Amylyx Pharmaceuticals Doses First Patient in Phase 2 Trial of AMX0035 as an ALS Therapy

Amylyx Pharmaceuticals has dosed the first patient in a Phase 2 clinical trial that will assess AMX0035’s ability to treat amyotrophic lateral sclerosis.

Researchers are seeking 132 participants for the CENTAUR study (NCT03127514). A unique feature of the trial is that Amylyx designed it in collaboration with patients and caregivers as well as ALS specialists.

AMX0035 is an oral combination of sodium phenylbutyrate and tauroursodeoxycholic acid. Both have demonstrated safety and preliminary signs of effectiveness in laboratory and animal studies.

At the moment, the only therapies the U.S. Food and Drug Administration has approved for ALS are Rilutek (riluzole) and Radicava (edaravone). Rilutek slows the progression of the disease and increases patients’ survival by about 10 percent, studies indicate. Meanwhile, Radicava has been shown to slow patients’ decline.

ALS patients continue to have many unmet medical needs, however.

In preclinical-trial studies in 2016, researchers discovered that AMX0035 limited cell death and neurotoxic inflammation in several ALS models, making it a promising therapy for humans.

CENTAUR is a 24-week, randomized, double-blind, placebo-controlled trial. Its primary objective is to see if AMX0035 is safe, if it can slow the progression of the disease, and if patients can tolerate it. Researchers will measure disease progression with the revised Amyotrophic Lateral Sclerosis Functional Rating Scale, or ALSFRS-R.

The trial will also evaluate AMX0035’s ability to strengthen muscles. One of the strength measures will be ATLIS, or the Accurate Test of Limb Isometric Strength. Researchers will also look at patients’ respiratory function and biomarkers.

When the 24-week trial is over, Amylyx will give participants a chance to take part in an extension of the study.

“The CENTAUR trial builds on promising results with AMX0035 in preclinical studies and clinical experience with the drug’s two components,” Dr. Sabrina Paganoni, who will be the trial’s principal investigator, said in a press release. “The team at NCRI [the Neurological Clinical Research Institute] worked closely with Amylyx to design an innovative trial that will evaluate AMX0035’s safety and efficacy, advance our understanding of several biomarkers, and provide insights into ALS disease biology.”

“The design and launch of the CENTAUR trial reflects a close collaboration between academia, industry, ALS organizations, and patients,” said Dr. Merit Cudkowicz, the chief of neurology at Massachusetts General Hospital. “Collaborative efforts among key stakeholders are essential to developing treatments for this complex and serious disease.”

The ALS Association and ALS Finding a Cure supported CENTAUR with a $2.96 million grant. Other partners in the trial program include Massachusetts General Hospital; the Neurological Clinical Research Institute, whose researchers will help oversee the study; and the Northeast ALS Consortium.

“The study reflects the outstanding progress the Amylyx team and our collaborators made to build a strong clinical program aimed at ultimately improving the lives of patients with this devastating disease,” added Justin Klee, president and co-founder of Amylyx.