Amylyx Pharmaceuticals Announces Publication of Preclinical Data Showing Potential Synergistic Effect of AMX0035 Compared to Individual Compounds

Excerpt from the Press Release:

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”) today announced the publication of preclinical data showing the effect of sodium phenylbutyrate (PB) and taurursodiol (TURSO, also known as ursodoxicoltaurine) on the transcriptomic and metabolomic profiles of primary skin fibroblasts from adults with sporadic amyotrophic lateral sclerosis (ALS) and adults without ALS. These results, which showed that AMX0035 had a greater and distinct effect on genes and metabolites involved in ALS-relevant pathways as compared to either PB or TURSO alone,are published in the peer-reviewed medical journal, Annals of Clinical and Translational Neurology.

“ALS is a relentless and complex disease, and while AMX0035 has been shown to meaningfully slow the loss of function and extend survival in people living with ALS in a randomized, placebo-controlled clinical trial, this study is the first to explore the molecular effects of the combination of AMX0035 versus the individual compounds in ALS patient-derived cells,” said Dr. Hibiki Kawamata and Dr. Giovanni Manfredi, leading authors of the study from the Feil Family Brain & Mind Research Institute, Weill Cornell Medicine. “We are pleased to demonstrate that the combination has a greater and distinct impact as compared to the individual compounds on primary skin fibroblasts from people with sporadic ALS, which affects more than 90% of people living with ALS. These findings may underlie the benefits that AMX0035 has been shown to have on individuals living with ALS.”

Data analyses from twelve primary fibroblast lines from healthy donors and twelve lines derived from people with ALS demonstrated that treatment with AMX0035 changed more genes and metabolites than treatment with either PB or TURSO individually.

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