Drug intelligence / Profile preview

N-acetylcysteine amide

Development stage
Phase 3
Lead developer
Nacuity Pharmaceuticals
Modality
Small Molecules
Administration
Oral, Ophthalmic
01

Overview

N-acetylcysteine amide is the amide form of N-acetylcysteine (NAC), a synthetic derivative and prodrug of the endogenous amino acid and antioxidant glutathione precursor L-cysteine. It has enhanced lipophilicity and membrane permeability compared to NAC, which may improve its pharmacokinetic properties. Upon administration, it increases glutathione levels in cells, scavenges reactive oxygen species (ROS), reduces oxidative stress, and prevents ROS-mediated cell damage and apoptosis. Its primary mechanisms are antioxidant activity and free radical scavenging. It is being developed primarily for rare eye diseases such as retinitis pigmentosa, cystinosis, Usher syndromes, and familial cerebral amyloid angiopathy[1][2].

Brand names
NPI-001NPI001NPI 001
Other names
n-acetylcysteine amideacetylcysteine amide(R)-2-acetamido-3-mercaptopropanamidenac amide
02

Targets

GSH (Glutathione synthesis / redox)

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