Drug intelligence / Profile preview

OT-A201 + pomalidomide

Development stage
Unknown
Lead developer
Onward Therapeutics
Modality
Small Molecules
Administration
Intravenous (OT-A201), Oral (pomalidomide)
01

Overview

OT-A201 + pomalidomide is an experimental drug combination investigated for use in patients with relapsed/refractory hematological malignancies or advanced/metastatic solid tumors. OT-A201 is a novel investigational agent—details about its precise mechanism or structure have not been disclosed publicly as of September 2025. Pomalidomide is a third-generation immunomodulatory agent (IMiD), structurally related to thalidomide, and is approved as monotherapy or in combination for multiple myeloma and Kaposi’s sarcoma. Pomalidomide acts primarily via binding to cereblon (a substrate receptor of the E3 ubiquitin ligase complex), thereby promoting degradation of specific target proteins, leading to anti-proliferative, pro-apoptotic, and immunomodulatory effects. The combination is being evaluated for antineoplastic activity, leveraging OT-A201’s unknown/novel modality alongside the established IMiD immunomodulatory and antineoplastic activities of pomalidomide[3][4].

Other names
CC-4047CC4047CC 4047
02

Targets

GSPT1 (G1 to S phase transition 1)PTGS2 (Prostaglandin-Endoperoxide Synthase 2)CK1α (Casein kinase I alpha)CRBN (Cereblon)IKZF3 (Zinc finger protein Aiolos)IKZF1 (Ikaros family zinc finger protein 1)

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