dexpramipexole

Dexpramipexole is a first-in-class oral small molecule under investigation primarily for eosinophilic asthma and other eosinophil-associated diseases. It is the (D)-enantiomer of pramipexole but lacks dopamine agonist activity. Dexpramipexole lowers blood and tissue eosinophils by an as-yet not fully elucidated mechanism that appears to involve modulation of mitochondrial function—specifically binding to subunits of the F1/FO ATP synthase complex—which may indirectly inhibit the mitochondrial permeability transition pore (mPTP). The drug was originally developed for amyotrophic lateral sclerosis (ALS), but development in ALS was discontinued after negative Phase III results. Its ability to lower eosinophils was discovered during these trials, leading to its current development focus on moderate-to-severe eosinophilic asthma and related conditions such as hypereosinophilic syndrome and chronic rhinosinusitis with nasal polyps. Dexpramipexole has demonstrated significant reductions in blood and tissue eosinophil counts in multiple clinical studies[2][5][8][10].

Other names

(R)-pramipexoleR-(+)-pramipexoledexpramipexol(6R)-N6-propyl-4,5,6,7-tetrahydro-1,3-benzothiazole-2,6-diamine