Amylyx Pharmaceuticals
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Amylyx Pharmaceuticals, Inc is a
biopharmaceutical A biopharmaceutical, also known as a biological medical product, or biologic, is any pharmaceutical drug product manufactured in, extracted from, or semisynthesized from biological sources. Different from totally synthesized pharmaceuticals, t ...
company headquartered in
Cambridge, Massachusetts Cambridge ( ) is a city in Middlesex County, Massachusetts, United States. It is a suburb in the Greater Boston metropolitan area, located directly across the Charles River from Boston. The city's population as of the 2020 United States census, ...
. Amylyx is best known for AMX0035, an experimental therapy for
amyotrophic lateral sclerosis Amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (MND) or—in the United States—Lou Gehrig's disease (LGD), is a rare, Terminal illness, terminal neurodegenerative disease, neurodegenerative disorder that results i ...
. AMX0035 was approved for medical use in Canada as Albrioza, in June 2022, and in the United States, as Relyvrio, in September 2022. It was withdrawn in April, 2024.


History

Amylyx Pharmaceuticals was founded in 2013 by Joshua Cohen and Justin Klee when the two were undergraduate students at
Brown University Brown University is a Private university, private Ivy League research university in Providence, Rhode Island, United States. It is the List of colonial colleges, seventh-oldest institution of higher education in the US, founded in 1764 as the ' ...
. The two proposed that taurursodiol and
sodium phenylbutyrate Sodium phenylbutyrate, sold under the brand name Buphenyl among others, is a salt of an aromatic fatty acid, 4-phenylbutyrate (4-PBA) or 4-phenylbutyric acid. The compound is used to treat urea cycle disorders, because its metabolites offer an ...
together might prevent dysfunction of
mitochondria A mitochondrion () is an organelle found in the cells of most eukaryotes, such as animals, plants and fungi. Mitochondria have a double membrane structure and use aerobic respiration to generate adenosine triphosphate (ATP), which is us ...
and the
endoplasmic reticulum The endoplasmic reticulum (ER) is a part of a transportation system of the eukaryote, eukaryotic cell, and has many other important functions such as protein folding. The word endoplasmic means "within the cytoplasm", and reticulum is Latin for ...
, safeguarding
neuron A neuron (American English), neurone (British English), or nerve cell, is an membrane potential#Cell excitability, excitable cell (biology), cell that fires electric signals called action potentials across a neural network (biology), neural net ...
s. Klee and Cohen were advised by Rudolph E. Tanzi, who ultimately served as the founding chair of Amylyx's
scientific research The scientific method is an empirical method for acquiring knowledge that has been referred to while doing science since at least the 17th century. Historically, it was developed through the centuries from the ancient and medieval world. The ...
board. After Klee and Cohen were able to achieve strong results in pre-clinical research, Tanzi connected the two to his colleagues at
Massachusetts General Hospital Massachusetts General Hospital (Mass General or MGH) is a teaching hospital located in the West End neighborhood of Boston, Massachusetts. It is the original and largest clinical education and research facility of Harvard Medical School/Harvar ...
, which culminated in clinical trials and FDA approval. The company was granted $2.2 million by the
ALS Association The ALS Association is an American nonprofit organization that funds global amyotrophic lateral sclerosis (ALS) research, provides care services and programs to people affected by ALS through its nationwide network of clinical care centers, and w ...
with funds raised through the
Ice Bucket Challenge The Ice Bucket Challenge, sometimes called the ALS Ice Bucket Challenge, is an activity involving the pouring of a bucket of ice water over a person's head, either by another person or self-administered, to promote awareness of the disease amyo ...
. Amylyx went public in January 2022. The company's shares began trading on the
Nasdaq The Nasdaq Stock Market (; National Association of Securities Dealers Automated Quotations) is an American stock exchange based in New York City. It is the most active stock trading venue in the U.S. by volume, and ranked second on the list ...
on January 7, 2022 under the symbol AMLX. After a disappointing Phase 3 trial of AMX0035 failed to produce significant differences versus a placebo, on April 4, 2024 Amylyx announced they would begin the process of withdrawing Albrioza and Relyviro from the North American market. Amylyx received praise for keeping its pre-approval promise to stop selling the drug if it turned out to be ineffective, an unusually ethical move which the FDA rarely requires. The company cited its independence from major pharmaceutical companies as a reason it could do so.


References

{{authority control 2013 establishments in Massachusetts American companies established in 2013 Biotechnology companies established in 2013 Biotechnology companies of the United States Companies based in Cambridge, Massachusetts Pharmaceutical companies established in 2013