Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases, today announced the publication of results from the pivotal CENTAUR trial evaluating AMX0035 – an investigational neuroprotective therapy designed to reduce the death and dysfunction of motor neurons – in people with ALS in the New England Journal of Medicine (NEJM).
“CENTAUR met its prespecified primary outcome, showing 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,” said Sabrina Paganoni, M.D., Ph.D., principal investigator of the CENTAUR trial, investigator at the Sean M. Healey & AMG Center for ALS at Mass General and Assistant Professor of PM&R at Harvard Medical School and Spaulding Rehabilitation Hospital. “These results represent a major milestone for the ALS community, and I am thrilled about the promise of this therapy for people with ALS.”
The CENTAUR trial of 137 individuals with ALS was conducted across 25 top medical centers in the U.S. through the Northeast ALS (NEALS) consortium. It 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 data published today in the New England Journal of Medicine show that AMX0035 demonstrated a clinically meaningful benefit and a favorable safety profile for people living with ALS. This development is a breakthrough for the ALS community and we are working collaboratively and expeditiously with agencies worldwide to bring this potential new treatment option forward,” said Josh Cohen, Co-CEO, Chairman and Co-Founder at Amylyx.
“The data published today makes a clear and compelling case that AMX0035 should be made available to people with ALS as soon as possible,” said Calaneet Balas, President and CEO of The ALS Association. “We look forward to working with Amylyx, the FDA, and the entire community to help make that happen. We are grateful to all the Ice Bucket Challenge donors whose contributions helped make this trial possible.”
CENTAUR Results:
Primary Outcomes:
After 24 weeks, patients treated with AMX0035 scored on average 2.32 points higher on the ALSFRS-R than the placebo group (p=0.03) using the study’s primary prespecified analysis. A change from baseline analysis was also conducted and indicated that the AMX0035 group scored 2.92 points higher at the end of 24 week follow up (p=0.01).
The ALSFRS-R 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. The ALSFRS-R measures many different daily functions so point differences can manifest differently in different patients. Some examples of a two point change on this scale include the difference between an individual eating successfully with some difficulty vs needing a feeding tube, or walking with assistance versus not walking at all.
Secondary Outcomes:
In line with the primary outcome, patients on AMX0035 also showed numerical benefits on secondary outcomes including measures of muscle strength, breathing and hospitalization frequency, although the study was not powered for secondary outcomes.
Progression in lung function (percent predicted slow vital capacity) was numerically slower in patients taking AMX0035 (Least Squares Difference=5.11 points, p=0.08).
Participants in the AMX0035 group were hospitalized numerically less often (Hazard Ratio = 0.54, p=0.09).
Rate of decline in overall muscle strength (percent predicted ATLIS) was numerically slower in patients taking AMX0035 (Least Squares Difference=2.82 points, p=0.12). The effect of AMX0035 on progression was nominally statistically significant for the upper limbs measurements (Least Squares Difference=4.27 points, p=0.04).
Overall Safety:
Nearly all participants ...
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