- AMX0035 is a combination of two small molecule drugs: Phenylbutyrate (PB) and Tauroursodeoxycholic Acid (TUDCA).
- The Centaur Trial (NCT03127514) looked at the functional impact of AMX0035 on the ALSFRS-R score of people living with ALS vs. a placebo. The total trial enrollment was 137.
- The statistically significant Centaur Trial results are documented here. In short, the drug causes significant slowing of ALSFRS-R decline.
- The open label extension of the Centaur Trial was utilized to observe the impact of AMX0035 on the long-term survival of people living with ALS.
- The readout contained 35 months of data (beginning from initial trial enrollment period in 2017/2018).
- The readout came from a comparison of trial participants who took AMX0035 vs. those who were administered a placebo.
- The following results were disclosed by the principal investigator of the Centaur Trial, Dr. Sabrina Paganoni, during the webinar:
- There was a 6.5 month longer median survival in the group originally randomized to AMX0035 than the placebo group. The median survival durations were 25 months and 18.5 months respectively.
- Please read the Oct 16th Amylyx Pharmaceuticals press release of their long-term survival data findings here.
Both the placebo and non placebo groups had the option to take riluzole and/or endaravone. That being said, there was a sensitivity analysis done in both the function and survival studies. The sensitivity analysis concluded that AMX0035 had affects on top of the standards of care aka riluzole or endaravone.
Q & A
Q: Could this work for variants of ALS, such as primary lateral sclerosis (PLS)? A: Given the MOA of AMX0035, it is reasonable to think that it may be effective. However, no PLS patients were enrolled in the Centaur Trial.
Q: What is the route of administration? A: The formulation used in the Centaur Trial was a powder form of both drugs that you mix with water.
Q: Will it be affordable? A: Not determined at this time.
Q: Will AMX0035 be available through Expanded Access? A: Not determined at this time.
Q: Were there notable side effects? A: Mild nausea and diarrhea was common and expected in first 3 weeks of the trial.