Biogen (BIIB)- The company announced interim results from a phase 1/2 study of tofersen (antisense oligonucleotide) being studied for the treatment of amyotrophic lateral sclerosis ((ALS)). Data was sufficiently encouraging to support initiation of phase 3 study in SOD1-ALS patients and will be presented at the American Academy of Neurology Annual Meeting ((AAN)) by co-principal investigator Timothy M. Miller, M.D., Ph.D., on May 7th as a late breaking oral presentation.
Interim analysis showed that 10 patients treated with tofersen 100mg over three-month period experienced lowering of SOD1 protein levels in the cerebrospinal