Amylyx Pharmaceuticals (NASDAQ:AMLX) plans to seek U.S. approval for its experimental treatment for post-bariatric hypoglycemia by the end of 2026, as it prepares for a potential launch in 2027 following positive late-stage trial results.
During its third-quarter earnings call, the company said an equity offering generated $545 million in net proceeds, bringing cash and cash equivalents to $768 million at the end of September. Amylyx is using the funds to prepare for a possible launch of avexitide, expand its commercial organization and invest in drug supply.
Avexitide is an investigational GLP-1 receptor antagonist being developed for post-bariatric hypoglycemia, or PBH, a condition that causes recurring low blood sugar after bariatric surgery. Chief Medical Officer Camille Bedrosian said there are currently no FDA-approved therapies for the condition.
Late-stage results and regulatory plans
Amylyx reported positive top-line results in August from the Phase 3 LUCIDITY trial, which enrolled people with PBH following Roux-en-Y gastric bypass surgery. The study met its primary endpoint, with avexitide producing a 55% reduction in the combined rate of level 2 and level 3 hypoglycemic events compared with placebo, Bedrosian said. The result had a p-value of 0.000003.
Bedrosian said the trial also met all secondary endpoints and that avexitide was generally well tolerated. Most adverse events were mild to moderate, and no serious adverse events were deemed related to treatment, she said. More than 90% of participants completed the 16-week double-blind treatment period, and all eligible participants entered the ongoing open-label extension.
Further results are scheduled for a late-breaking presentation at ObesityWeek on Nov. 15. Amylyx said it remains on track to submit a new drug application to the FDA by year-end. Avexitide has received the agency’s breakthrough therapy and orphan drug designations.
The scope of any potential approval remains uncertain. Bedrosian said the application will include data from the broader avexitide program, including a Phase 2b study involving people who had other upper gastrointestinal or bariatric surgeries. Co-CEO Justin Klee said it was too early to determine the eventual label or indication.
Launch preparations and patient estimates
Chief Commercial Officer Dan Monahan said the company estimates that approximately 160,000 people in the U.S. have PBH, including approximately 118,000 who received Roux-en-Y gastric bypass surgery and approximately 48,000 who received sleeve gastrectomy. Amylyx said its estimates are supported by claims analyses, surveys and discussions with clinicians.
Co-CEO Josh Cohen said the claims work initially identified people with hypoglycemic events following bariatric surgery that could not be explained by other causes. Monahan said a PBH-specific ICD-10 diagnosis code took effect in October, although he expects adoption to take time.
Endocrinologists are expected to be the primary prescribers if avexitide is approved, Monahan said, while some bariatric surgery centers will also be a focus. Amylyx is expanding disease education and hiring staff across marketing, market access, patient services and commercial operations.
Monahan said payer information exchanges are planned for the first quarter of 2027, when the company expects to discuss potential coverage requirements. It is too early to discuss specific pricing, he said. Amylyx did not provide enrollment figures for its ongoing expanded access program or a forecast for the number of patients who might begin treatment at launch.
Quarterly spending and pipeline
Third-quarter operating expenses rose 38% from a year earlier to $49.7 million. Research and development expenses increased to $28.4 million from $19.9 million, primarily because of spending on LUCIDITY and other program costs, Chief Financial Officer Jim Frates said. Selling, general and administrative expenses rose to $21.3 million from $16.2 million, partly reflecting personnel costs for pre-commercialization activities and the vesting of performance stock unit awards.
Beyond avexitide, Amylyx is targeting a 2027 investigational new drug filing for AMX0318, a long-acting GLP-1 receptor antagonist. The company is also advancing its Phase 1 LUMINA trial of AMX0114 in people with ALS. Cohen said enrollment in the third dose cohort was complete and enrollment in the fourth had begun.
Bedrosian said early results from the first two LUMINA cohorts showed the drug was well tolerated, but it was too early to assess biomarker results. Klee also cited ongoing work on AMX0035 in Wolfram syndrome and said the company’s existing programs are its primary focus for the next 12 months.
