Virtus Therapeutics
Virtus Therapeutics is developing a new class of compounds for the treatment pantothenate kinase-associated neurodegeneration (PKAN), a debilitating neurodegenerative disease of children and young adults. The disease is due to a mutation in the pantothenate kinase 2 (PANK2) gene which encodes a mitochondrial pantothenate kinase responsible for the phosphorylation of vitamin B5, the first step in the biosynthesis of Co-Enzyme A. Our lead compounds, which are called VTACs, activate a second pantothenate kinase PanK3 in order to compensate for the loss of PanK2. Biochemical data demonstrated that VTACs are strong activators of human PanK3. Our lead compounds VTAC-1 and VTAC-2 have long half lives and excellent CNS exposure. The compounds are currently being evaluated in mouse models of PKAN.