Amylyx Completes Patient Recruitment for Phase 2 Study Testing AMX0035 for ALS

Ana Pena, PhD avatar

by Ana Pena, PhD |

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Amylyx has completed patient recruitment for the Phase 2 clinical trial CENTAUR, which addresses the safety and effectiveness of AMX0035 as an oral treatment for amyotrophic lateral sclerosis (ALS).

In 2017, Amylyx launched the CENTAUR study (NCT03127514) at several centers across the U.S. with plans to enroll 132 individuals diagnosed with sporadic or familial ALSNow, the required number of patients has been reached, completing the recruitment phase.

“Given the urgency for patients with ALS, we’re encouraged to reach this milestone,” Justin Klee, president and co-founder of Amylyx told ALS News Today.

AMX0035 is an oral combination therapy made of two small molecules, tauroursodeoxycholic acid (TUDCA) and sodium phenylbutyrate (PB).

The medication was designed to reduce nerve cell death by blocking key cellular pathways implied in cellular stress and death.

In preliminary studies, AMX0035 reduced those signals at two important cell compartments, the mitochondria and the endoplasmic reticulum, both of which are strongly implicated in the death of nerve cells.

Preclinical testing in animal models of ALS, Alzheimer’s disease, and mitochondrial diseases showed that AMX0035 can effectively inhibit nerve cell death and neurotoxic inflammation.

Each of the agents that make up AMX0035, PB or TUDCA, have demonstrated strong effectiveness in several preclinical models of ALS.

But more recent studies suggest that combining both can improve effectiveness compared with single therapy.

In addition, TUDCA or PB alone have given signs of being safe and well-tolerated in clinical studies with ALS patients.

In 2017, AMX0035 was granted orphan drug status by the U.S. Food and Drug Administration (FDA) as a potential medicine for ALS.

In the CENTAUR trial, patients are randomized to receive either twice-daily oral doses of AMX0035 or a placebo for 24 weeks.

The trial’s primary goals are to see if therapy with AMX0035 is safe and well-tolerated and able to delay the loss of capacities during the treatment period.

The later will be measured by the ALSFRS-R scale, a doctor-reported instrument for monitoring disability progression in ALS.

Other efficacy measures will be the treatment’s impact in muscle strength, respiratory function, and biomarkers of ALS, including markers of neuronal death and neuroinflammation.

At the end of the 24-week trial period, patients who wish may move to an open-label extension study and continue to receive treatment.

The study is a collaboration between Amylyx, ALS Finding a Cure Foundation, ALS Association, Northeast ALS Consortium, Massachusetts General Hospital Neurology Clinical Research Institute and the Leandro P. Rizzuto Foundation.

“This collaborative effort to assess the impact of AMX0035 on disease progression involves a committed team of individuals who share a strong personal connection to ALS,” Sabrina Paganoni, MD, PhD, from Healey Center for ALS at Massachusetts General Hospital and the study’s principal investigator said.

“We thank the CENTAUR study participants and their families, site investigators and staff who are devoted to pushing ahead with us on this mission. It’s exciting to be fully underway as we continue to study AMX0035”, she added.

“Thousands of people suffer from this disease, so we’re working as quickly as possible to evaluate AMX0035 as a treatment for ALS,”  said Joshua Cohen, Amylyx’s CEO and co-founder.