Amylyx Pharmaceuticals Announces Publication of Data Showing the Encouraging Effects of AMX0035 on Cerebrospinal Fluid Biomarkers of Core Alzheimer’s Disease Pathology and Neurodegeneration

Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today announced the publication of exploratory analyses on cerebrospinal fluid (CSF) biomarkers from participants with Alzheimer’s disease (AD) from the Phase 2 PEGASUS trial. Data analyses suggest that treatment with AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO]) resulted in consistent changes in AD and neurodegeneration CSF biomarkers in participants with a broad range of disease severity. The results were published in the peer-reviewed medical journal Alzheimer’s & Dementia: Translational Research & Clinical Interventions, a journal of the Alzheimer’s Association.

“Alzheimer’s disease is defined by amyloid plaques and tau tangles, but it’s now understood that these pathologies are accompanied by alterations in multiple cell and molecular pathways, including neuronal dysfunction, neurodegeneration, and oxidative stress, driving the progression of this relentless disease,” said Steven E. Arnold, MD, Professor of Neurology at Harvard Medical School and Translational Neurology Head and Managing Director of the Interdisciplinary Brain Center and the inaugural E. Gerald Corrigan, PhD Endowed Chair at Massachusetts General Hospital. “The results from this exploratory analysis suggest that AMX0035 engages important pathways implicated in the pathogenesis of Alzheimer’s disease and other neurodegenerative diseases.”

Of the 95 participants in the intent-to-treat (ITT) cohort of the PEGASUS trial, 67 had CSF samples at baseline and Week 24. Within this CSF subcohort, treatment effects were analyzed for biomarkers spanning multiple pathophysiological processes in AD. These biomarkers included core AD biomarkers, such as amyloid beta 42/40 (Aβ42/40) ratio, phosphorylated tau181 (p-tau181) and total tau; biomarkers reflecting synaptic and neuronal degeneration, including neurogranin and fatty acid binding protein-3 (FABP3); biomarkers associated with gliosis, including YKL-40 (also known as chitinase 3-like protein 1); the oxidative stress marker 8-hydroxy-2-deoxyguanosine (8-OHdG); and additional biomarkers associated with neurodegeneration, inflammation, and metabolism.