-- Topline data readout from Phase 3 LUCIDITY clinical trial of avexitide
in post-bariatric hypoglycemia on track; anticipated in Q3 2026
-- Cash runway expected to fund operations through potential avexitide
commercialization and into 2028
-- Management to host conference call and webcast today at 8:00 a.m.
Eastern Time
CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 07, 2026--
Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) ("Amylyx" or the "Company") today reported financial and business results for the first quarter ended March 31, 2026.
"With enrollment complete in the pivotal Phase 3 LUCIDITY clinical trial, we have a clear line of sight to the anticipated Q3 topline data readout, bringing us one step closer to the potential of delivering the first approved therapy for the post-bariatric hypoglycemia community," said Joshua Cohen and Justin Klee, Co-CEOs of Amylyx. "We understand the devastating daily burden of this condition and are operating with a sense of urgency to advance our program. We have initiated regulatory and commercial readiness activities to help ensure we are positioned to move swiftly following LUCIDITY topline data. Supported by an expected cash runway extending into 2028, we are executing with focus and discipline as we work to bring this treatment to the PBH community in 2027, if approved."
First Quarter and Recent Updates:
-- Amylyx completed enrollment for the pivotal Phase 3 LUCIDITY clinical
trial of avexitide, an investigational, first-in-class glucagon-like
peptide-1 (GLP-1) receptor antagonist with U.S. Food and Drug
Administration (FDA) Breakthrough Therapy Designation in post-bariatric
hypoglycemia $(PBH)$, in March 2026. LUCIDITY enrolled 78 participants and
is a 16-week, multicenter, randomized, double-blind, placebo-controlled
trial evaluating the efficacy and safety of avexitide in adults with PBH
following Roux-en-Y gastric bypass (RYGB) surgery. Participants who
complete the 16-week double-blind period are eligible to enter a 32-week
open-label extension (OLE) period.
-- Amylyx announced the initiation of an Expanded Access Program (EAP) for
the use of avexitide to treat U.S. adults with PBH following RYGB surgery
in May 2026. Initial eligible patients include individuals who have
completed the pivotal Phase 3 LUCIDITY clinical trial and participants in
a prior trial of avexitide in PBH following RYGB surgery.
-- Amylyx completed enrollment of Cohort 2 (n=12) of the Phase 1 LUMINA
clinical trial of AMX0114, an investigational antisense oligonucleotide
$(ASO)$ targeting calpain-2 with FDA Fast Track Designation for the
potential treatment of amyotrophic lateral sclerosis (ALS), in March
2026. The LUMINA trial is a randomized, double-blind, placebo-controlled,
multiple ascending dose clinical trial of AMX0114 in people living with
ALS. LUMINA is evaluating the safety, tolerability, pharmacokinetics, and
pharmacodynamics of AMX0114 in people living with ALS and assessing both
novel and broadly researched ALS biomarkers, including change from
baseline in neurofilament light chain (NfL) levels.
Upcoming Expected Milestones:
-- Topline data readout for Phase 3 LUCIDITY clinical trial of avexitide
in PBH is on track and anticipated in the third quarter of 2026. If
approved, commercial launch of avexitide is anticipated in 2027. LUCIDITY
is evaluating the FDA-agreed-upon primary outcome of reduction in the
composite of Level 2 and Level 3 hypoglycemic events through Week 16.
LUCIDITY was informed by data from five prior PBH clinical trials of
avexitide showing consistent effects, most notably statistically
significant reductions in Level 2 and Level 3 hypoglycemic events.
Avexitide was generally well-tolerated, with a favorable safety profile
replicated across previous clinical trials.
-- Presentation of early biomarker data from Cohort 1 (n=12) of the Phase
1 LUMINA clinical trial of AMX0114 in ALS is expected at the 2026
European Network to Cure ALS (ENCALS) Annual Meeting in June 2026. Cohort
1 of LUMINA is investigating the first and lowest of four doses being
evaluated in the trial. The data are expected to provide initial
information about the levels of the ALS biomarkers being assessed from
the first dose in the LUMINA trial. The Company previously presented
early safety and tolerability data from Cohort 1 of LUMINA showing
AMX0114 was generally well-tolerated, with no treatment-related serious
adverse events (SAEs).
-- Investigational New Drug $(IND)$-enabling studies for AMX0318, a novel
GLP-1 receptor antagonist for long-acting administration to treat PBH and
other rare diseases, are underway with an IND filing targeted for 2027.
AMX0318 was selected as a development candidate after demonstrating
robust preclinical and chemical properties, including a favorable
pharmacokinetic profile that may support long-acting administration, a
robust chemical stability profile, strong in vitro potency, evidence of
in vivo activity and tolerability, and high solubility. AMX0318 was
identified through a research collaboration with Gubra A/S (Gubra), a
company specializing in peptide-based drug discovery and preclinical
contract research services.
Financial Results for the First Quarter Ended March 31, 2026
R&D Expenses: Research and development expenses for the first quarter of 2026 were $27.6 million, compared to $22.1 million for the same period in 2025. The increase was primarily due to an increase in spending related to the clinical development of avexitide in PBH. Milestone payments totaling $4.0 million to Gubra were also recognized following the selection and handover of AMX0318 as a development candidate for PBH and other rare diseases. The increase was offset primarily by decreased spending related to AMX0035 for the treatment of progressive supranuclear palsy $(PSP)$. Research and development expenses include $1.8 million of stock-based compensation expense for the quarter ended March 31, 2026, compared to $1.8 million of stock-based compensation expense for the quarter ended March 31, 2025.
SG&A Expenses: Selling, general, and administrative expenses for the first quarter of 2026 were $16.2 million, compared to $15.7 million for the same period in 2025. This increase was primarily due to an increase in consulting and professional services. Selling, general, and administrative expenses include $4.4 million of stock-based compensation expense for the quarter ended March 31, 2026, compared to $5.0 million of stock-based compensation expense for the quarter ended March 31, 2025.
Net Loss: Net loss for the three months ended March 31, 2026 was $41.3 million, or $0.37 per share, compared to net loss of $35.9 million, or $0.42 per share, for the same period in 2025.
Cash Position: Cash, cash equivalents, and short-term investments were $279.8 million at March 31, 2026, compared to $317.0 million at December 31, 2025. Based on its current operating plans, Amylyx expects a cash runway into 2028.
Investor Conference Call Information
Amylyx's management team will host a conference call today, May 7, 2026, at 8:00 a.m. ET to discuss financial results and provide an update on the business. To access the conference call, please dial +1 (888)-880-3330 (U.S. & Canada) or +1 (646)-357-8766 (international) at least 10 minutes prior to the start time and ask to be joined into the Amylyx Pharmaceuticals call. A live audio webcast of the call will be available under "Events and Presentations" in the Investor section of the Company's website, https://investors.amylyx.com/events-presentations. The webcast will be archived and available for replay for 90 days following the event.
Available Information
Amylyx periodically provides other information for investors on the Company's corporate website, https://amylyx.com, and the Company's investor relations website, https://investors.amylyx.com. This includes press releases and other information about financial performance, information on corporate governance, and details related to our annual meeting of stockholders. Amylyx intends to use its website as a means of disclosing material non-public information and for complying with its disclosure obligations under Regulation FD. Accordingly, investors should monitor Amylyx's website, in addition to following the Company's press releases, SEC filings, and public conference calls and webcasts.
About Avexitide
Avexitide is an investigational, first-in-class glucagon-like peptide-1 (GLP-1) receptor antagonist that has been evaluated in five Phase 1 and Phase 2 clinical trials for post-bariatric hypoglycemia (PBH) and has also been studied in congenital hyperinsulinism $(HI)$. The U.S. Food and Drug Administration (FDA) has granted avexitide Breakthrough Therapy Designation for both indications, Rare Pediatric Disease Designation in congenital HI, and Orphan Drug Designation for the treatment of hyperinsulinemic hypoglycemia (which includes PBH and congenital HI). In PBH, an exaggerated GLP-1 response leads to excessive insulin secretion, resulting in recurrent hypoglycemic events. Avexitide is a GLP-1 receptor antagonist designed to competitively bind to the GLP-1 receptor on pancreatic islet beta cells and inhibit the exaggerated GLP-1-driven insulin response characteristic of PBH, reducing inappropriate insulin secretion and stabilizing blood glucose levels. In two Phase 2 PBH clinical trials, avexitide demonstrated highly statistically significant reductions in hypoglycemic events.
About Post-Bariatric Hypoglycemia (PBH)
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