Our Vision for SY-1425 is to be the Foundation of Care for RARA-positive Patients
SY-1425 is an oral first-in-class selective retinoic acid receptor alpha (RARα) agonist that we are developing for genomically defined subsets of patients whose disease is characterized by the overexpression of the RARA gene. We are advancing SY-1425 into two planned clinical trials in RARA-positive patients: a Phase 3 trial in newly diagnosed higher-risk myelodysplastic syndrome (HR-MDS) and a randomized Phase 2 trial in newly diagnosed unfit acute myeloid leukemia (AML).
Data from an earlier, now fully enrolled Phase 2 study showed that SY-1425 in combination with azacitidine had high response rates, rapid onset of responses, and clinically meaningful durability in RARA-positive newly diagnosed AML patients who are not suitable candidates for standard chemotherapy. The data also showed that SY-1425 in combination with azacitidine was generally well-tolerated with no increase in toxicities beyond what has been previously seen with either agent alone. Additionally, data from a translational analysis of these trial patients also suggest that RARA-positive patients may be less likely to respond to the current standard of care.
Taken together, these data supported our decision to advance SY-1425 in combination with azacitidine into a Phase 3 trial in RARA-positive newly diagnosed patients with higher-risk MDS, a hematologic malignancy that is closely related to AML, and into a randomized Phase 2 trial in combination with venetoclax and azacitidine in RARA-positive newly diagnosed unfit AML patients.