Tofersen is an investigational antisense medicine designed to inhibit the production of superoxide dismutase 1 (SOD1), which is a well understood genetic cause of amyotrophic lateral sclerosis (ALS).
Tofersen is currently being studied in the Phase 3, randomized, placebo-controlled ATLAS study to evaluate whether tofersen can delay clinical onset when initiated in presymptomatic individuals with ALS and a SOD1 genetic mutation. More details about ATLAS(NCT04856982) can be found at clinicaltrials.gov.
SOD1-ALS is a rare, fatal, neurodegenerative disorder caused by a mutation in the SOD1 gene leading to a progressive loss of motor neurons. As a result, people with SOD1-ALS experience increasing muscle weakness, loss of movement, difficulty breathing and swallowing and eventually succumb to the disease. SOD1-ALS is diagnosed in approximately 2% of all ALS cases, impacting about 330 people in the United States. Approximately 5-10% of people with ALS are thought to have a genetic form of the disease; however, they may not have a known family history of the disease.
Safety and efficacy have not been evaluated by any regulatory authorities for the indications described.