Amylyx Pharmaceuticals Announces Publication of Preclinical Data Showing Potential Synergistic Effect of AMX0035 Compared to Individual Compounds
“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.
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. The majority of changes in genes and metabolites were unique to treatment with AMX0035 versus PB or TURSO alone, suggesting that the combination of PB and TURSO has certain distinct effects that are not exclusively additive effects of PB and TURSO. Further, most changes affected the expression of genes involved in ALS-relevant pathways, including nucleocytoplasmic transport, unfolded protein response, mitochondrial function, RNA metabolism, and innate immunity. These studies add new learnings regarding the mechanistic activity of PB and TURSO and supplement our preclinical work that has shown synergistic reduction in neuronal death through the hypothesized mechanism of combined inhibition of endoplasmic reticulum and mitochondrial stress.
AMX0035 (sodium phenylbutyrate and taurursodiol) is an oral fixed-dose medication approved with conditions as ALBRIOZA™ to treat amyotrophic lateral sclerosis (ALS) in
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding beliefs about the preclinical and clinical study analyses of AMX0035, the potential benefits of AMX0035 on individuals living with ALS, the Company’s efforts to advance potential regulatory approval of AMX0035 and the potential of AMX0035 or other future therapeutic candidates as a treatment for ALS and other neurodegenerative diseases. Any forward-looking statements in this press release are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. Risks that contribute to the uncertain nature of the forward-looking statements include: the success, cost, and timing of Amylyx’ program development activities, Amylyx’ ability to execute on its strategy, regulatory developments, expectations regarding the timing of regulatory review of AMX0035 and the inherent unpredictability of the regulatory review process, Amylyx’ ability to fund operations, and the impact that the ongoing COVID-19 pandemic will have on Amylyx’ operations, as well as those risks and uncertainties set forth in Amylyx’