Biogen Inc. (BIIB) said The Journal of the American Medical Association (JAMA) Neurology has published final results from the completed Phase 3 VALOR study and its open-label extension study OLE that evaluates QALSODY or tofersen for the treatment of superoxide dismutase 1 or SOD1 amyotrophic lateral sclerosis or ALS with over 3.5 years of follow-up.
Timothy Miller, M.D., PhD, principal investigator of VALOR and ALS Centre Director at Washington University School of Medicine, said that for people living with ALS, irreversible loss of muscle strength is a foundational symptom of the disease. In the QALSODY study, 27% of study participants in the early-start group experienced improvements in muscle strength over about 3 years.
Amyotrophic lateral sclerosis is a rare and relentlessly progressive neurodegenerative disorder that destroys motor neurons in the brain and spinal cord, the cells responsible for voluntary muscle control.
SOD1-ALS is diagnosed in approximately 2 percent of all ALS cases, with about 330 people in the United States living with the disease. The disease is ultimately fatal, with most patients surviving only three to five years after the first symptoms appear.
QALSODY, chemically known as tofersen, is an antisense oligonucleotide designed to bind to SOD1 mRNA to reduce SOD1 protein production. In the U.S., QALSODY is indicated for the treatment of ALS in adults who have a mutation in the SOD1 gene.
In April 2023, the QALSODY 100 mg/15mL injection was approved for the treatment of amyotrophic lateral sclerosis (ALS) in adults with a mutation in the superoxide dismutase 1 (SOD1) gene. In the United States, QALSODY received accelerated approval based on a reduction in plasma neurofilament light chain (NfL) observed in patients treated with QALSODY. However, continued approval for this indication will be contingent on confirmatory clinical trials.
VALOR was a six-month Phase 3, randomised, double-blind, placebo-controlled study to evaluate the effects of tofersen 100 mg in adults with ALS associated with a SOD1 mutation.
A total of 108 people took part in the VALOR study, in which 72 received tofersen, and 36 received a placebo. Of these, 95 continued into the open-label extension. After the extension, the average opportunity for follow-up was 4.9 years, in the range 3.6-5.4 years.
In addition to the ongoing OLE of VALOR, QALSODY is being studied in the Phase 3, randomised, placebo-controlled ATLAS study to evaluate whether QALSODY can delay the clinical onset of ALS when initiated in presymptomatic individuals with an SOD1 genetic mutation and biomarker evidence of disease activity, such as elevated plasma NfL.
Over the year, Biogen shares traded in the range of $110.04 -$185.17.
BIIB closed Monday's trade at $174.70, down 0.06%.
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