Our Vision for Tamibarotene is to be the Foundation of Care for RARA-positive Patients
Tamibarotene (formerly 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 currently investigating tamibarotene in our SELECT-MDS-1 (Phase 3) trial in RARA-positive patients with newly diagnosed higher-risk myelodysplastic syndrome (HR-MDS) and plan to initiate our SELECT-AML-1 (randomized Phase 2) trial in RARA-positive patients with newly diagnosed unfit acute myeloid leukemia (AML).
Data from an earlier, now fully enrolled Phase 2 study showed that tamibarotene 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 tamibarotene 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 tamibarotene in combination with azacitidine into the ongoing SELECT-MDS-1 in RARA-positive newly diagnosed patients with higher-risk MDS, a hematologic malignancy that is closely related to AML, and the planned randomized SELECT-AML-1 trial in combination with venetoclax and azacitidine in RARA-positive newly diagnosed unfit AML patients.