A $35 Million GLP-1 Bet Pays Off
It looks like Amylyx Pharmaceuticals‘ $35 million gamble on the GLP-1 receptor agonist avexitide has been rewarded: The drug reduced the frequency of life-threatening blood sugar dips by over 60% for patients with a rare endocrine condition.
The approved GLP-1 agonists semaglutide and tirzepatide work by acting like the peptide to stimulate the body to lose weight, while avexitide will act in an opposite manner, blocking the effects of the weight-loss peptide. In the phase 3 Lucidity trial, 78 patients with post-bariatric hypoglycemia (PBH) were given daily injections of either avexitide or placebo.
PBH is a rare complication of gastric bypass surgery, where patients suffer from perilous plunges in blood sugar. It can occur after a meal, though not necessarily, Cohen, co-CEO of Amylyx.
“Sometimes it’s like, ‘Oh, here’s a ball that relates to the giving,'” Cohen said. “Patients describe it happening when they’re stressed or exercising or several other things—and their bodies also become very reactive.”
Participants in the trial had about one case of drastic blood sugar drop per week, Amylyx’s other co-CEO Justin Klee told Fierce.
“All these events are considered medical emergencies, level two or level three hypoglycemic events,” Klee explained. “Every single one.”
Patients have to live “small lives” without any treatments available, Klee said, “where they have to be very careful about what they eat and what they do.” Cohen mentioned patients who only shower when someone else is in the bathroom with them or who have been injured in car accidents because of hypoglycemia while driving.
Phase 3 Lucidity Trial Delivers Strong Results
Avexitide slowed the rate of these hypoglycemia events by 55% through Week 16 of the trial as compared to placebo, thus meeting the trial’s primary endpoint. The drug also met all of the trial’s secondary endpoints and didn’t lead to any severe side effects or changes in body weight, Amylyx reported, with the most common side effects being diarrhea and injection site reactions.
“When we shared the results with the steering committee, they were overjoyed at the results,” Klee said. Their patients were just so excited about what this will mean for their patients.”
“It is a best-case scenario,” wrote analysts from Stifel in a note to investors, adding that the drug had “a very solid primary” and the “biological rationale is very good” and the projected market size of $1.5 billion or more.
By the end of the year, Amylyx will file for approval with the FDA; already preparing heavily for a commercial launch. The Cambridge, Massachusetts-based biopharma has been preparing for the success of Lucidity this year, having had five positive readouts in PBH so far.
“If there’s something important for people you want to get them, they have an obligation to do it as soon as possible,” Klee said.
Because avexitide already has breakthrough therapy designation, he added, Amylyx anticipates potentially receiving priority review.
The company is primarily focused on getting avexitide approved in PBH, but Cohen said the company is also eyeing other surgeries that can impact the body’s nutrient metabolism.
“There are many other surgeries people get that can cause a similar effect,” he said, “whether that’s gastric surgery for gastric cancers, esophageal surgeries for esophageal cancers [or] Nissen fundoplication, which is a surgery for severe gastric reflux.”
Another possible market for avexitide is Japan, where gastric cancer is the top killer of cancer and numerous procedures are performed to treat the disease, Cohen said.
Rebuilding the Pipeline After ALS Setback
In the summer of 2024, the company bought avexitide from Eiger Biopharma for $35.1 million shortly after the company withdrew its amyotrophic lateral sclerosis (ALS) drug Relyvrio from the market. The med got the FDA’s approval in 2022, but then a confirmatory phase-3 trial was not successful.
Eventually, Relyvrio failed to have a positive impact, but Amylyx hasn’t yet abandoned the quest for a cure for patients with ALS. Currently, the biopharma is conducting a phase 1 trial of an antisense oligonucleotide, AMX0114, in ALS patients. AMX0114 is designed to inhibit Calpain-2, an enzyme that is overactive in ALS that is associated with the devastating neurodegeneration.
Amylyx Reports Major Phase 3 Success
Amylyx has reported positive results from its Phase 3 LUCIDITY trial of avexitide, an investigational GLP-1 receptor antagonist designed to treat post-bariatric hypoglycemia. The 78-patient study met its primary endpoint, with avexitide reducing Level 2 and Level 3 hypoglycemic events by 55% compared with placebo over 16 weeks.
For Amylyx, the results represent a major clinical milestone and could open the door to a potential treatment for patients who currently have no FDA-approved therapy specifically for post-bariatric hypoglycemia.
Amylyx Targets Dangerous Blood Sugar Crashes
Post-bariatric hypoglycemia can occur after weight-loss surgery, particularly Roux-en-Y gastric bypass. Patients can experience sudden and potentially severe drops in blood glucose, sometimes resulting in confusion, loss of consciousness, seizures, or the need for emergency care.
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
