> ## Content Index
> Fetch the complete content index at: https://www.fdaweb.com/llms.txt
> Use this file to discover other available public pages before exploring further.

# Amylyx Pharma Reports Promising ALS Trial Results
- URL: https://www.fdaweb.com/amylyx-pharma-reports-promising-als-trial-results/
- Published: 2020-09-03T12:00:00.000Z
- Updated: 2026-09-14T16:27:39.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5147580

Amylyx Pharmaceuticals announces clinical trial results published in the [*New England Journal of Medicine (NEJM)*](https://cts.businesswire.com/ct/CT?id=smartlink&url=https%3A%2F%2Fwww.nejm.org%2Fdoi%2Ffull%2F10.1056%2FNEJMoa1916945&esheet=52277026&newsitemid=20200902005945&lan=en-US&anchor=New+England+Journal+of+Medicine+%28NEJM%29&index=1&md5=b62eb70332e83af57a140b66689e59d2&ref=fdaweb.com) for amyotrophic lateral sclerosis (ALS) investigational drug AMX0035 showed a “clinically meaningful and statistically significant treatment benefit on the Revised ALS Functional Rating Scale (ALSFRS-R), the most commonly used scale in clinics worldwide to measure function in ALS.” The CENTAUR trial of 137 individuals with ALS demonstrated that treatment with AMX0035 was well tolerated and decreased the rate of decline in the ALSFRS-R compared to placebo in people with ALS, the company says.

In the CENTAUR trial, Amylyx says that after 24 weeks, patients treated with AMX0035 scored on average 2.32 points higher on the rating scale than the placebo group/using the study’s primary prespecified analysis. The scale is a 48-point questionnaire measuring daily functions such as the ability to walk, dress independently, self-feed, speak and breathe. “Just a 1-2 point change in the ALSFRS-R score can indicate a significant reduction in a patient’s ability to function independently,” it says.

AMX0035 is an investigational neuroprotective therapy designed to reduce neuronal death and dysfunction, Amylyx says. “AMX0035 targets endoplasmic reticulum and mitochondrial dependent neuronal degeneration pathways in ALS and other neurodegenerative diseases,” it says.