A once high-flying Boston-area drug company mounted somewhat of a comeback Tuesday, reporting positive results from a clinical trial testing a potentially first-of-its-kind treatment for the dangerously low blood sugar levels some patients experience after bariatric surgery.
Amylyx Pharmaceuticals said its medicine, known as avexitide, hit the main goal of the late-stage study by spurring a 55% reduction in serious to severe hypoglycemic events compared to a placebo. Such events starve the brain of the sugar it needs for fuel, leading to confusion, dizziness, slurred speech and loss of coordination. In more dire cases, patients can faint, have seizures or become comatose.
According to Amylyx, its medicine also appeared safe. Researchers classified a majority of the adverse events seen in the trial as mild or moderate, with the most common being diarrhea and bruising or redness at the injection site. Amylyx now plans to, before the end of the year, formally ask the Food and Drug Administration to approve avexitide.
“What we heard very consistently from endocrinologists and people with [post-bariatric hypoglycemia] is that every one of these events is a medical emergency. Preventing even a single event matters,” Justin Klee, one of the company’s co-founders and co-CEOs, said in an interview.
Klee added that, when the Amylyx team notified the medical experts sitting on the trial’s steering committee about these findings, they were “over the moon.” There are currently no approved drugs for post-bariatric hypoglycemia, a condition which, according to research cited by the Cleveland Clinic Journal of Medicine, affects around one-tenth to one-third of patients who undergo the procedure.
“This is a totally debilitating disease for people who get it,” said Amylyx’s other co-founder and co-CEO, Joshua Cohen, who described the new results as a “home run” and far above most expectations.
Cohen and Klee declined to say whether Amylyx will meet with FDA staff before filing for approval. However, they did note how the agency already granted avexitide a special status meant to speed the development and review of certain treatments for serious or life-threatening conditions.
Avexitide acts on the same GLP-1 receptors as the new, mega-blockbuster class of diabetes and weight loss medications sold by Eli Lilly and Novo Nordisk. Yet, unlike those drugs, avexitide blocks the receptors to decrease insulin production and stabilize blood sugar levels.
Amylyx acquired the drug for $35 million through the 2024 bankruptcy proceedings of Eiger BioPharmaceuticals. According to Klee and Cohen, the purchase made strategic sense for several reasons, including that Eiger had already run a handful of mid-stage trials focused on either post-bariatric hypoglycemia or “congenital hyperinsulinism,” a genetic disorder wherein the pancreas creates too much insulin.
The duo therefore bet that Amylyx would have to notch just one positive, late-stage experiment to submit a marketing application. They said their team has been putting together an application all this year, and that commercial launch preparations are underway.
In a note to clients Monday, analysts at Mizuho Securities estimated that risk-adjusted revenue from avexitide could reach $1.3 billion by 2040.
Joseph Thome, an analyst at TD Cowen, has modeled peak annual sales of avexitide hitting $1.5 billion in the U.S., though that figure “may prove conservative as diagnosis and awareness improve.”
Amylyx disclosed after markets closed Monday that the avexitide trial results would come early Tuesday. The company’s stock price subsequently rose 14% in after-hours trading.
Amylyx shares have steadily rebounded since spring of 2024, when the company said it would be pulling its only product — a medication for amyotrophic lateral sclerosis — from the market. That rare move came after a large clinical trial meant to confirm the drug’s benefits instead found it no better than a placebo at slowing the fatal, nerve-destroying disease.
Amylyx continues to assess that drug, codenamed AMX0035, as a treatment for a rare disorder known as Wolfram syndrome. It also has an experimental, genetic medicine for ALS.
The company reported a net loss of $43 million in the second quarter. It had $251 million in cash, cash equivalents and short-term investments as of June 30, a runway that should keep it operational into 2028.
