BioCentury
ARTICLE | Clinical News

SOD1 antisense oligonucleotide improves survival in ALS model

August 17, 2018 3:30 AM UTC

Researchers at Biogen Inc. (NASDAQ:BIIB), Ionis Pharmaceuticals Inc. (NASDAQ:IONS) and colleagues reported data from a rat model of superoxide dismutase 1 (SOD1)-mutated amyotrophic lateral sclerosis (ALS) showing that a single bolus dose of intrathecal IONIS-SOD1Rx (BIIB067) increased survival by up to 64 days compared with vehicle. In a mouse model of the disease, IONIS-SOD1Rx also preserved compound muscle action potential and maintained muscle function of the tibialis anterior muscles in the hind limbs vs. vehicle. Data were published in the Journal of Clinical Investigation.

IONIS-SOD1Rx is an antisense inhibitor of SOD1 that is in a Phase I/IIa trial to treat patients with ALS...