Amylyx Pharmaceuticals Announces Q3 2024 Financial Results

Amylyx Pharmaceuticals, Inc., based in Cambridge, Massachusetts, released its financial results for the third quarter ending September 30, 2024. The company made significant advancements in its late-stage pipeline, aiming to provide treatments for communities with unmet medical needs.

During this quarter, Amylyx shared positive preliminary data from its Phase 2 HELIOS clinical trial, which focused on individuals with Wolfram syndrome. The trial indicated that their treatment, AMX0035, showed notable improvements in various disease progression measures and maintained these improvements over time. The company is now planning to consult with the FDA and other stakeholders to shape their Phase 3 program for Wolfram syndrome. Additionally, Amylyx is preparing to launch a Phase 3 program for their key asset, avexitide, aimed at treating post-bariatric hypoglycemia (PBH) in the first quarter of the next year. Interim data from the ongoing Phase 2b/3 ORION trial of AMX0035 in progressive supranuclear palsy (PSP) is expected by mid-2025.

In the third quarter, Amylyx reported positive outcomes for all 12 participants in the HELIOS trial. The trial, which is an open-label and single-arm study, tested AMX0035 in adults with Wolfram syndrome. The results demonstrated improvements in pancreatic function, as indicated by the C-peptide response after 24 weeks of treatment. This contrasts with the usual decline in pancreatic function as the disease progresses. Other secondary endpoints, including hemoglobin A1c (HbA1c), glucose levels monitored continuously, and visual acuity, also showed overall improvements or stability. The safety profile of AMX0035 was consistent with previous data.

The company acquired avexitide, a glucagon-like peptide-1 (GLP-1) receptor antagonist ready for Phase 3 trials. Avexitide, which has received FDA Breakthrough Therapy and Orphan Drug designations for hyperinsulinemic hypoglycemia, has been tested in five clinical trials for PBH and three for congenital hyperinsulinism (HI). Previous studies showed that avexitide significantly reduced hypoglycemia events in PBH patients. The FDA has suggested that a reduction in hypoglycemia events could be a key endpoint for approval following positive Phase 3 trial results.

Amylyx also published promising data on AMX0035’s effects on cerebrospinal fluid biomarkers related to Alzheimer’s disease (AD) and neurodegeneration in a peer-reviewed journal. The Phase 2 PEGASUS trial’s exploratory analyses suggested that AMX0035 consistently altered biomarkers in AD patients, providing initial evidence of its impact on multiple neurodegenerative pathways, including tau proteins.

The company presented the Phase 1 study design for AMX0114, a new treatment for amyotrophic lateral sclerosis (ALS), at the 2024 Northeast ALS Consortium Annual Meeting. The LUMINA trial will be a multicenter, randomized, placebo-controlled study evaluating AMX0114’s safety and biological activity in approximately 48 ALS patients. Amylyx received clearance from Health Canada to start the trial, with plans to begin by the end of 2024 or early 2025. The FDA, however, has requested more information, resulting in a clinical hold, but Amylyx is working to address these concerns and may proceed with the trial outside the U.S. if necessary. Early cohort data from LUMINA is expected in 2025.

In terms of financial performance, Amylyx reported a net product revenue of $0.4 million for the three months ended September 30, 2024. This figure results from adjustments to the company’s gross-to-net accrual estimates for prior sales of RELYVRIO and ALBRIOZA. The company announced in April 2024 that it would discontinue marketing authorizations for these products based on results from the Phase 3 PHOENIX trial, which did not meet its primary and secondary endpoints.

Amylyx’s cost of sales for the third quarter was $0.8 million, associated with estimated losses on firm commitments under commercial manufacturing supply agreements for AMX0035, established before the PHOENIX trial results. Research and development expenses amounted to $21.2 million, a decrease from the previous year, attributed to reduced clinical expenses and payroll costs. Selling, general, and administrative expenses were $17.8 million, also lower than the previous year, due to decreased payroll and consulting costs following the restructuring plan announced in April 2024.

Ultimately, Amylyx reported a net loss of $72.7 million for the quarter. As of September 30, 2024, the company held $234.4 million in cash, cash equivalents, and marketable securities, expecting this to sustain operations into 2026.