achieved comparable PK (AUC and Cmax) exposures to IV ATO, based on preliminary data available as of July 2022 in our Phase 1 dose confirmation trial. Additionally, SY-2101 showed high oral bioavailability of ~80% and this preliminary data continues to support a favorable tolerability and safety profile.
