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Drug intelligence / Profile preview
ARN2966 (also known as 2-PMAP) is a brain-penetrant, orally active small molecule that acts as a post-transcriptional modulator of amyloid precursor protein (APP) expression. It selectively inhibits the translation of APP mRNA without affecting its transcription levels, thereby reducing the steady-state levels of full-length APP and its downstream cleavage products, including amyloid-beta (Aβ) peptides (Aβ40 and Aβ42). Originally developed by New York University and Aria Neurosciences for the treatment of Alzheimer's disease, ARN2966 has also been investigated for its potential to block the App-Cd74 signaling axis, which drives macrophage pyroptosis and renal inflammation in autosomal dominant tubulointerstitial kidney disease due to UMOD mutations (ADTKD-UMOD).
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