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 advancing SY-2101 into a dose confirmation study, which will be followed by a planned Phase 3 study in newly diagnosed APL patients.
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.
In a Phase 1 clinical trial, SY-2101 demonstrated oral bioavailability, achieved exposure levels in the range of the approved IV dose, and was generally well-tolerated.
