Joshua Cohen and Justin Klee, Co-CEOs of Amylyx, shared, “In the first quarter, the company made significant progress across its pipeline, including the strong clinical execution of its pivotal Phase 3 LUCIDITY trial of avexitide in post-bariatric hypoglycemia. The team remains focused on avexitide and expects to complete enrollment by 2025, with topline data anticipated in the first half of 2026. Additionally, progress was made with the Phase 1 LUMINA trial of AMX0114 in ALS, with early cohort data expected later this year. We are also excited to present new long-term data from the Phase 2 HELIOS trial of AMX0035 in Wolfram syndrome, along with topline data from the Phase 2b portion of the ORION trial of AMX0035 in progressive supranuclear palsy (PSP) expected in the third quarter of 2025. With strong momentum and expected cash runway through 2026, we remain confident in achieving our goals and look forward to our upcoming milestones.”

First Quarter and Recent Updates:
LUCIDITY Trial for Avexitide: Amylyx began dosing participants in the pivotal Phase 3 LUCIDITY trial of avexitide, a GLP-1 receptor antagonist with FDA Breakthrough Designation, for post-bariatric hypoglycemia (PBH). In April 2025, the first participant was dosed in this trial, which will involve approximately 75 participants across 20 U.S. sites. The trial is designed to evaluate avexitide’s efficacy and safety in patients with PBH following Roux-en-Y gastric bypass surgery. Avexitide has already shown dose-dependent effects in five previous PBH clinical trials and has a favorable safety profile, positioning it as a potential first approved therapy for PBH.

LUMINA Trial for AMX0114: The company also began dosing participants in the Phase 1 LUMINA trial of AMX0114, an investigational antisense oligonucleotide targeting calpain-2, for ALS. The trial, which aims to evaluate the safety and biological activity of AMX0114, is expected to provide early data later in 2025. The trial will assess ALS biomarkers, including neurofilament light (NfL) levels.

Public Offering: In January 2025, Amylyx closed an underwritten public offering of 19.7 million shares, generating net proceeds of approximately $65.5 million, which will extend its cash runway through the end of 2026.

Upcoming Expected Milestones:
LUCIDITY Trial Enrollment Completion: Amylyx expects to complete enrollment in the Phase 3 LUCIDITY trial of avexitide in PBH by 2025, with topline data expected in the first half of 2026. If successful, commercial launch is anticipated in 2027.

AMX0035 Data Presentations: The company will present Week 48 data from the Phase 2 HELIOS trial of AMX0035 in Wolfram syndrome at the Joint Congress of the European Society for Pediatric Endocrinology and the European Society of Endocrinology in May 2025. Additionally, Amylyx plans to present data from the Phase 2 PREVENT and Phase 2b trials of avexitide at ENDO 2025 in July.

ORION Trial Data: An unblinded analysis of the Phase 2b portion of the ORION trial of AMX0035 for PSP is expected in the third quarter of 2025. This will inform the decision to proceed with the Phase 3 portion of the trial.

LUMINA Trial Data: Early cohort data from the Phase 1 LUMINA trial of AMX0114 in ALS is also expected in 2025, with sites opening in the U.S. for screening, enrollment, and dosing.

Financial Results for the First Quarter Ended March 31, 2025:
R&D Expenses: The company reported R&D expenses of $22.1 million for the first quarter of 2025, down from $36.6 million in the same period of 2024. This decrease was primarily due to reduced spending on AMX0035 for ALS, personnel-related costs, and a reduction in preclinical development activities.

SG&A Expenses: SG&A expenses for the first quarter of 2025 totaled $15.7 million, compared to $57.8 million in the same period of 2024. The decrease was due to lower payroll, personnel-related costs, and reduced consulting and professional services expenses.

Net Loss: Amylyx reported a net loss of $35.9 million, or $0.42 per share, for the first quarter of 2025, compared to a net loss of $118.8 million, or $1.75 per share, for the same period in 2024.

Cash Position: As of March 31, 2025, Amylyx’s cash, cash equivalents, and marketable securities totaled $204.1 million, up from $176.5 million at the end of 2024. The company expects its cash runway to extend through the end of 2026.

Investor Conference Call:
Amylyx will host a conference call on May 8, 2025, at 8:00 a.m. ET to discuss these financial results and provide an update on its business. Details for accessing the conference call are available on the company's website.

Source: https://www.amylyx.com/news/amylyx-pharmaceuticals-reports-first-quarter-2025-financial-results