Amlyx Pharmaceuticals is a small, young company in Cambridge, MA. It was started in 2015 by two Brown University graduates to develop drugs to specifically combat neurodegenerative diseases. Their initial focus has been on ALS -- Lou Gehrig's disease -- but has now expanded into Alzheimer's disease. When their investigative drug, AMX0035, was shown to be effective in treating ALS, they began an investigation of its use to treat Alzheimer's disease.
The phase 2 trial, called the Pegasus Trial, has just been completed, and they are now slicing and dicing the data. The trial involved 100 test subjects over a period of 24 months. The test subjects were in the late stage of mild cognitive impairment due to Alzheimer's disease or the early stage of Alzheimer's dementia. They expect to share their results sometime in the first half of 2021.
The phase 1 trial was focused on ALS patients, so we don't have a preview of what to expect with the new trial for Alzheimer's patients. It was not a combined phase 2/3 trial, so it appears to me that we will need a phase 3 trial before the FDA will consider approving it. The results with this 100 (ultimately 96) test subjects will likely influence approval to begin a phase 3 trial. But I'll note here that they allowed the phase 3 trials of aducanumab to proceed based primarily on promising results in the small phase 1 trial that were not replicated in the phase 2 trial. (In Beating the Dementia Monster, we discussed my puzzlement in 2016 over why they were proceeding with the phase 3 trial of aducanumab in the absence of positive results from the phase 2 trial.)
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