Avexitide Part 3 LUCIDITY accomplished dosing and the corporate says it’s on observe for a near-term top-line readout, with administration and the CFO pointing to potential readout timing round Q3 2026.
Expanded entry program (EAP) launched within the U.S. to offer avexitide to as much as 250 adults with publish‑bariatric hypoglycemia after Roux‑en‑Y gastric bypass, supposed to make sure continuity of remedy for trial members; the drug stays investigational.
Industrial and monetary readiness: Amylyx is drafting NDA sections, constructing a launch group and illness‑consciousness efforts concentrating on an estimated ~160,000 U.S. PBH sufferers, and ended Q1 with $279.8 million in money to fund its runway into 2028 whereas getting ready for a possible 2027 launch if accredited.
Amylyx Prescription drugs (NASDAQ:AMLX) executives highlighted continued progress throughout the corporate’s pipeline throughout its first-quarter 2026 earnings name, with a heavy concentrate on the pivotal Part 3 LUCIDITY trial evaluating avexitide in post-bariatric hypoglycemia (PBH) and preparations for a possible 2027 industrial launch if the drug is accredited.
Avexitide Part 3 LUCIDITY trial nearing top-line outcomes
Co-CEO Justin Klee mentioned the quarter was “marked by execution throughout our pipeline,” pointing first to the LUCIDITY examine of avexitide, which the corporate describes as an investigational first-in-class GLP-1 receptor antagonist with FDA Breakthrough Remedy designation in PBH. Klee mentioned the corporate randomized and dosed the final participant in late March, calling it “a big milestone,” and mentioned the corporate stays “on observe for a top-line readout subsequent quarter.”
Chief Medical Officer Dr. Camille L. Bedrosian described PBH as a power metabolic situation pushed by an exaggerated GLP-1 response after meals consumption, resulting in recurrent hypoglycemia and neuroglycopenia. She emphasised there are presently no FDA-approved therapies. Bedrosian mentioned LUCIDITY is evaluating avexitide 90 mg as soon as day by day in PBH following Roux-en-Y gastric bypass surgical procedure, utilizing an FDA-agreed major endpoint: discount within the composite of degree 2 and degree 3 hypoglycemic occasions by week 16.
Bedrosian mentioned LUCIDITY was designed with “the aim of replication,” noting that 5 prior avexitide trials in PBH knowledgeable the dose, endpoint, and inclusion standards. In Q&A, administration mentioned they intend to report extra info past the first endpoint, together with secondary endpoints that look individually at degree 2 and degree 3 occasions. Bedrosian defined that degree 3 occasions are adjudicated by an impartial group of endocrinologists blinded to check information.
When requested about evaluating baseline traits in Part 3 versus earlier research and the potential placebo response, Bedrosian mentioned the trial stays ongoing and blinded and the corporate wouldn’t touch upon particulars of the examine inhabitants. She added that prior Part 2 trials confirmed statistically vital and clinically significant reductions in hypoglycemic occasions and that the Part 3 examine was powered to detect a clinically significant discount “even underneath probably the most conservative circumstances.”
Expanded entry program launched for PBH sufferers
Bedrosian additionally disclosed that Amylyx not too long ago launched a U.S. expanded entry program (EAP) to offer avexitide for as much as 250 adults with PBH following Roux-en-Y gastric bypass surgical procedure. She mentioned this system was launched in response to requests from sufferers and physicians and is meant to offer continuity of remedy for sufferers finishing parts of the LUCIDITY program. Preliminary eligible sufferers embody adults who’ve accomplished LUCIDITY or participated in earlier avexitide PBH trials.
“We’re beginning the EAP now as a result of we wanna make sure there’s continuity of remedy for folks within the LUCIDITY examine,” Bedrosian mentioned in response to a query concerning the timing of this system relative to typical Part 3 timelines.
Administration repeatedly emphasised that avexitide stays investigational and isn’t accredited by the FDA for any indication.
Industrial and regulatory preparations underway
Klee mentioned Amylyx is advancing three “strategic imperatives” for avexitide: shifting towards top-line information, strengthening NDA readiness, and constructing launch readiness. He mentioned the corporate is already drafting NDA sections to help a possible submission.
In Q&A, Bedrosian mentioned the corporate’s early NDA work is meant to enhance effectivity after top-line outcomes. She cited a “nice sense of urgency” given the shortage of accredited therapies in PBH, including that the corporate has an skilled group with prior regulatory submission expertise.
Chief Industrial Officer Dan Monahan outlined efforts to arrange for a possible U.S. launch, together with hiring throughout advertising, market entry, and industrial operations, and a near-term concentrate on PBH illness state schooling. Monahan mentioned the corporate plans to launch a PBH consciousness marketing campaign this summer time, emphasizing pathophysiology, well timed analysis, and illness burden.
Monahan mentioned Amylyx’s impartial claims evaluation and subject engagement help its estimate of roughly 160,000 folks dwelling with PBH within the U.S. following sleeve gastrectomy and Roux-en-Y gastric bypass. He added that market analysis signifies endocrinologists have a “excessive intent to deal with PBH if there have been to be an accredited drugs.”
When requested how the corporate identifies sufferers utilizing claims information, Monahan mentioned the evaluation begins with bariatric surgical procedure sufferers after which filters for documented non-diabetic hypoglycemia, adopted by extra indicators and signs related to PBH, reminiscent of fatigue, dizziness, seizures, blood glucose exams, and emergency room visits. He mentioned repeated analyses have bolstered the corporate’s confidence in its prevalence estimate. Administration additionally mentioned it’s validating claims findings by outreach to medical websites.
Co-CEO Josh Cohen additionally pointed to alerts of rising recognition of PBH, citing a March publication from Stanford researchers modeling U.S. prevalence and noting that CMS revealed an inventory of ICD-10 codes probably efficient Oct. 1, 2026, that features a PBH-specific code. Administration mentioned an ICD-10 code can be useful for monitoring and analysis however just isn’t required for commercialization given different identification strategies reminiscent of claims evaluation.
On industrial deployment, Monahan mentioned the corporate is initiating go-to-market efforts and recommended salesforce expectations in keeping with a “uncommon endocrine launch.” He additionally mentioned medical affairs has begun hiring regional scientific administrators (MSLs), with hires in place. He added that the corporate expects to start with facilities of excellence and key opinion leaders and later broaden outreach into broader endocrinology as illness state schooling progresses.
Pipeline updates past avexitide
Klee offered updates throughout different applications:
AMX0318: The corporate’s long-acting GLP-1 receptor antagonist has IND-enabling research underway, with a 2027 IND submitting focused.
AMX0035 (Wolfram syndrome): Amylyx expects to current longer-term week 96 information from the Part 2 open-label HELIOS trial at an upcoming scientific assembly.
AMX0114 (ALS): The corporate totally enrolled cohort 2 of the Part 1 LUMINA trial in March. Administration mentioned it expects to current early biomarker information from cohort 1 in June at ECTRIMS in Madrid, Spain. Bedrosian mentioned the biomarkers embody measures associated to the calpain-2 ASO mechanism in addition to biomarkers associated to the ALS illness course of.
Throughout Q&A, administration mentioned it’s troublesome to foretell biomarker motion on the lowest dose cohort for AMX0114 till information are in hand, noting the dose is on the low finish of ranges sometimes studied for intrathecal ASOs. The corporate mentioned its biomarker aim is to evaluate whether or not the medical information replicate preclinical biology and whether or not there could also be impacts on prognostic ALS biomarkers.
Monetary outcomes and money runway
Chief Monetary Officer James Frates mentioned first-quarter outcomes have been “according to our plans” and mirrored concentrate on the Part 3 LUCIDITY trial and “focused investments” throughout the pipeline. The corporate ended the quarter with $279.8 million in money and marketable securities, in contrast with $317 million on the finish of the fourth quarter of the prior yr. Frates mentioned the corporate expects that capital to fund its anticipated money runway into 2028, together with anticipated milestones such because the LUCIDITY top-line readout in Q3 2026 and potential FDA approval and industrial launch of avexitide in 2027.
Complete working bills have been $43.8 million, up 16% from the identical interval in 2025. R&D bills rose to $27.6 million from $22.1 million, pushed primarily by elevated spending associated to avexitide medical growth. Frates additionally famous a $4 million milestone fee to Gubra following identification of AMX0318 as a growth candidate for PBH and different uncommon ailments. SG&A bills have been $16.2 million, in contrast with $15.7 million, primarily on account of increased consulting {and professional} companies prices tied to launch preparation for avexitide.
Frates mentioned money utilization was barely increased in Q1 than This fall because of the Gubra milestone funds and fee of the annual company bonus.
Closing the decision, Cohen reiterated the corporate’s concentrate on LUCIDITY execution and mentioned Amylyx is getting ready for a regulatory submission after top-line outcomes whereas scaling industrial and medical groups for a possible 2027 launch “if accredited.”
About Amylyx Prescription drugs (NASDAQ:AMLX)
Amylyx Prescription drugs, Inc is a biopharmaceutical firm devoted to growing remedies for uncommon and debilitating neurological ailments. Based in 2013 and headquartered in Cambridge, Massachusetts, the corporate focuses on leveraging novel approaches to focus on mobile pathways implicated in neurodegeneration. Amylyx’s analysis platform facilities on small-molecule therapies designed to guard neurons and help mobile well being in sufferers with situations that presently have restricted or no disease-modifying remedy choices.
The corporate’s lead product, AMX0035, is marketed underneath the commerce identify Relyvrio following U.S.
