FDA Again Highlights Lacking Data From Amylyx's Amyotrophic Lateral Sclerosis Candidate

The FDA released briefing documents for Amylyx Pharmaceuticals Inc's AMLX experimental amyotrophic lateral sclerosis drug, as a second adcomm is scheduled tomorrow.
While acknowledging that ALS treatments are "desperately needed," FDA reviewers wrote that Amylyx's submission of new analyses is "not independent data and is simply a new method for analyzing the same survival data presented in the original NDA submission."
See all the FDA documents here.
The new data packages involve patients who switched from treatment to placebo after the Phase 2 trial, suggesting better survival results than the study initially indicated.
"The Applicant has conducted this new analysis to attempt to estimate what survival would have been in patients assigned to placebo had they never entered the [open-label extension]. This can only be done with strong, untestable assumptions," the documents state.
Outside advisors on the FDA's committee voted against recommending Amylyx's AMX0035 for approval in March, deciding that a single randomized Phase 2 study wasn't robust enough to prove efficacy.
The briefing documents mentioned that Amylyx's additional submissions contain no new data from Phase 2 or open-label extension. They also noted that AMX0035 is in an ongoing Phase 3 trial in ALS, which is slated for completion in late 2023 or early 2024.
On the biomarker data, reviewers said that the results may suggest AMX0035 has pharmacodynamic activity in Alzheimer's patients, but "there is no clear or consistent relationship between the biomarkers that had nominally significant findings and the ones that did not to suggest a true treatment benefit on nervous system inflammation or neuronal degeneration."
Price Action: AMLX shares are down 5.70% at $17.05 on the last check Tuesday.

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