ELITE 2020 Drug Researchers and Developers Joshua Cohen and Justin Klee of Amylyx Pharmaceuticals Inc.

Joshua Cohen and Justin Klee

Co-Founders and Co-CEOs

Amylyx Pharmaceuticals Inc.

Potential New Treatment for ALS

On the face of it, the story seems like a scientific fairy tale: Two Brown University undergrads strike up a friendship on a tennis court and develop a shared interest in neurons, and more specifically, how they die. The two guys, Joshua Cohen (a biomedical engineering major) and Justin Klee (a neuroscience major) decided to pursue their hunch about a novel approach that could slow neuronal death. And so, in 2013, they founded Amylyx Pharmaceuticals Inc. Its mission: Develop new treatments for neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS) and Alzheimer’s disease. Their baby: A compound called AMX0035.

Their hypothesis was that the combination of two therapies might have a positive effect on ALS. AMX0035 includes two small molecules—taurursodiol and sodium phenylbutyrate—which 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). When AMX0035 worked in a petri dish, they started Amylyx out of a university dorm room.

Despite the harrowing challenge it has been to treat ALS—over 60 ALS clinical trials have failed in the past 20 years—they designed their clinical trial, called CENTAUR, with input from patients, the Northeast ALS (NEALS) Consortium, the Neurological Clinical Research Institute at Massachusetts General Hospital, and the Barrow Neurological Institute with hope for a different outcome. The trial, which is partly funded by Ice Bucket Challenge donations, included 137 participants aged 18 to 80 who had fast-progressing ALS.

In December 2019, the results from the CENTAUR Phase 2 clinical trial showed AMX0035 significantly slowed disease progression in patients with ALS compared to placebo. Amylyx is working to push ahead with AMX0035 as quickly as possible.

In seven years, Joshua and Justin went from a concept in their dorm room to collaborating with leaders in the ALS community, leading to a successful trial in a devastating disease. In the words of Justin Klee: “With these results, Amylyx now has a responsibility to move ahead as efficiently as possible, as people living with ALS don’t have time to wait.”

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