Amylyx Pharmaceuticals Announces Publication of Data Showing the Encouraging Effects of AMX0035 on Cerebrospinal Fluid Biomarkers of Core Alzheimer’s Disease Pathology and Neurodegeneration - Seite 2

The exploratory analyses showed that compared to placebo, AMX0035 reduced levels of p-tau181 and total tau. AMX0035 treatment also reduced levels of synaptic and neuronal degeneration biomarkers in the CSF, specifically neurogranin and FABP3, as well as YKL-40, a biomarker that has been shown to correlate with cortical volume loss and rate of cognitive decline. A 2023 publication showed AMX0035 reduced YKL-40 in ALS.

“These data lend further support to the preclinical and clinical evidence that AMX0035 has the potential to treat neurodegenerative diseases associated with tau dysfunction and tau aggregation. One such disease is progressive supranuclear palsy, also known as PSP. Our ORION trial studying AMX0035 in PSP remains ongoing,” commented Camille L. Bedrosian, MD, Chief Medical Officer at Amylyx. “There is a pressing unmet need for new and effective treatments in PSP, and we are encouraged by our findings that further support the potential of AMX0035.”

About AMX0035

AMX0035 is an investigational, oral, fixed-dose combination of sodium phenylbutyrate (PB) and taurursodiol (TURSO; also known as ursodoxicoltaurine outside of the U.S.). AMX0035 is designed to slow or mitigate neurodegeneration by targeting endoplasmic reticulum (ER) stress and mitochondrial dysfunction, two connected central pathways that lead to cell death and neurodegeneration. Preclinical studies have provided evidence that AMX0035 may reduce cell death and improve cellular function, also supporting the synergistic effect of AMX0035 compared to individual compounds. AMX0035 is being studied as a potential treatment in neurodegenerative diseases, including Wolfram syndrome and progressive supranuclear palsy (PSP).

About the PEGASUS Trial

PEGASUS (NCT03533257) was a randomized, double-blind, multi-center, placebo-controlled trial evaluating the safety, tolerability and activity of AMX0035 in 95 adults with mild cognitive impairment or mild to moderate dementia due to Alzheimer’s disease (AD) over 24 weeks of treatment. The trial was designed to evaluate safety and tolerability in this patient population while also assessing the effects of PB and TURSO on mechanistic targets of engagement and disease biology in a broad group of people with AD.