AMX0035 is a drug designed to prevent nerve cell death and degeneration. It seems to improve cellular metabolism and promote protein folding in cellular and animal models of neurodegenerative disease. When metabolism and protein folding are not functioning properly, neurons ultimately die.

In ALS, Alzheimer’s Disease and other neurological diseases metabolism and protein folding seem to be dysfunctional. AMX0035 helps boost the metabolism and synthesize heat shock proteins that help to fold other proteins into their proper shapes. Through these mechanisms, AMX0035 prevents neuronal degeneration and death.

AMX0035 in Clinical Trials

AMX0035 is in late Phase 2 trial for ALS, it reached Phase 2 for Alzheimer’s Disease and is in preclinical testing for Wolfram Syndrome.

In the NEWS

Today, December 17, 2019, Amylyx Pharmaceuticals and the Sean M. Healey & AMG Center for ALS at Mass General announced that AMX0035 demonstrated a significant treatment benefit for people with ALS in the CENTAUR study. The participants in the trial who are on AMX0035 had a statistically significant slowing of ALS disease progression as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) compared to placebo, which is the primary outcome of the trial.

Detailed results from CENTAUR will be submitted for peer-reviewed publication and presentation at a future medical congress.

Quotes from the Experts

“Today marks a significant step forward in the fight to develop new treatments for ALS,” said Dr. Sabrina Paganoni, M.D., Ph.D., principal investigator of the CENTAUR study, an investigator at the Healey Center for ALS at Mass General and Assistant Professor of PM&R at Harvard Medical School and Spaulding Rehabilitation Hospital said, “The study results highlight AMX0035 as a potentially beneficial new treatment for people with ALS, and the design and execution of the CENTAUR trial are a testament to true collaboration across the many stakeholders in this fight. Thank you to everyone who made this possible, and I look forward to presenting the full study results in the coming months.”

Justin Klee, President, and Co-Founder of Amylyx stated that with these results, Amylyx now has a responsibility to move ahead as efficiently as possible, as people living with ALS don’t have time to wait. We will work closely with the FDA and the ALS community, including the wonderful Northeast ALS Consortium leadership and member sites that conducted the CENTAUR trial, to decide the next steps and will continue to keep everyone informed.”

Participants in CENTAUR were given the option, after the trial, to enroll in an open-label extension study to receive treatment with AMX0035. Nearly 90% of participants, who completed CENTAUR, elected to enroll in the extension study. Interim data from the ongoing extension study will be presented in 2020.

In addition, the company will provide an update on regulatory plans and further details on expanded access plans in early 2020.

Dr. Merit Cudkowicz, Chief Medical Officer from ALS Finding a Cure®, Director of the Healey Center for ALS, Chief of Neurology at Mass General, and the Julieanne Dorn Professor of Neurology at Harvard Medical School commented 0n the results, saying, “ALS Finding a Cure is proud to have catalyzed and supported the CENTAUR study, and I am encouraged by what the results mean for people living with ALS. Our team at the Mass General Neurological Clinical Research Institute is proud of this collaboration with Amylyx and our colleagues in the Northeast ALS Consortium on this important study. We look forward to advancing this research and what it might mean for those living with ALS.”

Dr. Rudolph Tanzi, Ph.D., Kennedy Professor of Neurology, Massachusetts General Hospital, Chair of the Cure Alzheimer’s Fund Research Leadership Group and chair of the Amylyx SAB, shared, “The positive results from the CENTAUR ALS study demonstrate that the novel mechanism of AMX0035 may represent a new treatment approach not only for ALS but for Alzheimer’s disease. I am very excited about the demonstrated benefit of AMX0035 in people with ALS, and I look forward to the results from the ongoing PEGASUS trial for people with Alzheimer’s disease.”

ALS, which is also known as Lou Gehrig’s disease, 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 and loss of the ability of the brain to initiate and control muscle movement. With muscle action progressively affected, patients in the later stages of the disease may become paralyzed and unable to breathe normally. 

Prohost Observations

This could be the best news the medical community, the specialists and the ALS patients have ever heard with regard to drugs with positive results on patients with ALS. We believe that the significant treatment benefit for people with ALS in the CENTAUR study marks the beginning of the walk on the road toward solving the puzzles of some deadly diseases that have not yet found treatments.

This is more than impressive news. It is what eliminates the obstacles from the road toward recognizing the true pathways of the chronic progressive and deadly diseases that cause the utmost miseries to humans in all age groups.

We will not abandon following up on Amylyx Pharmaceuticals.