Amylyx Pharmaceuticals
| Company type | Public |
|---|---|
| Industry | Biopharmaceutical |
| Founded | 2013 |
| Founder | Joshua Cohen, Justin Klee |
| Headquarters | Cambridge , United States |
Key people | Rudolph E. Tanzi (scientific advisory chair) |
| Products | AMX0035 (Albrioza, Relyvrio) |
Amylyx Pharmaceuticals, Inc is a biopharmaceutical firm headquartered in Cambridge, Massachusetts. The company is recognized for developing AMX0035, an investigational treatment targeting amyotrophic lateral sclerosis (ALS). AMX0035 received approval in Canada under the name Albrioza in June 2022 and later in the United States as Relyvrio in September 2022; however, it was withdrawn from the market in April 2024.
History
Established in 2013 by Joshua Cohen and Justin Klee during their undergraduate studies at Brown University, Amylyx created a therapeutic combination of taurursodiol and sodium phenylbutyrate designed to safeguard neurons by mitigating dysfunction in mitochondria and the endoplasmic reticulum. Their scientific advisory was led by Rudolph E. Tanzi, who also served as the founding chair of the company’s research board. After encouraging outcomes in preclinical testing, Tanzi facilitated connections with researchers at Massachusetts General Hospital, which enabled subsequent clinical trials and eventual FDA approval. The ALS Association contributed $2.2 million to Amylyx’s development efforts, supported by funds raised through the Ice Bucket Challenge.
Amylyx became a publicly traded company in January 2022, with its stock listed on the Nasdaq under the ticker AMLX.
Following a Phase 3 clinical trial of AMX0035 that did not demonstrate statistically significant efficacy compared to placebo, Amylyx declared on April 4, 2024, its intention to withdraw both Albrioza and Relyvrio from the North American markets. The company received commendations for adhering to its pre-approval promise to cease sales if the treatment proved ineffective—a notable ethical decision enabled by its independence from large pharmaceutical corporations.