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Amylyx Pharmaceuticals, Inc.

Kent Leslie | Massachusetts, United States

Amylyx Pharmaceuticals, Inc.

Kent Leslie | Massachusetts, United States

Phase II Study to Assess Safety and Target Engagement in AMYX0035 in AD

Amylyx uses a combination therapy approach to both halt nerve cell death and block harmful inflammation in the brain and is now ready to test their promising combination therapy, AMYX0035, in human Alzheimer’s patients. Amylyx’s drug is a proprietary formulation of two compounds, Sodium Phenylbutyrate which targets “epigenetics” pathways or modifications of genes, and TUDCA, a bile acid that targets energetic stress within neurons. These 2 compounds work synergistically to protect brain cells from death, block the downstream toxic immune response and improve overall function in the brain. This clinical trial will be co-funded by the ADDF and the Alzheimer’s Association through a special joint effort and will include testing of safety and changes in disease-relevant brain imaging and spinal fluid biomarkers in 50 patients.