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Drug intelligence / Profile preview
OT-A201 + pomalidomide
- Development stage
- Phase 1
- 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
CK1α (Casein kinase I alpha)CRBN (Cereblon)PTGS2 (Prostaglandin-Endoperoxide Synthase 2)GSPT1 (G1 to S phase transition 1)IKZF1 (Ikaros family zinc finger protein 1)IKZF3 (Zinc finger protein Aiolos)TNFA (Tumor necrosis factor alpha (soluble))
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