Sabrina Paganoni, MD, PhD: AMX0035 and the Future of ALS Care


The co-director of the Neurological Clinical Research Institute at Massachusetts General Hospital offered her perspective on the expected developments for ALS care in next few years, and where AMX0035 might fit in that landscape.

“For us, as investigators, but also as clinicians—I follow patients in the clinic and meet their families—it’s incredibly exciting to think that we are contributing to a project that could actually substantially impact people’s lives.”

For clinicians like Sabrina Paganoni, MD, PhD, who participate in clinical studies, there’s a rewarding part of the work when an effective therapy gets approval. Particularly so when the disease being treated is a fatal one, such as amyotrophic lateral sclerosis (ALS). Paganoni, who is the co-director of the Neurological Clinical Research Institute at Massachusetts General Hospital, has been involved in the development of an agent that has shown some promise in ALS: Amylyx Pharmaceuticals’ AMX0035.

The drug is a combination of sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA), and recent data from the phase 2 CENTAUR trial (NCT03127514) showed that it resulted in a statistically significant (P <.05) slowing of disease progression in patients with ALS.1 The full results of the study are expected to publish before the end of the year, but in the meantime, Paganoni offered her perspective to NeurologyLive.

She spoke to what might be the next steps for AMX0035 on its road toward the FDA and what her experience as part of the CENTAUR trial was like. She also offered some perspective on what she’s expecting and hoping to happen in ALS care over the next few years.


Amylyx Pharmaceuticals announces AMX0035 demonstrated statistically significant treatment benefit for people with ALS in the CENTAUR trial [news release]. Cambridge, MA. Amylyx Pharmaceuticals. December 17, 2019. Accessed August 19, 2020.

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