By Jacob Ahlstrom | Posted - Sep 29th, 2020





Phase II ALS Clinical Trial Produces Promising Results for AMX0035

The Pharmaceutical company AMYLYX recently released promising results for the ALS drug AMX0035 after Phase II clinical trials were completed. The results display a significant slowing of disease progression in patients with ALS (Amyotrophic Lateral Sclerosis). 

On Friday, September 4th, AMYLYX physicians presented the results during an online seminar conference explaining the positive outcome of the AMX0035 phase II clinical trial.  

What is AMX0035? 

AMY0035 has come a long way to get to this point in clinical trials. The drug was initiated by two college students who had the idea of targeting two different clinical pathways that are both essential in ALS cell biology. These pathways are called the mitochondria and the endoplasmic reticulum (ER). The mitochondria are often referred to as the powerhouse of the cell. It is where all the cell energy is created. The ER is like the delivery system for the cell. When new proteins are made and need to be sent to different parts of the cell, they travel through an intricate tubing system within the cell called the ER. When these systems stops working the cell starts to shut down. Both are found dysfunctional in cells with ALS.  

The idea of making a drug that could repair these two systems soon became a reality when they ran test that proved AMX0035 could reverse cell death in cell models. The drug then went on to clinical trial testing and is now done with Phase II testing involving active patients.  

For the last 10 years researchers at AMYLYX have been working on preparing AMX0035 for this day. In 2019 they started Phase II, making the patients their number one priority. They encouraged patients to use existing medications like riluzole and edaravone during their testing. This has not been done in previous clinical trials but hopefully this can become the new norm for ALS clinical trials. The research team also allowed the patients who finished clinical trial testing to continue the treatment for extended testing to provide prolonged treatment to the patients and to assess the long-term impact of the medication.  

Clinical Trials 

The Phase II clinical trial is a double blind 2 to 1 placebo-controlled trial. This means that for every 2 people with the actual drug 1 person is given a placebo or fake drug they are told is real as a control. This trial’s number one focus is to determine the safety and tolerability of AMX0035 with the purpose of measuring the impact of AMX0035 on physical function in patients with ALS.  

To measure drug safety, the researchers keep detailed records of any adverse events reported by patient, lab test and other assessments during study visits. As for the physical functions, the researchers recorded disease progression using the ALSFRS-R (the Revised ALS functional rating scale) which is the most commonly used rating scale for ALS.  


Scoring for the ALSFRS-R is broken down into 12 different categories. 

  • A score is assigned for each of the 12 activities 

  • Each activity is scored on a scale from 0 (function absent) to 4 (function normal) 

  • The largest score is 48 points 

The Promising Results 

In this trial named the CENTAUR trial 137 participants were randomly divided into a two-to-one ratio where 67% were given AMX0035 and the other 33% percent were given a placebo. The trial lasted for 6 moths. During this time the participants treated with AMX0035 had better scores on the ALSFRS-R compared to the placebo group.  

AMX0035 Results

  AMX0035 Patients Placebo Patients
Adverse Events During the Study  97%  96%
Dose Reduction due to Gastrointestinal Events   3%  0%
Dose Interruption due to Gastrointestinal Events   9%  2%
Serious Adverse Events During the Study  12%  19%
Respiratory Serious Adverse Events   3%  8% 


“The participants treated with AMX0035 demonstrated a significant slowing of ALS disease progression as measured by the ALSFRS-R. This is a milestone in our fight against ALS,”

Sabrina Paganoni, MD, PhD, principal investigator of the CENTAUR study. 


Safety Results

  AMX0035 Placebo Actual v.s Control
 ALSFRS-R Score Decline   1.24 points/month  1.66 points per month  0.42 points per month difference
 Retained Function  6-weeks  0-weeks  6-week increase in retained function


The main side effects of AMXZ0035 were gastrointestinal events such as nausea, diarrhea, and abdominal pain. These were primarily reported during the first 3 weeks. Serious adverse events were much higher in the placebo groups with 5 % more placebo patients discontinuing the trial due to respiratory failure. 

These results provide evidence that AMX0035 can help patients retain their physical function for longer compared to patients actively taking endaravone and riluzole. This is an incredible feat given the debilitating nature of ALS. This is the first time that an ALS therapy has effectively accomplished anything more than the reduction of symptoms. It has opened new doors in preventing nerve cell death and offers new hope to people with ALS and their Families. 

“Today’s news builds upon the progress we have made in ALS research,” said Cohen. “This experimental medicine has demonstrated that it can help patients retain their physical function, which is an incredible feat given the debilitating nature of this disease. It is our hope that AMX0035 will one day be available for patients and we are committed to making that a reality.”

"Patients and their families do not have time to wait," said Klee. "People with ALS progressively lose their ability to function and care for themselves, so we want to do everything we can to help them slow down this devastating disease. We will be working with the FDA to determine next steps and the path for patients to gain access to AMX0035. We’ll continue to share our plans with the community as they develop."

-Amylyx co-founders and co-CEOs Joshua Cohen and Justin Klee



Jacob Ahlstrom
About the Author

Jacob Ahlstrom - Jacob is a Neuroscience undergraduate at Brigham Young University. Jacob's interest in researching and writing about ALS is fueled by his hope to make the process easier for everyone else. Over the last year he has worked alongside Seth Christensen to find ways to educate and connect ALS patients.