Amylyx Scores $135 Million for ALS Drug with Hopes for Quick Approval


Born from a simple question asked in a Brown University dorm room eight years ago -“why do neurons die” – Amylyx Pharmaceuticals has been laser-focused on preventing degradation in neurodegenerative diseases by preserving neurons.  

Today the Boston-based company founded by those college classmates announced an infusion of $135 million in Series C funds to support the late-stage development of its lead candidate, AMX0035, for amyotrophic lateral sclerosis (ALS). 

The oral therapy is a combination of two neuroprotective molecules – sodium phenylbutyrate and taurursodiol. Both molecules are already used in the clinic safely, a factor that usually leads to some ease in establishing safety. Together they block stress signals within mitochondria and the endoplasmic reticulum to prevent cell death. 

In September, AMX0035 proved its merit for efficacy. A Phase II/III trial showed a slower rate of decline in muscle strength and lung function, in addition to fewer hospitalizations compared with those getting a placebo. All 137 adults in the trial showed rapidly progressing disease, a criterion for trial enrollment designed to garner the strongest results possible. 

Armed with the promising results of this trial, Amylyx is already seeking approval in Canada.  

“The submission of the NDS in Canada represents a significant milestone in our efforts to develop a new treatment option for people living with ALS who have no time to wait,” said Joshua Cohen, Amylyx co-CEO. 

While AMX0035 was granted Orphan Drug Designation from the FDA for ALS in 2017, things aren’t moving along as rapidly in the states as they are in the north. After meetings with the FDA, Amylyx is initiating a Phase III trial in the third quarter of this year, rather than submitting an NDA. It would appear that the FDA is requiring another trial before approving the treatment. 

The drug agency was blasted after approving Biogen’s Aduhelm for Alzheimer’s for moving too quickly and approving the drug without clear proof of impact on the disease progression. Now they’re facing heavy criticism for moving too slowly for ALS patients.  

Igor Golovniov/SOPA Images/LightRocket via Getty Images

While the number of those impacted with ALS pales in comparison to Alzheimer’s – 20,000 versus 6 million –  ALS is fatal and can progress very rapidly. The average life expectancy after diagnosis is a paltry two to five years. The disease has no biomarkers for disease identification and lacks the funding and resources provided to Alzheimer’s. 

ALS Association President Calaneet Balas asked in an article for Stat News, “Why won’t the FDA approve AMX0035 based on clinical benefit when it approved Aduhelm based on biomarker data?” 

The ALS Association is begging the FDA to see the unmet therapeutic need in the ALS community and respond with the regulatory flexibility seen in Aduhelm’s approval for Alzheimer’s. In response, FDA reaffirmed its commitment to the 2019 ALS clinical trial guidance, recognizing the need and promising flexibility but without details to actual plans. 

“The ALS community deserves to know exactly what the FDA is doing to help expedite approval of promising treatments,” a blog post by the ALS Association said.  

Pointing to the facts that Amylyx’s AMX0035 met both its main efficacy and safety goals while significantly slowing patients functional decline, the Association finds it frustrating that “it could be several years before Americans with ALS are able to access AMX0035, if at all.”  

Groups like this will continue to pressure the FDA publicly to speed therapy development for ALS patients and families.  


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