Drug intelligence / Profile preview

BST-236 + venetoclax

Development stage
Preclinical
Lead developer
BioSight
Modality
Metabolically Activated Prodrugs → Prodrugs/Conditional Activator Small Molecules → Small Molecules
Administration
Intravenous, Oral
01

Overview

BST-236 + venetoclax is a combination regimen of **BST-236 (INN aspacytarabine)** and **venetoclax**. BST-236 is a novel prodrug of cytarabine, designed to deliver high doses of cytarabine specifically to target cells while reducing systemic toxicity, particularly in elderly or unfit patients with acute myeloid leukemia (AML). Mechanistically, BST-236 is an asparagine-conjugated cytarabine, which protects the active cytarabine from rapid deactivation and enables enhanced intracellular release, leading to cell death via apoptosis[1][5]. Venetoclax is a selective, orally bioavailable BCL-2 inhibitor that promotes apoptosis by inhibiting the anti-apoptotic protein BCL-2, commonly over-expressed in leukemic cells. This combination is under investigation as a potential regimen for patients with AML who may be refractory or unfit for intensive therapy, building on the distinct mechanisms of action of each component.

02

Targets

BCL-2 (BCL-2 family)DNA

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