BIIB
Biogen Inc. is a biopharmaceutical company with commercial-stage products in neurology and immunology, including the monoclonal antibody TYSABRI and small molecules TECFIDERA and VUMERITY for multiple sclerosis, along with LEQEMBI, a small molecule approved for Alzheimer's disease, and SKYCLARYS, a small molecule for rare neurological disease. The company faces significant generic and biosimilar competition for its established products, with TECFIDERA experiencing substantial revenue decline from multiple generic entrants and TYSABRI affected by biosimilar competition approved in 2023. Manufacturing operations are concentrated in the United States, though key products including LEQEMBI, TECFIDERA, and VUMERITY rely on international production facilities, creating exposure to tariffs and supply chain disruptions.
Pipeline
SPINRAZA is an antisense oligonucleotide (ASO) designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency.
The precise mechanism by which omaveloxolone exerts its therapeutic effect in patients with Friedreich's ataxia is unknown. Omaveloxolone has been shown to activate the Nuclear factor (erythroid-derived 2)-like 2 (Nrf2) pathway in vitro and in vivo in animals and humans.
Tacrolimus binds to an intracellular protein, FKBP-12. A complex of tacrolimus-FKBP-12, calcium, calmodulin, and calcineurin (a ubiquitous mammalian intracellular enzyme) is then formed and the phosphatase activity of calcineurin is inhibited.
Tofersen is an antisense oligonucleotide that causes degradation of SOD1 mRNA through binding to SOD1 mRNA, which results in a reduction of SOD1 protein synthesis.