RefinedOncology
Targeting significant unmet needs in hematological malignancies
Chronic myelomonocytic leukemia (CMML) is a rare blood cancer classified as an MDS/MPN overlap neoplasm as it exhibits features of both ineffective and abnormal blood cell production and overproduction of monocytic cells. Critically, CMML carries a significant risk of progression, with approximately a quarter of patients progressing to aggressive Acute Myeloid Leukemia (AML). There are limited treatment options for patients with CMML, with standard of care consisting of hypomethylating agents.


A clinically validate cell surface target to eradicate malignant monocytes and resistant cell populations
Leukocyte Immunoglobulin-like Receptor B4 (LILRB4), is a cell-surface protein highly expressed on monocytes and macrophages, which functions as an immune checkpoint that suppresses immune responses, primarily by inhibiting T-cell activation. In hematologic malignancies, its expression is significantly increased on neoplastic monocytic cells in both CMML and monocytic AML, where it is associated with a poor prognosis by both shielding the cancer from the immune system and promoting tumor survival and infiltration.
RO-202, a phase 2 ready mAb targeting LILRB4
Our asset, RO-202, is a first-in-class humanized Fc-active IgG1 anti-LILRB4 mAb designed to deplete malignant monocytic cells in Chronic Myelomonocytic Leukemia (CMML), MDS, and monocytic Acute Myeloid Leukemia (AML).
RO-202 has completed a Phase 1 study and has shown promising efficacy in CMML patients. We have Orphan Drug Designation and Fast Track Designation from the FDA.




