Rare Daily Staff

Amylyx Pharmaceuticals and the Sean M. Healey & AMG Center for ALS at Mass General said that the company’s experimental ALS therapy AMX0035 demonstrated a significant treatment benefit for people with ALS in the CENTAUR study.

ALS, also known as Lou Gehrig’s disease, is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. The progressive neurodegeneration in ALS eventually leads to the death of motor neurons and loss of the ability of the brain to initiate and control muscle movement. With muscle action progressively affected, patients in the later stages of the disease may become paralyzed and unable to breathe normally.

AMX0035 is a first-in-class, investigational therapy designed to reduce neuronal death and dysfunction. AMX0035 targets mitochondrial and endoplasmic reticulum dependent neuronal degeneration pathways.

In the phase 2 study, participants taking AMX0035 had a statistically significant slowing of ALS disease progression as measured by the ALS Functional Rating Scale-Revised compared to placebo, the primary outcome of the trial. Amylyx said it will submit detailed results from CENTAUR for peer-reviewed publication and presentation at a future medical congress.

Participants in CENTAUR were given the option after the trial to enroll in an open-label extension study to receive treatment with AMX0035. Nearly 90 percent of participants who completed CENTAUR elected to enroll in the extension study. Interim data from the ongoing extension study will be presented in 2020.

In addition, the company will provide an update on regulatory plans and further details on expanded access plans in early 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 co-founder of Amylyx. “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.”

Photo: Justin Klee, president and co-founder of Amylyx

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