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Sigma1R ligand (including lead candidates EPGN2544, EPGN2665, and EPGN644) is a program of selective small-molecule ligands targeting the sigma-1 receptor (S1R), developed by Epigen Biosciences (formerly Epigen Pharmaceuticals) for the treatment of neurodegenerative and cognitive disorders, including Alzheimer's disease, amyotrophic lateral sclerosis (ALS), and Fragile X syndrome. S1R is an endoplasmic reticulum chaperone protein that plays a critical modulatory role in neurodegeneration, cell survival, and cognitive function. Activation of S1R by these ligands has been shown to improve cognition, promote cell survival, and facilitate the release of the neuroprotectant brain-derived neurotrophic factor (BDNF). Preclinical studies of EPGN644 demonstrated efficacy in mouse models of Alzheimer's disease upon oral administration, while optimized candidates EPGN2544 and EPGN2665 were developed with superior overall profiles and novel composition of matter claims for further development in ALS and other indications.
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