Comments on Amylyx Pharmaceuticals’ New Drug Application for AMX0035

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RE: Docket No. FDA-2018-N-0410

Dear FDA Advisory Committee:

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 advance scientific research for the prevention, treatment and cure of ALS. We are
committed to providing access to the most promising therapies for people living with this
devastating disease. We have no time to waste.

ALS is a terminal and progressive disease that causes muscle weakness, difficulty speaking
and swallowing and, generally, complete paralysis. ALS can, in some cases, also cause changes
in intellectual function, mood, behavior or personality. While technically considered a “rare
disease,” experts predict an individual’s lifetime risk of acquiring ALS is about 1 in 300 by the
age of 85. There is no cure, yet.

In the 45 years of the existence of the Les Turner ALS Foundation, only two drugs have been
approved by the FDA to treat ALS. We desperately need more options to treat ALS. 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, the adequacy and clinical
meaningfulness of the data was clearly demonstrated in a well-designed, robust and
randomized controlled trial at 25 top clinical trial sites in the United States. There is an urgent
need for access to safe and effective therapies and regulatory flexibility for unmet medical
needs. We have no time to waste.

ALS is scary. Really scary. For people living with ALS and their caregivers, life has become so
tumultuous that they often feel exhausted, overwhelmed and hungry for emotional comfort and
effective treatments.

Since no two people with ALS are alike or will progress the same way, our Les Turner ALS
Foundation ALS Support Services Coordinators work with people with ALS on a personalized
basis to manage the rapid progression, psychosocial needs and constant losses of function. We
do this by working in close partnership with clinicians at the Lois Insolia ALS Clinic at the Les
Turner ALS Center at Northwestern Medicine. We help them adjust to speech changes, help
them and their families make decisions about nutrition and breathing support, and help them
with the constant flow of new equipment to ensure safety in the home.

Each year, we lose about one-third of the people we directly serve to ALS. This month alone,
we tragically lost an 18 year-old boy in a few short months to a very rapid form of ALS. Despite
our 45 years of experience, we were not able to keep up with the progression of this disease.
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 a
disease that takes lives within an average of 2-5 years, this slowing of progression extends the
time the families who face this life-threatening disease have together. Slower disease
progression is meaningful to each person living with ALS and their loved ones.

Slowing the disease progression means more graduations, more weddings and more family
holidays. Slowing the disease progression means cutting up your own food, speaking on your
own and even breathing on your own. Slowing the disease progression means more handwritten
letters to loved ones, more climbs up the stairs to sleep in your room and simply more time with
those you love. We have no time to waste.

We urge the FDA advisory committee to recommend full approval of AMX0035. We have no
time to waste.

Sincerely,

Andrea Pauls Backman, MBA
Chief Executive Officer

 

Disclosure: As a non-profit, we receive less than 2% of our annual funding from Amylyx
Pharmaceuticals, Inc. as well as other pharmaceutical companies.