Sabrina Paganoni, MD, PhD: AMX0035 and the Current ALS Treatment Landscape


The co-director of the Neurological Clinical Research Institute at Massachusetts General Hospital discussed how the investigational agent might fit into the landscape of amyotrophic lateral sclerosis treatment as it progresses through the pipeline.

“Two treatments have been approved by the FDA, however, even with those 2 drugs, ALS remains an aggressive disease that’s still fatal.”

Amyotrophic lateral sclerosis (ALS) still lacks a cure. Despite this presence as a fatal disease, the future is looking bright with 2 approved treatments and a pipeline of increasing activity. For physicians treating the disease, such as Sabrina Paganoni, MD, PhD, it’s an exciting time to be involved.

Paganoni, who is the co-director of the Neurological Clinical Research Institute at Massachusetts General Hospital, is particularly familiar with an investigational agent for ALS developed by Amylyx Pharmaceuticals that’s a combination drug consisting of sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA), called AMX0035. It is designed to simultaneously target 2 pathways: endoplasmic reticulum stress and mitochondrial dysfunction.

In this interview with NeurologyLive, Paganoni describes her perspective on the landscape in ALS, and her experience with the agent as the principal investigator of the phase 2 trial assessing it, CENTAUR (NCT03127514). The results of the CENTAUR trial are expected to publish before the end of the year.

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