Avexitide Part 3 LUCIDITY accomplished dosing and the corporate says it’s on monitor 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 therapy for trial members; the drug stays investigational.
Business 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 Prescribed 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 stated the quarter was “marked by execution throughout our pipeline,” pointing first to the LUCIDITY research of avexitide, which the corporate describes as an investigational first-in-class GLP-1 receptor antagonist with FDA Breakthrough Remedy designation in PBH. Klee stated the corporate randomized and dosed the final participant in late March, calling it “a major milestone,” and stated the corporate stays “on monitor 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 at present no FDA-approved therapies. Bedrosian stated LUCIDITY is evaluating avexitide 90 mg as soon as each day in PBH following Roux-en-Y gastric bypass surgical procedure, utilizing an FDA-agreed main endpoint: discount within the composite of stage 2 and stage 3 hypoglycemic occasions by way of week 16.
Bedrosian stated LUCIDITY was designed with “the objective of replication,” noting that 5 prior avexitide trials in PBH knowledgeable the dose, endpoint, and inclusion standards. In Q&A, administration stated they intend to report extra info past the first endpoint, together with secondary endpoints that look individually at stage 2 and stage 3 occasions. Bedrosian defined that stage 3 occasions are adjudicated by an impartial group of endocrinologists blinded to check knowledge.
When requested about evaluating baseline traits in Part 3 versus earlier research and the potential placebo response, Bedrosian stated the trial stays ongoing and blinded and the corporate wouldn’t touch upon particulars of the research inhabitants. She added that prior Part 2 trials confirmed statistically vital and clinically significant reductions in hypoglycemic occasions and that the Part 3 research 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 lately 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 stated this system was launched in response to requests from sufferers and physicians and is meant to offer continuity of therapy for sufferers finishing parts of the LUCIDITY program. Preliminary eligible sufferers embrace adults who’ve accomplished LUCIDITY or participated in earlier avexitide PBH trials.
“We’re beginning the EAP now as a result of we wanna ensure there’s continuity of therapy for individuals within the LUCIDITY research,” Bedrosian stated in response to a query in regards to 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.
Business and regulatory preparations underway
Klee stated Amylyx is advancing three “strategic imperatives” for avexitide: shifting towards top-line knowledge, strengthening NDA readiness, and constructing launch readiness. He stated the corporate is already drafting NDA sections to assist a possible submission.
In Q&A, Bedrosian stated the corporate’s early NDA work is meant to enhance effectivity after top-line outcomes. She cited a “nice sense of urgency” given the dearth of accredited therapies in PBH, including that the corporate has an skilled crew with prior regulatory submission expertise.
Chief Business Officer Dan Monahan outlined efforts to arrange for a possible U.S. launch, together with hiring throughout advertising and marketing, market entry, and industrial operations, and a near-term concentrate on PBH illness state schooling. Monahan stated the corporate plans to launch a PBH consciousness marketing campaign this summer season, emphasizing pathophysiology, well timed analysis, and illness burden.
Monahan stated Amylyx’s impartial claims evaluation and area engagement assist its estimate of roughly 160,000 individuals residing 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 knowledge, Monahan stated 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, corresponding to fatigue, dizziness, seizures, blood glucose exams, and emergency room visits. He stated repeated analyses have bolstered the corporate’s confidence in its prevalence estimate. Administration additionally stated it’s validating claims findings by way of outreach to scientific 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 printed an inventory of ICD-10 codes doubtlessly efficient Oct. 1, 2026, that features a PBH-specific code. Administration stated an ICD-10 code could be useful for monitoring and analysis however will not be required for commercialization given different identification strategies corresponding to claims evaluation.
On industrial deployment, Monahan stated the corporate is initiating go-to-market efforts and advised salesforce expectations per a “uncommon endocrine launch.” He additionally stated 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 develop outreach into broader endocrinology as illness state schooling progresses.
Pipeline updates past avexitide
Klee supplied 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 knowledge from the Part 2 open-label HELIOS trial at an upcoming scientific assembly.
AMX0114 (ALS): The corporate absolutely enrolled cohort 2 of the Part 1 LUMINA trial in March. Administration stated it expects to current early biomarker knowledge from cohort 1 in June at ECTRIMS in Madrid, Spain. Bedrosian stated the biomarkers embrace measures associated to the calpain-2 ASO mechanism in addition to biomarkers associated to the ALS illness course of.
Throughout Q&A, administration stated it’s tough to foretell biomarker motion on the lowest dose cohort for AMX0114 till knowledge are in hand, noting the dose is on the low finish of ranges sometimes studied for intrathecal ASOs. The corporate stated its biomarker objective is to evaluate whether or not the scientific knowledge 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 stated first-quarter outcomes had 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 12 months. Frates stated 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 had 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 scientific growth. Frates additionally famous a $4 million milestone cost to Gubra following identification of AMX0318 as a growth candidate for PBH and different uncommon ailments. SG&A bills had been $16.2 million, in contrast with $15.7 million, primarily as a consequence of increased consulting {and professional} providers prices tied to launch preparation for avexitide.
Frates stated money utilization was barely increased in Q1 than This fall because of the Gubra milestone funds and cost of the annual company bonus.
Closing the decision, Cohen reiterated the corporate’s concentrate on LUCIDITY execution and stated 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 Prescribed drugs (NASDAQ:AMLX)
Amylyx Prescribed 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 assist mobile well being in sufferers with situations that at present have restricted or no disease-modifying therapy choices.
The corporate’s lead product, AMX0035, is marketed underneath the commerce title Relyvrio following U.S.
