Amylyx Nabs $5M to Take Aim at Nerve Cell Death in ALS Patients

Xconomy Boston — 

While the cause of amyotrophic lateral sclerosis, or ALS, remains a mystery, many researchers are trying to develop a cure by stopping its effect—the death of nerve cells that leads to muscle deterioration and the patient’s eventual death.

One Cambridge, MA-based drug developer, Amylyx Pharmaceuticals, has received a $5 million Series A financing to take its nerve cell-targeting treatment, AMX0035, into a Phase 2 trial for ALS patients. The treatment is a combination of sodium phenylbutyrate, a drug commonly used to remove ammonia from the body, and tauroursodeoxycholic acid, a derivative of stomach bile acid.

With the two drugs combined into one, the company believes it can possibly block nerve cell death and neurotoxic inflammation, two potential causes of ALS. The company expects the trial to begin in late 2016 or early 2017.

The funding was provided by Morningside Venture, the ALS Investment Fund, and former Genzyme CEO Henri Termeer. Since selling Genzyme to Sanofi for $20 billion in 2011, Termeer has been an active investor and advisor to startup biotechs, including Moderna Therapeutics, Lysosomal Therapeutics, Aura Biosciences, and X4 Pharmaceuticals.

The $5 million funding follows a $3 million grant for the Phase 2 trial that Amylyx received from the ALS Accelerated Therapeutics Initiative, which is a collaboration between the ALS Association and ALS Finding a Cure. Amylyx says it has raised $10 million for its drug, called AMYX0035.

Amylyx believes its combination drug can provide the equivalent of stress relief in nerve cells, helping them stabilize their energy production and their ability to weed out misproduced proteins. In ALS, those processes are thrown out of whack, leading to inflammation or cellular death, according to Amylyx.

Some 30,000 or so patients have the disease in the U.S. at any given time. The muscles of ALS patients slowly stop functioning. Many eventually die in their sleep after the muscles that help them breathe stop functioning—if they haven’t opted for a ventilator or other respiratory aid. Other drugs, such as Cytokinetics’ tirasemtiv (NASDAQ: CYTK), aim to improve the breathing of ALS patients. Cytokinetics recently signed a licensing deal with Toyko-based Astellas.

Isaac Chang from Morningside, a private equity group founded in 1986 by a family in Hong Kong, is joining the company’s board, as is Felix von Coerper of the ALS Investment Fund. Termeer will be a board observer.