The New England Journal of Medicine Publishes Pivotal Tofersen Data that Show Benefits in Rare, Genetic Form of ALS

  • Publication includes the Phase 3 VALOR SOD1-ALS trial and its open-label-extension study, underscoring the importance of these longer-term data
  • 12-month data show earlier initiation of tofersen slowed decline across critical measures of function and strength in people diagnosed with SOD1-ALS
  • Results offer important learnings regarding the biology of SOD1-ALS and design of ALS clinical trials

BiogenInc.( Nasdaq BIIB) moment blazoned that The New England Journal of Medicine( NEJM) has published detailed results from the Phase 3 VALOR study and the combined analysis of VALOR and its open marker extension( OLE) study assessing tofersen for the treatment of superoxide dismutase 1( SOD1) amyotrophic side sclerosis( ALS). There’s presently no treatment targeted for SOD1- ALS.

“ I see three crucial take home points from these data. First, tofersen easily leads to lowering of SOD1 protein, as would be anticipated. Second there’s substantial lowering of neurofilament situations, which I interpret as potentially decelerating the underpinning complaint process. And third, there’s a meaningful clinical benefit when looking at the after time points in the open marker extension, ” said Timothy Miller,M.D.,Ph.D., top investigator of VALOR and ALS Centerco-Director at Washington University School of Medicine,St. Louis. “ We’re thankful to the fidelity from actors, their families, and the spots for taking part in this important study. ”

“ The ALS community has been laboriously pursuing new drugs for decades. To have data like these published in NEJM gives us energy and stopgap. We’re now seeing in the data what we suspected about tofersen for a long time – that it has the implicit to make a clinical difference for people living with SOD1- ALS, ” said Merit Cudkowicz,M.D.,co-principal investigator of the VALOR trial andco-founder of the Northeast ALS Consortium, Director of the Healey & AMG Center for ALS and Chair of Neurology at Massachusetts General Hospital and the Julieanne Dorn Professor of Neurology at Harvard Medical School. “ The lowering of neurofilament, a marker of axonal injury and neurodegeneration along with the clinical data, highlights the eventuality of tofersen. ”

About VALOR and the OLE
VALOR was a six- month Phase 3, randomized, double-eyeless, placebo- controlled study to estimate the goods of tofersen 100 mg in grown-ups with ALS associated with a SOD1 mutation. In total, 108 actors were randomized in VALOR( n = 72 to tofersen 100 mg and n = 36 to placebo). Of these actors, 95 enrolled in the ongoing OLE. At the time of the analysis all actors had an occasion for at least 12 months of follow- up, with a standard exposure to tofersen of roughly 20 months( range 1 – 34 months).

The primary endpoint of VALOR was change from birth to week 28 in ALS Functional Rating Scale- Revised( ALSFRS- R) total score. Secondary endpoints included changes in total cerebrospinal fluid SOD1 protein attention, tube neurofilament light chain( NfL), slow vital capacity and handheld dynamometry in 16 muscles.
About Tofersen
Tofersen is an antisense medicine being estimated for the implicit treatment of SOD1- ALS. Tofersen binds and degrades SOD1 mRNA to reduce conflation of SOD1 protein product. In addition to the ongoing open marker extension of VALOR, tofersen is being studied in the Phase 3 ATLAS study designed to estimate whether tofersen can 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.

About Amyotrophic Side Sclerosis and SOD1- ALS
Amyotrophic side sclerosis( ALS) is a rare, progressive and fatal neurodegenerative complaint that results in the loss of motor neurons in the brain and the spinal cord that are responsible for controlling voluntary muscle movement. People with ALS experience muscle weakness and atrophy, causing them to lose independence as they steadily lose the capability to move, speak, eat, and ultimately breathe. Average life expectation for people with ALS is three to five times from time of symptom onset.1

Biogen’s nonstop Commitment to ALS
For over a decade, Biogen has been committed to advancing ALS exploration to give a deeper understanding of all forms of the complaint. The company has continued to invest in and colonist exploration despite making the delicate decision to discontinue a late- stage ALS asset in 2013. Biogen has applied important literacy to its portfolio of means for inheritable and other forms of ALS, with the thing of adding the probability of bringing a eventuality remedy to cases in need. These applied literacy include assessing genetically validated targets in defined case populations, pursuing the most applicable modality for each target and employing sensitive clinical endpoints. moment, the company has a channel of investigational medicines being estimated in ALS, including tofersen and BIIB105.

About Biogen
As settlers in neuroscience, Biogen discovers, develops, and delivers worldwide innovative curatives for people living with serious neurological 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, Sir Kenneth Murray, and Nobel Prize winners Walter Gilbert and Phillip Sharp. moment, Biogen has a leading portfolio of drugs to treat multiple sclerosis, has introduced the first approved treatment for spinal muscular atrophy, and developed the first and only approved treatment to address a defining pathology of Alzheimer’s complaint. Biogen is also commercializing biosimilars and fastening on advancing one of the assiduity’s most diversified channels in neuroscience that will transfigure the standard of care for cases in several areas of high unmet need.

In 2020, Biogen launched a bold 20- time,$ 250 million action to address the deeply interrelated issues of climate, health, and equity. Healthy Climate, Healthy Lives ™ aims to exclude fossil energies across the company’s operations, make collaborations with famed institutions to advance the wisdom to ameliorate mortal health issues, and support underserved communities.

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