Benzinga | Apr 20, 2021 07:09AM EDT

Ionis Initiates Pivotal Clinical Study Of ION373 In Alexander Disease

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced it has initiated a pivotal clinical study of ION373 in patients with Alexander disease, a rare and generally fatal neurological disorder. Alexander disease patients experience progressive deterioration, leading to severe disability and loss of independence. Childhood onset is associated with more serious disease, with patients rarely surviving beyond adolescence. Alexander disease is caused by a genetic mutation that leads to overproduction and toxic accumulation of glial fibrillary acidic protein (GFAP) in the brain. ION373 is an investigational antisense medicine designed to reduce the level of GFAP.