nivegacetor

Nivegacetor (also known as RO7269162 and RG6289) is an orally bioavailable small molecule gamma-secretase modulator (GSM) developed by Roche for the treatment of Alzheimer's disease. Unlike traditional gamma-secretase inhibitors that broadly block the enzyme's activity and often lead to toxicity by interfering with Notch signaling, nivegacetor allosterically modulates the gamma-secretase enzyme to shift its cleavage of amyloid precursor protein (APP). This modulation results in a decrease in the production of longer, neurotoxic amyloid-beta peptides (specifically Aβ42 and Aβ40) and a reciprocal increase in shorter, non-aggregating, and less toxic peptides (Aβ37 and Aβ38). By altering the amyloid-beta profile, nivegacetor aims to prevent the formation of amyloid plaques and mitigate downstream neurodegeneration. It is currently being evaluated in Phase 2 clinical trials for patients with prodromal Alzheimer's disease or mild cognitive impairment.

Brand names

nivegacetor

Other names

nivegacetor