In September, we shared with you the exciting news that AMX0035, a trial ALS treatment drug developed by Amylyx Pharmaceuticals that had just completed the phase 2 trial, showed a statistically significant benefit to people living with ALS. This promising news lead The ALS Association and I AM ALS to launch a petition asking the FDA and Amylyx to work together to make AMX0035 available to the ALS community as soon as possible.
And while the news of a possible significant benefit to people living with ALS has created much optimism in the ALS community, the story doesn’t end there. All patients who completed the Phase 2 study were eligible to enroll in an “open label extension” (OLE) study to receive AMX0035 with no placebo comparator for up to an additional 30 months. Out of the 98 participants who were eligible for OLE enrollment, 92 percent (or 90 participants), opted to enter the OLE.
The primary goal of an OLE is to gather information about safety and tolerability of the new drug in long term, day to day use outside the trial environment, but they also can and do provide information on the effectiveness of the drug being tested. In this case, those findings make an even more compelling case for providing AMX0035 to the ALS community while Phase 3 trials take place, not after.
Published this month in the journal Muscle and Nerve, the results of the OLE showed median overall survival was 25.0 months among participants originally randomized to AMX0035 compared to 18.5 months among participants originally randomized to the placebo. This represents a statistically significant 6.5 month increase in time survived.
“The findings from the open label extension (OLE) study of AMX0035 further show that the treatment should be made available to people with ALS as soon as possible. People participating in the OLE study appeared to live several months longer than they would have without AMX0035,” The ALS Association stated in response to the study results. “These findings were in addition to the findings reported last month showing a significant functional benefit to people with ALS. We urge the FDA and Amylyx to work together to bring this treatment to market as soon as possible and we stand ready to help ensure that happens.”
To date, nearly 52,000 people have signed on to the petition to the FDA and Amylyx. There are still a few more days for you to add your voice to those calling for this treatment to be made available to people with ALS as quickly as possible. Sign on to the petition NOW and make your voice heard for the ALS community.