ALS Treatment AMX0035 Slows Disease Progression


Top-line results from the phase 2 CENTAUR trial showed a statistically significant slowing of ALS disease progression in patients treated with AMX0035.

Sabrina Paganoni, MD, PhD

Sabrina Paganoni, MD, PhD

Amylyx Pharmaceuticals has announced that treatment with its investigational agent AMX0035 (Elybrio) resulted in a statistically significant (P <.05) slowing of disease progression in patients with amyotrophic lateral sclerosis (ALS) compared with placebo.

The results were based on change from baseline of assessments on the ALS Functional Rating Scale-Revised (ALSFRS-R), the primary end point of the CENTAUR clinical trial (NCT03127514).

“Today marks a significant step forward in the fight to develop new treatments for ALS,” said Sabrina Paganoni, MD, PhD, principal investigator of the CENTAUR study, investigator at the Healey Center for ALS at Mass General, and assistant professor of PM&R at Harvard Medical School and Spaulding Rehabilitation Hospital, in a statement.

The study “has been a great example of ALS community collaboration with partnerships between Amylyx Pharmaceuticals and academia, and specifically the NEALS Consortium, The Neurological Clinical Research Institute at MGH, and the Barrow Neurological Institute,” Paganoni told NeurologyLive.

The trial included 135 participants aged 18 to 80 who were treated for 24 weeks with either AMX0035 twice daily or placebo in a 2:1 randomization. Notably, the oral suspension can also be administered via PEG tube. In addition to change in slope of ALSFRS-R over 24 weeks, the trial also assessed incidence of adverse events, as well as the proportion of participants who were able to remain on the study drug until planned discontinuation. Secondary outcomes included measures of blood-based and imaging biomarkers, change in isometric muscle strength, change in slow vital capacity decline, as well as tracheostomy, hospitalizations, and overall survival.

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“We designed the inclusion/exclusion criteria to target a relatively homogenous, fast-progressing patient population. That was possible because the study design was modeled after PRO-ACT, which is the largest publicly available clinical trial database that includes data from over 10,000 ALS patients who participated in previous clinical trials,” Paganoni told NeurologyLive. “By looking at the rate of progression of patients in this trial and also in separate databases, we were able to design inclusion/exclusion criteria to enroll a population of fast progressors because we knew that would increase our statistical power to see a treatment effect. These patients are not biologically different from other people with ALS, but this was a statistical strategy to increase our power to see a treatment effect with a relatively smaller trial.”

Notably, nearly 90% of the trial participants who completed the 24-week study went on to enroll in the ongoing open-label extension, which will readout data in 2020.

“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,” said Justin Klee, president and cofounder of Amylyx, in a statement. “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 next steps and will continue to keep everyone informed.”

The company plans to provide updates on regulatory plans, as well as information on expanded access, in early 2020.


Amylyx Pharmaceuticals announces AMX0035 demonstrated statistically significant treatment benefit for people with ALS in the CENTAUR trial [news release]. Cambridge, MA: Amylyx Pharmaceuticals. December 17, 2019. Accessed December 17, 2019.

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