The Food and Drug Administration offered mixed messages Friday for Amylyx Pharmaceuticals' experimental neurological disease treatment, and AMLX stock crashed.
Amylyx is working on a treatment for amyotrophic lateral sclerosis, or ALS. Also known as Lou Gehrig's disease, the condition impacts voluntary muscle movement. On Wednesday, Amylyx will go before the FDA's Peripheral and Central Nervous System Drugs Advisory Committee with the hopes of snagging a recommendation for its drug — again.
But the briefing documents posted ahead of that meeting Friday were mixed. The FDA laid out the flexible paths it has for approval, but also said Amylyx's treatment doesn't meet the bar for an accelerated approval. Amylyx is currently waiting on the results of a Phase 3 study of the drug it calls AMX0035. The FDA could approve the drug later this month without it, though.
"In the case of AMX0035 for ALS, we do not have data for an effect of AMX0035 on a (goal) that is reasonably likely to predict clinical benefit for ALS," the FDA said in the document. It noted the results so far are acceptable to support a traditional approval, but not an accelerated one.
On today's stock market, AMLX stock plummeted 23.2% to 18.08.
The Controversy Surrounding AMLX Stock
It's been an uphill battle for Amylyx in ALS treatment. In a study called Centaur, patients showed improvements in muscle function. But the benefits on survival weren't clear. The FDA is waiting on data from another study called Phoenix to help confirm the results.
Still, Amylyx asked for approval based on the Centaur study. After a split advisory committee said the benefits of the drug outweigh its risks in March, the FDA pushed back its review date until Sept. 29 to examine additional data from Amylyx.
The briefing documents posted Friday acknowledge officials in Canada have approved the ALS drug. It now sells as Albrioza. The FDA noted the approval in Canada is based on "promising evidence" rather than "substantial evidence," similar to an accelerated approval from the FDA.
However, the agency pointed out that the data Amylyx submitted for review don't confirm the benefit of AMX0035 in ALS treatment.
Now, the FDA's mixed messages have sent AMLX stock tumbling to a low not seen since mid-June.
Highly Rated, But Only On One Metric
Amylyx says it plans to use the Wednesday meeting to present additional analyses of the survival benefit, including survival in the Centaur study vs. traditional ALS rates. The company also says it has new evidence from a third study called Pegasus in Alzheimer's patients. This study shows the benefit of AMX0035 in certain neurological disease targets.
"We look forward to presenting data to support AMX0035 for the treatment of ALS, a rapidly progressive and universally fatal disease, to the reconvened Peripheral and Central Nervous System Drugs Advisory Committee," the company said in an email to Investor's Business Daily.
Promisingly, Amylyx shares have a best-possible Relative Strength Rating of 99. This puts AMLX stock in the leading 1% of all stocks in terms of 12-month performance, according to IBD Digital.
But shares have a middling Composite Rating of 57, reflecting shaky fundamental and technical factors.
Follow Allison Gatlin on Twitter at @IBD_AGatlin.
