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Ann: Appendix 4C and Quarterly Update, page-23

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    lightbulb Created with Sketch. 1149
    Some may find the Endpoints article interesting.
    Seeing that this was in the 4C I did not think it would be out of place.

    "The ALS/MND addressable market is US$3.6Bn with Riluzole already reaching ~US$1Bn in annual sales.
    According to the International Alliance of ALS/MND Associations, MND affects over 350,000 of the world’s
    population and kills more than 100,000 people every year."

    Check the high lighted part if nothing else.



    Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

    For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

    US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

    “Amylyx’s goal is that every person who is eligible for RELYVRIO will have access as quickly and efficiently as possible as we know people with ALS and their families have no time to wait,” co CEOs Joshua Cohen and Justin Klee said in an emailed statement. “Our teams are in place and ready to help people living with ALS gain access to this important new therapeutic option. While Amylyx is working on launching RELYVRIO, healthcare professionals will be able to write prescriptions for RELYVRIO immediately by enrolling their patients into our comprehensive support program that we are implementing.”

    There’s no information yet on price, and Endpoints News followed up with Amylyx for comment. Relyvrio, costs about $163,000 in Canada, where the drug was conditionally approved in June and is branded as Albrioza.

    Relyvrio is not a cure for ALS, and FDA scientists repeatedly questioned its ability to slow disease progression. The green light was based on a single, placebo-controlled Phase II study showing a limited, yet statistically significant, impact on a rating scale that measures how quickly patients’ symptoms progress. Amylyx also submitted data from an open-label extension purporting to show the drug extends patients’ lives by a few months.

    ALS is a devastating, universally fatal disease, with most patients succumbing within two to five years after their symptoms start. The FDA previously approved two other ALS drugs in riluzole, greenlit in the 1990s, and edaravone, in 2017. But their effects on function, survival and symptom management are modest. Earlier this year, the agency also approved a new, oral formulation of edaravone, which is normally given through IV infusion.

    But with its approval Thursday, Relyvrio becomes the third new therapy available to American ALS patients and represents the culmination of a resonant and highly organized advocacy campaign. Organizations such as the ALS Association and I Am ALS had mounted full-court press awareness campaigns and lobbying efforts, recruiting several members of Congress to sponsor legislation that would make it easier to approve new treatments.

 
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