Tofersen’s development is progressing in fits and starts. That was evident at the U.S. FDA’s March 22 meeting of the Peripheral and Central Nervous System Drugs Advisory Committee, which unanimously agreed Biogen Inc.’s failed phase III study predicted a clinical benefit in treating amyotophic lateral sclerosis (ALS) that includes the rare superoxide dismutase 1 component.
Biogen Inc.’s phase III trial of tofersen in a form amyotrophic lateral sclerosis (ALS) has missed its main goal, but the company said it is still talking with regulators after seeing positive trends in the data.
A single injection of SOD1-targeting RNA into the subpial space, which is below the innermost meningeal layer, was able to spread throughout the spinal cord and, via retrograde delivery, into brain centers that project to the spinal cord in several animal models, including primates.
A single injection of SOD1-targeting RNA into the subpial space, which is below the innermost meningeal layer, was able to spread throughout the spinal cord and, via retrograde delivery, into brain centers that project to the spinal cord in several animal models, including primates.