Key clinical point: The investigational SOD1 antisense oligonucleotide tofersen reduced SOD1 protein in patients with amyotrophic lateral sclerosis.

Major finding: A reduction from baseline in cerebrospinal fluid SOD1 concentrations was observed in the tofersen 40-, 60-, and 100-mg cohorts with the maximal reduction observed in the 100 mg–treated group (37% vs. no reduction in the placebo group; P less than .002).

Study details: A double-blind, randomized, placebo-controlled, phase 1/2 study of 50 patients.

Disclosures: Biogen sponsored the study, and some of the investigators are Biogen employees. Dr. Miller is on Biogen’s medical advisory board and receives clinical research support from Biogen. In addition, he consults, has licensing agreements with, and is a principal investigator for other companies.