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NURTURE-studien publiceras i tidskriften Neuromuscular Disorders. I studien undersöktes presymptomatisk behandling med Spinraza® (nusinersen) av spädbarn med Spinal Muskel Atrofi

Pressmeddelande   •   Okt 02, 2019 13:52 CEST

  • Behandling med Spinraza innebar en 100 procentig överlevnad utan att någon patient behövde permanent ventilationshjälp.
  • Milstolpar för motorisk funktion som uppnåddes var att 100 procent av studiedeltagarna kunde sitta utan stöd och att 88 procent klarade att gå på egen hand. Dessa resultat skiljer sig avsevärt mot vad man kan förvänta sig utifrån sjukdomens naturliga förlopp.
  • Studieresultaten bekräftar den befintliga säkerhetsprofilen och den dokumenterade effekten av Spinraza som är den enda godkända behandlingen av SMA hos spädbarn, barn och vuxna.

  • Cambridge, Mass.,Oct 2, 2019 –Biogen Inc. (Nasdaq: BIIB) today announced that the journal Neuromuscular Disorders has published data from NURTURE, the first study investigating a treatment targeting the underlying cause of spinal muscular atrophy (SMA) in infants treated pre-symptomatically. Data from the NURTURE study demonstrated that infants who initiated treatment with nusinersen prior to the onset of clinical symptoms attained unparalleled results compared to the natural history of the disease. As of March 2019 all participants were alive, without the need for permanent ventilation and experienced continuous improvements with the majority achieving motor milestones in timelines consistent with normal development. The results also demonstrated durability of effect with children making progress throughout the study.

    NURTURE is an ongoing, Phase 2, open-label study of 25 infants with the genetic diagnosis of SMA (most likely to develop SMA Type 1 or 2) who received their first dose of nusinersen in the pre-symptomatic stage and before six weeks old. The study, conducted at 15 sites in seven countries, has results up to 45.4 months. When compared with the natural history of the disease, the results are significant in their impact on changing the course of SMA.

    As of March 2019 all infants in the study were 25 months or older, past the typical age of symptom onset for SMA Type 1 and Type 2, and were alive without the need for permanent ventilation. In comparison to the natural history of SMA, many of these infants would likely have passed away or require permanent ventilation on average by 13.5 months. In both the children with two and three copies of SMN2, treatment with Spinraza demonstrated rapid onset of improvement and durability of effect with their mean Children’s Hospital of Pennsylvania Infant Test of Neuromuscular Disorders (CHOP-INTEND) score of motor function reaching the maximum mean score of 64 for all participants with three copies of SMN2 (n=10) and a mean of 62.1 for those with two copies of SMN2 (N=15).

    Additional highlights include:

    • The majority of study participants achieved motor milestones in timeframes consistent with the World Health Organization (WHO) standards, with 100 percent sitting independently and 88 percent walking independently.
    • Hammersmith Infant Neurologic Examination, Section 2 (HINE-2) development of motor function scores increased for all participants with the mean score for both those with two or three SMN2 copies approaching the maximum score of 26 points at the last assessment.
    • SPINRAZA demonstrated longer term efficacy up to nearly four years, with participants continuing to make progress.
    • SPINRAZA was well-tolerated with no new safety concerns identified after up to nearly four years of treatment.

    These published results from the NURTURE study were previously presented at the 2019 Cure SMA Annual SMA Conference and the 5th Congress of the European Academy of Neurology.

    Om Spinraza
    Spinraza är avsett för behandling av spinal muskelatrofi av typ 5q. Biverkningar som observerats är fall av allvarlig infektion, såsom meningit. Det har även förekommit rapporter om kommunicerande hydrocefalus, aseptisk meningit och överkänslighet (t.ex. angioödem, urtikaria och hudutslag). För information om kontraindikationer, varningar och försiktighet, biverkningar, dosering och förpackningar se www.fass.se.

    About Biogen
    At Biogen, our mission is clear: we are pioneers in neuroscience. Biogen discovers, develops and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases as well as related therapeutic adjacencies. One of the world’s first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Kenneth Murray and Nobel Prize winners Walter Gilbert and Phillip Sharp, and today has the leading portfolio of medicines to treat multiple sclerosis, has introduced the first approved treatment for spinal muscular atrophy, commercializes biosimilars of advanced biologics and is focused on advancing research programs in multiple sclerosis and neuroimmunology, neuromuscular disorders, movement disorders, Alzheimer’s disease and dementia, ophthalmology, immunology, neurocognitive disorders, acute neurology and pain.

    Biogen Safe Harbor
    This news release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, about the potential benefits, safety and efficacy of SPINRAZA; the results of certain real-world data; the identification and treatment of SMA; our research and development program for the treatment of SMA; and the potential benefits and results from early treatment of SMA. These statements may be identified by words such as “aim,” “anticipate,” “believe,” “could,” “estimate,” “expect,” “forecast,” “goal,” “intend,” “may,” “plan,” “possible,” “potential,” “will,” “would” and other words and terms of similar meaning. You should not place undue reliance on these statements or the scientific data presented.

    These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including without limitation risks relating to the occurrence of adverse safety events and/or unexpected concerns that may arise from additional data or analysis; failure to obtain regulatory approvals in other jurisdictions; risks of unexpected costs or delays; failure to protect and enforce our data, intellectual property and other proprietary rights and uncertainties relating to intellectual property claims and challenges; regulatory authorities may require additional information or further studies; product liability claims; and third party collaboration risks. The foregoing sets forth many, but not all, of the factors that could cause actual results to differ from our expectations in any forward-looking statement. Investors should consider this cautionary statement, as well as the risk factors identified in our most recent annual or quarterly report and in other reports we have filed with the U.S. Securities and Exchange Commission. These statements are based on our current beliefs and expectations and speak only as of the date of this news release. We do not undertake any obligation to publicly update any forward-looking statements, whether as a result of new information, future developments or otherwise.

    References

    1 Darras BT, Chiriboga CA, Iannaccone ST, et al. Nusinersen in later-onset spinal muscular atrophy: Long-term results from the phase 1/2 studies. Neurology. 2019 May 21;92(21):e2492-e2506.

    2 Finkel R, Chiriboga C, Vajsar J, et al. Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study. Lancet. 2016;388(10063):3017-3026.

    3 Darras B, Jones Jr. H.R., Ryan M., De Vivo D. Chapter 8 - Spinal Muscular Atrophies. In: Vivo BTD, ed. Neuromuscular Disorders of Infancy, Childhood, and Adolescence (Second Edition). San Diego: Academic Press; 2015:117-145.

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    MEDIA CONTACTS :
    Mats Ekelund
    +46 76 182 36 27
    mats.ekelund@biogen.com