- In the Phase 3 VALOR study, the primary endpoint as measured by the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) did not reach statistical significance; however, signs of reduced disease progression across multiple secondary and exploratory endpoints were observed
- The totality of evidence from VALOR and its ongoing open-label extension showed that participants who started tofersen earlier experienced better outcomes, further suggesting a positive clinical effect
- Topline data being presented today at the American Neurological Association 2021 Annual Meeting
- Given the high unmet medical need, Biogen will expand its ongoing early access program (EAP) to the broader SOD1-ALS population
Tofersen is an antisense medicine being estimated for the implicit treatment of SOD1-ALS. Tofersen binds to SOD1 mRNA, allowing for its declination by RNase-H in an trouble to reduce conflation of SOD1 protein product. Tofersen is also being studied in the Phase 3 ATLAS study, which is designed to estimate the capability of tofersen to delay clinical onset when initiated in presymptomatic individualities with a SOD1 inheritable mutation and biomarker substantiation of complaint exertion. Biogen certified tofersen from Ionis Pharmaceuticals,Inc. under a cooperative development and license agreement.
At Biogen, our charge is clear we’re settlers in neuroscience. Biogen discovers, develops and delivers worldwide innovative curatives for people living with serious neurological and neurodegenerative conditions as well as affiliated remedial closeness. One of the world’s first global biotechnology companies, Biogen was innovated in 1978 by Charles Weissmann, Heinz Schaller, Kenneth Murray and Nobel Prize winners Walter Gilbert and Phillip Sharp. Moment Biogen has the leading portfolio of drugs to treat multiple sclerosis, has introduced the first approved treatment for spinal muscular atrophy, commercializes biosimilars of advanced biologics and is concentrated on advancing exploration programs in multiple sclerosis and neuroimmunology, Alzheimer’s complaint and madness, neuromuscular diseases, movement diseases, ophthalmology, neuropsychiatry, immunology, acute neurology and neuropathic pain.
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