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. As part of the Boston metropolitan area, the cities population of the 2020 U.S. census was 118,403, making it the fourth most populous city in the state, behind Boston, ...

. Amylyx is best known for

AMX0035 Sodium phenylbutyrate/ursodoxicoltaurine, also known as sodium phenylbutyrate/taurursodiol and sold under the brand name Albrioza among others, is a fixed-dose combination medication used for the treatment of amyotrophic lateral sclerosis (ALS). ...

, an experimental therapy for

amyotrophic lateral sclerosis Amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (MND) or Lou Gehrig's disease, is a neurodegenerative disease that results in the progressive loss of motor neurons that control voluntary muscles. ALS is the most comm ...

. AMX0035 was approved for medical use in Canada as Albrioza, in June 2022, and in the United States, as Relyvrio, in September 2022.

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 research university in Providence, Rhode Island. Brown is the seventh-oldest institution of higher education in the United States, founded in 1764 as the College in the English Colony of Rhode Island and Providenc ...

. 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 disfunction of

mitochondria A mitochondrion (; ) is an organelle found in the Cell (biology), cells of most Eukaryotes, such as animals, plants and Fungus, fungi. Mitochondria have a double lipid bilayer, membrane structure and use aerobic respiration to generate adenosi ...

and the

endoplasmic reticulum The endoplasmic reticulum (ER) is, in essence, the transportation system of the eukaryotic cell, and has many other important functions such as protein folding. It is a type of organelle made up of two subunits – rough endoplasmic reticulum ( ...

, safeguarding

neuron A neuron, neurone, or nerve cell is an electrically excitable cell that communicates with other cells via specialized connections called synapses. The neuron is the main component of nervous tissue in all animals except sponges and placozoa. N ...

s. Klee and Cohen were advised by Rudolph E. Tanzi, who ultimately served as the founding chair of Amylyx's

scientific advisory board Science is a systematic endeavor that builds and organizes knowledge in the form of testable explanations and predictions about the universe. Science may be as old as the human species, and some of the earliest archeological evidence for ...

. 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 the original and largest teaching hospital of Harvard Medical School located in the West End neighborhood of Boston, Massachusetts. It is the third oldest general hospital in the United Stat ...

, which culminated in clinical trials. 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 chapters, and works with ALS ...

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.{{Cite web , last=Beltran , first=Luisa , title=The Year's First Three IPOs All Struggled. This Is Why. , url=https://www.barrons.com/articles/2022-ipos-fall-weak-51641580444 , access-date=2022-11-16 , website=www.barrons.com , language=en-US The company's shares began trading on the

Nasdaq The Nasdaq Stock Market () (National Association of Securities Dealers Automated Quotations Stock Market) is an American stock exchange based in New York City. It is the most active stock trading venue in the US by volume, and ranked second ...

on January 7, 2021 under the symbol AMLX.

References

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