Our Oral Testimony to the FDA Advisory Committee on the Approval of AMX0035

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Open Public Hearing

FDA Advisory Committee on AMX0035

March 30, 2022

Good afternoon. My name is Andrea Pauls Backman, CEO of the Les Turner ALS Foundation. My only disclosure is that the Les Turner ALS Foundation receives less than 2% of all annual revenues from pharmaceutical companies, including Amylyx Pharmaceuticals. We are grateful to this Advisory Committee for your dedication to reviewing the science and hearing from the ALS community regarding AMX0035.

My mother, Sally, died from ALS in 2010. So, I come to you today not only as a patient advocate, but as a grieving daughter. I bring both my professional and my very personal passion to my remarks today.

Founded in 1977, the Les Turner ALS Foundation is the oldest independent ALS group in the country. For 45 years, it has been our mission to provide the most comprehensive care and support to people living with ALS and their families so they can confidently navigate the disease. And, for 45 years, we have advanced scientific research for the prevention, treatment and cure of ALS.

Each year, at the Foundation, one-third of the people we directly serve die from ALS.  This month I attended the funeral of an 18-year-old boy who died within a few short months to a very rapid form of ALS. No parents should have to bury a child this way.

The photos you see on the screen represent some of the thousands of people we have had the privilege to serve through our personalized services. ALS can affect anyone, anywhere, at any age. These are the Faces of ALS.

We only have two drugs approved by the FDA to treat ALS. We desperately need more options. We have no time to waste to approve AMX0035.

AMX0035 is the first ALS therapeutic to demonstrate both a statistically significant survival AND functional benefit in ALS. There is no safety signal in AMX0035, and the adequacy and clinical meaningfulness of the data was clearly demonstrated in a well-designed, robust, randomized controlled trial at 25 top clinical trial sites in the U.S. AMX0035 is not perfect, but it is effective in the treatment of people living with ALS.

AMX0035 has been shown to slow ALS disease progression by two points on the ALSFRS-R scale and extend life by 6.5 months. These numbers may not sound like much, but for people who lose their lives within an average of 2 to 5 years, slowing progression extends the precious time families have together.

Slowing the disease progression means more graduations, more weddings, and more family holidays. It means cutting up your own food, speaking on your own, and even breathing on your own. It means more time with those you love. We have no time to waste.

As these FACES of ALS and thousands of others attest, there is an urgent need for access to safe and effective therapies AND regulatory flexibility for unmet medical needs in ALS. We urge the FDA advisory committee to recommend full approval of AMX0035. There is no other moral choice. We have no time to waste.  Thank you.