Core Insights - Biogen announced the final results of the Phase 3 VALOR study and its open-label extension, demonstrating that early initiation of QALSODY (tofersen) is associated with a slower decline in clinical function, breathing, and strength in patients with SOD1-ALS, along with a reduced risk of death or permanent ventilation [1][2][7] Company Overview - Biogen is a leading biotechnology company focused on innovative science to develop new medicines, with a commitment to advancing ALS research and treatment [14][13] - The company has received approval for QALSODY in 44 countries, including accelerated approval in the US [3] Study Details - The VALOR study was a six-month Phase 3, randomized, double-blind, placebo-controlled trial involving 108 participants, with 72 receiving tofersen and 36 receiving a placebo [4] - The open-label extension study had a median follow-up of 4.9 years, with significant findings regarding muscle strength improvements in 27% of early-start participants [5][6] Treatment Mechanism - QALSODY is an antisense oligonucleotide designed to reduce SOD1 protein production, which is implicated in the degeneration of motor neurons in SOD1-ALS [8][11] - The treatment is administered intrathecally with a specific dosing regimen, and its approval in the US is based on the reduction of plasma neurofilament light chain (NfL) [8][10] Future Research - Biogen is advancing additional ALS research, including the ongoing QALSODY ATLAS study aimed at evaluating the treatment's potential to delay clinical onset in presymptomatic individuals with SOD1 mutations [10][7] - The company is also exploring a robust discovery pipeline targeting TDP43 pathology, which is present in 97% of ALS cases [13]