Synonyms: compound 41 [PMID: 34550687] | FRM024
Compound class:
Synthetic organic
Comment: FRM-024 is a CNS-penetrating γ secretase modulator that was developed for its capacity to improve amyloid processing and potential as an Alzheimer's disease therapeutic [1]. Gamma secretase modulators reduce the levels of the toxic longer amyloid β (Aβ) chains (Aβ42, Aβ43) that accummulate in Alzheimer's brains. They do not alter total Aβ levels. In particular, such modulators may afford potential for clinical intervention in patients with inherited loss-of-function mutations in presenilin genes which increase production of the toxic forms of Aβ.
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Bioactivity Comments |
FRM-024 reduces Aβ42 in H4 cells overexpressing WT human APP751 with an EC50 of 9 nM, and decreases Aβ42 by >50% in mouse and rat brains in vivo [1]. But note that in this primary report, FRM-024 was not tested against any mutant forms of presenilin enzymes that have been associated with familial Alzheimer's disease [1]. |