2021 Champions for Cures and Care - Flipbook - Page 20
ESSEY COMMITMENT TO CURES AWARD
AMYLYX PHARMACEUTICALS
“Why do neurons die?” These four words, asked in a dorm room at Brown University,
would be the inspiration to launch a seven-year journey to develop novel therapeutics for
ALS and other neurodegenerative diseases.
This incredible story sounds like a Hollywood movie: Two undergrads strike up a friendship
on a tennis court and discover a shared interest in neurons, and, more specifically, how
they die. They decide to pursue their hunch about a novel approach that could slow
neuronal death, and take the world of science by storm. But this story is true.
After methodically poring over an endless supply of scientific literature, Joshua Cohen (a
biomedical engineering major) and Justin Klee (a neuroscience major) decided the best
way to prevent degradation in these diseases was to focus on preserving neurons. They
hypothesized that the combination of two therapies might have a positive effect on ALS.
Joshua received his degree in biomedical engineering from Brown University and gained
research experience at Brown University and at the National Institute of Standards in
Technology. Justin also conducted research at Brown University in neural systems and
neurophysiology and Alzheimer’s disease at Harvard Medical School, before receiving his
degree in Neuroscience from Brown University. In 2013, the two friends founded Amylyx
Pharmaceuticals, dedicated to the development of therapeutics for the treatment of
neurodegenerative disorders.
Josh Cohen and Justin Klee, Co-CEOs & Co-Founders, Amylyx Pharmaceuticals
The company’s current focus is on a compound called AMX0035, which includes two small
molecules that act synergistically to prevent nerve cell death by blocking stress signals
within the mitochondria (which provide cells with energy), and the endoplasmic reticulum
(which is involved in making proteins). The company incorporates unconventional
approaches through strong partnerships with industry leaders, scientists, doctors, and
organizations.
Despite the challenges of launching an ALS clinical trial, they designed their study,
named CENTAUR, with input from patients, the Northeast ALS (NEALS) Consortium,
the Neurological Clinical Research Institute at Massachusetts General Hospital, and
the Barrow Neurological Institute. The trial was partly funded by Ice Bucket Challenge
donations. In December 2019, the results from the CENTAUR Phase 2 trial showed
AMX0035 significantly slowed disease progression in patients with ALS compared to