Drug intelligence / Profile preview

C273

Development stage
Preclinical
Modality
Small Molecules
01

Overview

C273 is a novel small molecule being developed as a preclinical candidate for Alzheimer's Disease. It functions as a partial inhibitor of mitochondrial complex I (mtCI), a key enzyme in the electron transport chain. This weak inhibition leads to the activation of AMPK, which in turn triggers a neuroprotective stress response. This response encompasses enhanced oxidative stress protection, promotion of autophagy, and increased mitochondrial biogenesis and turnover, ultimately providing protection against amyloid-beta (Aβ) toxicity. Preclinical studies have demonstrated its efficacy in vivo, preserving cognition, improving long-term potentiation, reducing oxidative stress and inflammation, and enhancing mitochondrial biogenesis in mouse models of Alzheimer's Disease. C273 has also shown excellent bioavailability, blood–brain barrier penetrance, and a clean safety profile.

02

Targets

tRNA (Transfer RNA)AMPK (Adenosine monophosphate–activated protein kinase)ND1 (Mitochondrial electron transport chain complex I)

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