Source/Disclosures
Disclosures: Paganoni reports receiving grants from the ALS Association, the ALS Finding a Cure initiative and Amylyx Pharmaceuticals during the conduct of the study and grants from Biohaven, Clene Nanomedicine, Prilenia, Ra Pharma and Revalesio Corporation outside the submitted work. Please see the study for all other authors’ relevant financial disclosures.
October 16, 2020
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AMX0035 reduces risk for death by nearly half in patients with ALS

Source/Disclosures
Disclosures: Paganoni reports receiving grants from the ALS Association, the ALS Finding a Cure initiative and Amylyx Pharmaceuticals during the conduct of the study and grants from Biohaven, Clene Nanomedicine, Prilenia, Ra Pharma and Revalesio Corporation outside the submitted work. Please see the study for all other authors’ relevant financial disclosures.
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AMX0035, an investigational neuroprotective therapy, reduced the risk for death by nearly half in patients with amyotrophic lateral sclerosis according to results of an overall survival analysis from the CENTAUR trial.

The survival benefit of AMX0035 occurred regardless of baseline use of riluzole or edaravone, either as single agents or in combination.

Brain MRI
Sabrina Paganoni

“We have many reasons to be encouraged today,” Sabrina Paganoni, MD, PhD, principal investigator of the CENTAUR trial, investigator at the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital and assistant professor at Harvard Medical School and Spaulding Rehabilitation Hospital, said in a press release. “In this trial, we have seen promising functional and survival benefits. ... I am so excited about the potential of this drug for the people living with ALS.”

The 24-week, randomized, double-blind, placebo-controlled phase 2/3 CENTAUR trial examined the safety and tolerability of AMX0035 and its effect on disease progression according to the ALS Functional Rating Scale-Revised. The trial also analyzed the drug’s impact on other ALS disease markers, including muscle strength, lung vital capacity and biomarkers of neuronal degeneration.

Survival analyses compared time to death (all-cause mortality) between participants originally assigned to treatment with AMX0035 with those originally assigned to the placebo group. The longest follow-up duration was 35 months.

The researchers observed a 44% reduced risk for death (HR = 0.56; 95% CI, 0.34-0.92) over the course of follow-up in patients originally assigned to AMX0035 compared with patients originally assigned to placebo. Patients originally assigned to active treatment with AMX0035 survived a median duration of 25 months (95% CI, 19-33.6 months) compared with patients originally assigned to placebo, who survived a median of 18.5 months (95% CI, 13.5-23.2 months). This represented a 6.5-month difference in survival, according to results of the study on AMX0035 published in Muscle & Nerve.

The active treatment and placebo groups experienced similar rates of death-equivalent events, according to the results published in Muscle & Nerve. Six participants originally assigned to AMX0035 (6.7%) and four participants originally assigned to placebo (8.3%) experienced these types of events.

Previous results from the CENTAUR study published in The New England Journal of Medicine demonstrated that AMX0035 also reduced the rate of functional decline compared with placebo in patients with ALS.

These results supported the “functional and long-term survival benefits” of AMX0035, Merit Cudkowicz, MD, director of the Sean M. Healey & AMG Center for ALS and chief of the neurology department at Massachusetts General Hospital, chief medical officer of ALS Finding a Cure and Julieanne Dorn professor of neurology at Harvard Medical School, said in the press release. “With these results, we have shown that AMX0035 may provide patients hope and the chance to function better and live longer lives.”

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