Amylyx (AMLX) Q2 Net Loss Narrows 43%

Amylyx Pharmaceuticals (AMLX -4.40%), a biotechnology company focused on treatments for neurodegenerative and endocrine diseases, reported its second quarter 2025 financial results on August 7, 2025. The most notable news is that it recorded no product revenue, in line with expectations after discontinuing its commercial product early in 2024, while posting a GAAP net loss per share of ($0.46). This GAAP loss was slightly wider than the consensus estimate of ($0.44), missing by $0.02 per share. Research and development costs increased year-over-year, supporting its clinical pipeline. The quarter was defined by focused execution on advancing clinical trials and managing cash, reflecting both the opportunity of its late-stage pipeline and the risks that come with having no recurring revenue.

Source: Analyst estimates for the quarter provided by FactSet.

About Amylyx Pharmaceuticals and Its Focus

Amylyx Pharmaceuticals is dedicated to developing new treatments for rare and serious conditions, particularly in neurodegeneration and endocrinology. Its core pipeline includes a late-stage trial in post-bariatric hypoglycemia (PBH), ongoing studies in Wolfram syndrome and progressive supranuclear palsy (PSP), and an early-stage trial for amyotrophic lateral sclerosis (ALS).

The company’s recent strategy has centered on expanding its lead asset pipeline while controlling costs. Its success relies on achieving clinical milestones in current trials, securing regulatory designations like Breakthrough Therapy or Orphan Drug status, and ultimately gaining approvals for its therapies. Intellectual property protection and targeted partnerships are also important to support its clinical advances and eventual market entry.

Quarter in Review: Developments and Financial Performance

The company recorded no product revenue (GAAP), which matched expectations. This is due to the decision to fully discontinue sales of RELYVRIO/ALBRIOZA earlier in 2024. With no sales coming in, the company continued investing in its clinical programs.

Research and development costs (GAAP) rose to $27.2 million, up 16.7% compared to Q2 2024, reflecting investment in ongoing trials. Spending was directed at the pivotal Phase 3 LUCIDITY trial for avexitide, a GLP-1 receptor antagonist designed as an injectable treatment for post-bariatric hypoglycemia. The trial’s recruitment is expected to finish in 2025, with major results forecast for the first half of 2026. Positive Phase 2b data for avexitide presented at ENDO 2025 showed a statistically significant 64% reduction in hypoglycemic episodes. The drug also retains Breakthrough, Orphan Drug, and Rare Pediatric Disease designations, which offer development advantages.

AMX0035, an oral combination therapy targeting cellular stress, progressed in trials for Wolfram syndrome and for PSP. Data from the 48-week open-label HELIOS study suggested continuing positive results for glycemic control and other measures in Wolfram syndrome patients. The company anticipates a go/no-go decision on Phase 3 for PSP in the second half of 2025, following review of unblinded data.

AMX0114, an antisense oligonucleotide in development for ALS, gained Fast Track designation from the U.S. Food and Drug Administration in June. The Phase 1 LUMINA study, which tests safety and early effects in people with ALS, started dosing with early data expected by year-end.

General and administrative costs (GAAP) dropped by approximately 72.8% year-over-year in Q2 2025, attributed to decreases in payroll, personnel-related costs, consulting, professional services, and other expenses. Stock-based compensation was lower across both major expense categories. The net loss (GAAP) narrowed by 43% from the prior year, although R&D costs are expected to stay elevated as clinical programs advance. The prior-year quarter included one-time restructuring costs, making the year-over-year comparison more favorable in this period.

Business Model, Products, and Strategic Dynamics

The company’s future is tied to the performance of its clinical-stage therapies. Avexitide, its injectable for PBH, targets roughly 160,000 patients in the U.S.—a population with few existing treatment options—based on the number of people who have undergone the two most common types of bariatric surgery: sleeve gastrectomy and Roux-en-Y gastric bypass. The product could be first to market if successful but faces the challenges typical for rare disease drugs, including high education needs for prescribers and patients.

AMX0035 is being evaluated for two rare neurological conditions: Wolfram syndrome, which affects about 3,000 people in the U.S, and PSP, which affects approximately seven in 100,000 people worldwide. Program progress in these indications is critical, with results expected in the next two to three quarters. AMX0114, if successful in early human studies, would move the company back toward the ALS market, which it previously served with its now-discontinued commercial product. No new partnerships or collaborations were announced in the quarter, though Amylyx continues work with existing research partners and contract manufacturers.

Outlook and Upcoming Milestones

Amylyx ended the quarter with $180.8 million in cash, cash equivalents, and marketable securities. Management expects this will last through the end of 2026. There was no corporate financial guidance for revenue or earnings provided. Management instead highlighted the ongoing cash runway and the timing of upcoming trial milestones.

Investors should focus on several key events in the coming year. The LUCIDITY trial for avexitide is expected to complete enrollment in 2025. Results are anticipated in the first half of 2026. The next twelve months will be critical as success or setbacks in these trials will determine the next phase for the company's pipeline and financial viability. AMLX does not currently pay a dividend.

Revenue and net income presented using U.S. generally accepted accounting principles (GAAP) unless otherwise noted.