Drug intelligence / Profile preview

BKX-001

Development stage
Preclinical
Lead developer
Ipsen
Modality
Small Molecules
Administration
Oral
01

Overview

**BKX-001** is an investigational oral small-molecule drug developed by BAKX Therapeutics as a direct activator of BAX (BCL-2-associated X protein), a key pro-apoptotic effector in the mitochondrial apoptosis pathway. It promotes programmed cell death selectively in tumor cells by triggering BAX activation downstream of anti-apoptotic BCL-2 family proteins like BCL-2, BCL-XL, and MCL-1, potentially overcoming resistance seen with upstream inhibitors. BKX-001 showed preclinical activity in leukemia cell lines, lymphomas, and select solid tumors, sparing healthy cells, and was partnered with Ipsen in a $14.5M deal for further development in hematological malignancies and solid tumors.[2][7][9]

02

Targets

BAX (Apoptosis regulator BAX)

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