Tofersen (Isis-SOD1Rx

Drug Name: Tofersen (Isis-SOD1Rx
Company: Biogen
Location: US-Massachusetts
Drug Type: RNA-Targeted Therapy
Conditions: ALS
Mechanism Type: SOD1-Lowering Therapy
Mechanism: IONIS-SOD1Rx is a generation 2.0 antisense drug specifically designed to inhibit production of mutant superoxide dismutase (SOD1). SOD1 mutations account for approximately 20% of familial ALS cases.
U.S. Status for ALS: Phase III

[1] Single and Multiple Dose Study of BIIB067 (Isis-SOD1Rx) in Adults With Amyotrophic Lateral Sclerosis (ALS)., 1 Feb 2016. Accessed 9 Mar 2016 from
[2] SOD1 in cerebral spinal fluid as a pharmacodynamic marker for antisense oligonucleotide therapy. Winer, L. et al. JAMA Neurol. 2013 Feb;70(2):201-7.
[3] An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study. Miller, TM et al. Lancet Neurol. 2013 May;12(5):435-42.

Last updated April 25th, 2019

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