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“Disappointment tinged with some encouragement”Phase 3 VALOR trial of Tofersen update

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    “Disappointment tinged with some encouragement”Phase 3 VALOR trial of Tofersen update

    We are disappointed to hear the announcement from Biogen that the Phase 3 VALOR trial of Tofersen hasn’t achieved its primary endpoint in significantly slowing disease progression.

    There was a lot of interest in this trial within the MND and scientific community and we know this is not the news people wanted to hear.

    The primary endpoint measure of the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS‐R) which evaluates the functional status of people living with MND did not show a statistically significant difference – that means treatment with Tofersen did not result in improvements in people living with MND compared to those given a placebo.

    There were however some potentially encouraging indications when looking at other measures of disease activity that require further detailed investigation. The trial evidence, along with evidence from the ongoing open-label extension (in which patients and the trial clinicians know they are receiving the trial drug) has shown trends, including breathing and muscle strength measures, indicating that people taking part who started Tofersen earlier may have experienced better outcomes.

    The data also showed that the levels of SOD1 protein in the cerebrospinal fluid were lowered by the drug. The levels of neurofilament protein (a potential marker of nerve cell damage) were also reduced, suggesting that some clinical effect was occurring.

    Biogen is conducting further analysis of the data to gain more understanding of what the results mean and how to move forward. The company has stated it intends to extend its ongoing Early Access Programme so that more people with MND caused by SOD1 gene mutations can receive the trial drug.

    The MND Association is in contact with Biogen and the researchers involved and we will of course provide updates as soon as they become available.

    The MND Association is extremely grateful to all involved in this important study particularly the study participants, families and carers, the researchers and the entire MND community, without whom these important studies would not be possible.

    The MND Association’s Director of Research Development Dr Brian Dickie said:
    “The disappointment that will be felt amongst the MND community is tinged with some encouragement that the trends in the secondary measures are consistently heading in the right direction. There are questions that need to be addressed, on whether the drug needs to be given earlier in the disease and over a longer time period, as well as whether the levels of the toxic SOD1 protein are still too high and need to be knocked down even more.”

    The positive indications from this trial are further evidence that MND research is on the cusp of making important and potentially life-changing breakthroughs. With a more coordinated approach and the injection of meaningful funding from the Government – as we are campaigning for with the United To End MND coalition – research will deliver the answers the MND community desperately wants and needs.

    You can read the response from Biogen to the MND Associaton’s Director of External Affairs Chris James here
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    #2
    Words impress less than actions
    Bulbar started Jan 2020. Mute and 100% tube fed but mobile and undefeated. Stay Strong 🤗😘🤗😁xx

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      #3
      More money down the drain. Time for a Coup de tats in these Decision making groups, The old ways aint working.

      Comment


        #4
        Yet more disappointment.

        Without knowing EXACTLY which type of MND is being treated, it is hard to imagine that success will follow.

        Otherwise the molecular modelling needs a whole lot of refinement.

        Very disappointing.
        Copyright Graham

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