MOFF attended the NEALS webinar hosted by Dr. Sabrina Paganoni and Dr. Merit Cudkowicz on Wednesday, Oct 21, 2020. Click here to gain full access to the webinar once it becomes available.
Background Information
- 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.
Key Findings
- 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.