Drug Type: Small Molecule
Mechanism Type: Mitochondrial protectant
Mechanism: Gnx4728 is an inhibitor of the mitochondrial Permeability Transition Pore (mPTP). Increases in mitochondrial permeability are associated with mitochondrial swelling and rupturing, calcium release and release of apoptotic factors. Congenia is collaborating with academic researchers on testing their proprietary inhibitor Gnx4728 in mouse models of ALS.
U.S. Status for ALS: Preclinical
 GNX-4728, a novel small molecule drug inhibitor of mitochondrial permeability transition, is therapeutic in a mouse model of amyotrophic lateral sclerosis. Martin, LJ et al. Front Cell Neurosci. 2014 Dec 19; 8:433.
Last updated March 9th, 2016