VIDEO: Retained physical function in patients with ALS taking AMX0035 spurs ‘excitement’

In this video perspective, Sabrina Paganoni, MD, PhD, discusses her presentation of results from the CENTAUR trial in ALS at the American Academy of Neurology annual meeting, which is being held virtually.

The CENTAUR trial evaluated the safety and efficacy of AMX0035, “a new investigational product for ALS.” The “excitement” surrounding the results stems from the fact that patients who took AMX0035 retained physical functions longer, including speaking, swallowing and walking, according to Paganoni, codirector of the Neurological Clinical Research Institute in the department of neurology at Massachusetts General Hospital, physician scientist at the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital and Spaulding Rehabilitation Hospital, and assistant professor of physical medicine and rehabilitation at Harvard Medical School.

Long-term follow-up in the CENTAUR trial demonstrated a 44% reduced risk for death among patients taking AMX0035 compared with placebo, Paganoni told Healio Neurology. The trial showed AMX0035 to be safe and tolerable as well as “very easy” to administer via mouth or feeding tube, with no requirement for lab or ECG monitoring, Paganoni continued.

The next steps for AMX0035 involve “discussions with regulatory agencies,” according to Paganoni.

“If approved, I expect AMX0035 to be widely used for the treatment of ALS,” she said. “This is such a hopeful and exciting time in ALS research.”