Drug intelligence / Profile preview

ocifisertib

Development stage
Phase 2
Lead developer
Treadwell Therapeutics
Modality
Small Molecules
Administration
Oral
01

Overview

Ocifisertib (CFI-400945) is an **orally administered, first-in-class small molecule inhibitor** of **polo-like kinase 4 (PLK4)**, a serine/threonine kinase that regulates centriole duplication and mitotic progression. Developed initially by the **University Health Network** and advanced by **Treadwell Therapeutics**, it exhibits antineoplastic activity by selectively inhibiting PLK4, disrupting cancer cell proliferation, particularly in hematological malignancies. It has received **FDA Orphan Drug Designation** for **acute myeloid leukemia (AML)** and is under evaluation in **Phase 1b/2 trials** for relapsed/refractory AML, MDS, and CMML, with **Phase II** studies in breast and prostate cancers.[1][3][7][11]

Other names
CFI-400945 fumarateCFI400945 fumarateCFI 400945 fumarate
02

Targets

PLK4 (Polo-like kinase 4)

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