8 October 2019 - - The last participant has completed the planned 24 weeks of study treatment in CENTAUR, a clinical trial assessing the safety and efficacy of AMX0035 in people with ALS, US-based pharmaceutical company Amylyx Pharmaceuticals, Inc said.

The CENTAUR study was the recipient of the ALS ACT grant, and is supported by ALS Finding a Cure,-a program of The Leandro P. Rizzuto Foundation, The ALS Association, the Northeast ALS Consortium, Massachusetts General Hospital Neurology Clinical Research Institute, and was funded in part by the ALS Ice Bucket Challenge.

ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. The progressive neurodegeneration in ALS eventually leads to the death of motor neurons.

When the motor neurons die, the ability of the brain to initiate and control muscle movement is lost.

With muscle action progressively affected, patients in the later stages of the disease may become paralyzed and unable to breathe normally.

AMX0035 is Amylyx's proprietary two-drug combination therapy in development to prevent nerve cell death and degeneration.

CENTAUR is a 24-week, randomized, double-blind, placebo-controlled Phase II clinical trial in people with ALS.

The trial's primary objectives are to evaluate the safety and tolerability of AMX0035 and assess the drug's impact on disease progression as measured by the revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) over the 24-week study period.

The trial will also evaluate the impact of AMX0035 on isometric strength as measured by ATLIS, respiratory function, and exploratory biomarkers of neuronal death.

There is also an open label extension to the trial allowing enrolled participants to continue to receive AMX0035.

In 2017, AMX0035 was granted orphan drug status by the US Food and Drug Administration as a potential medicine for ALS. Amylyx reached its recruitment goal for the CENTAUR study in March 2019.

AMX0035 is a proprietary combination of two small molecules, sodium phenylbutyrate and tauroursodeoxycholic acid (TUDCA).

Each compound has demonstrated strong efficacy in several cellular and animal models of ALS.

When individually tested in ALS clinical trials, PB and TUDCA have both shown safety, tolerability, and preliminary signs of efficacy.

In preclinical trials, Amylyx demonstrated a synergistic effect between the two compounds in preventing nerve cell death and degeneration.

Amylyx Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company developing a novel therapeutic for Amyotrophic Lateral Sclerosis, Alzheimer's disease and other neurodegenerative diseases.