Biogen announces publication of long term results from Phase 3 VALOR study

Biogen (BIIB) announced that The Journal of the American Medical Association Neurology has published final results from the completed Phase 3 VALOR study and its open-label extension study evaluating QALSODY for the treatment of superoxide dismutase 1 amyotrophic lateral sclerosis with over 3.5 years of follow-up. These results show that early initiation of QALSODY was associated with numerically slower decline in measures of clinical function, breathing and strength, as well as reduction in the risk of death or permanent ventilation. Sustained reductions in neurofilament, a marker of neurodegeneration, further validate the clinical results and demonstrate QALSODY’s impact on the underlying biology of SOD1-ALS. QALSODY 100 mg/15mL injection is approved for the treatment of amyotrophic lateral sclerosis in adults who have a mutation in the superoxide dismutase 1 gene. In the United States, QALSODY received accelerated approval based on reduction in plasma neurofilament light chain observed in patients treated with QALSODY. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial. QALSODY has received approval in 44 countries around the world, including accelerated approval in the US and conditional or standard approval in other countries. VALOR was a six-month Phase 3, randomized, double-blind, placebo-controlled study to evaluate the effects of tofersen 100 mg in adults with ALS associated with a SOD1 mutation. In total, 108 participants were randomized in VALOR. Of these participants, 95 enrolled in the OLE. At the completion of the OLE, the median opportunity for follow-up was 4.9 years. At the conclusion of VALOR and the OLE, the most common adverse events were headache, procedural pain, fall, back pain and extremity pain. Serious neurological AEs of myelitis or radiculitis, papilledema and/or increased intracranial pressure, and chemical or aseptic meningitis were reported in nine participants. These events were manageable with standard of care and resolved. One myelitis event and one chemical meningitis event led to treatment discontinuation. Safety findings are consistent with previously reported results.