Drug intelligence / Profile preview

TRO40303

Development stage
Phase 2
Lead developer
Roche
Modality
Small Molecules
Administration
Intravenous
01

Overview

TRO40303 is a mitochondrial-targeted small molecule drug developed for its cytoprotective and cardioprotective properties. It acts by binding to the translocator protein (TSPO) at the cholesterol site on the mitochondrial outer membrane, which is associated with regulation of the mitochondrial permeability transition pore (mPTP). By inhibiting mPTP opening triggered by oxidative stress, TRO40303 reduces reactive oxygen species production, calcium overload, and release of apoptotic factors in cells subjected to ischemia/reperfusion injury. Preclinical studies demonstrated that TRO40303 significantly reduced infarct size in animal models of myocardial infarction when administered prior to reperfusion. Additionally, it has shown hepatoprotective effects in models of acute hepatitis and liver failure by preventing mitochondrial permeabilization and cell death. The drug was initially identified for neuroprotective potential but has been primarily investigated for cardiac ischemia-reperfusion injury and acute myocardial infarction[1][3][6][8].

Other names
3,5-seco-4-nor-cholestan-5-one oxime-3-ol
02

Targets

TSPO (Translocator Protein (18 kDa))

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