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Amylyx Pharmaceuticals preps NDA filing for ALS drug AMX0035  

A Phase 3 trial focused on the drug is in the works.

By Brian Buntz | September 15, 2021

AmylyxCambridge, Massachusetts–based Amylyx Pharmaceuticals intends to submit a New Drug Application (NDA) to FDA for AMX0035 (sodium phenylbutyrate and taurursodiol) to treat amyotrophic lateral sclerosis (ALS), which is also known as Lou Gehrig’s disease. 

The company decided to file after a series of meetings with the FDA, including a pre-NDA meeting on July 15.  

AMX0035 was the subject of the Phase 2 CENTAUR trial, which involved 137 participants with ALS. In that trial, the drug met its primary efficacy endpoint of slowing ALS based on the ALS Functional Rating Scale-Revised (ALSFRS-R). 

A forthcoming Phase 3 trial will evaluate the safety and efficacy of the drug over 48 weeks. 

Before reversing course, FDA had asked Amylyx to submit positive Phase 3 trial results before filing a New Drug Application.

To date, there are limited treatment options available for ALS. A 2020 paper in Neuropharmacology. 

Concluded that the two drugs with demonstrated efficacy in treating ALS, Riluzole (Rilutek) and edaravone (Radicava), only “show only modest effects on disease progression.”

“AMX0035 has shown potential to provide those living with ALS and their families hope for the future,” said Dr. Sabrina Paganoni, principal investigator of the CENTAUR trial, in a statement. 


Filed Under: Neurological Disease
Tagged With: ALS, AMX0035, Amylyx, Amylyx Pharmaceuticals, amyotrophic lateral sclerosis, Lou Gehrig’s disease
 

About The Author

Brian Buntz

As the pharma and biotech editor at WTWH Media, Brian has almost two decades of experience in B2B media, with a focus on healthcare and technology. While he has long maintained a keen interest in AI, more recently Brian has made making data analysis a central focus, and is exploring tools ranging from NLP and clustering to predictive analytics.

Throughout his 18-year tenure, Brian has covered an array of life science topics, including clinical trials, medical devices, and drug discovery and development. Prior to WTWH, he held the title of content director at Informa, where he focused on topics such as connected devices, cybersecurity, AI and Industry 4.0. A dedicated decade at UBM saw Brian providing in-depth coverage of the medical device sector. Engage with Brian on LinkedIn or drop him an email at bbuntz@wtwhmedia.com.

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