Drug intelligence / Profile preview

NPI-001 + NPI-002

Development stage
Preclinical
Lead developer
Nacuity Pharmaceuticals
Modality
Small Molecules
Administration
Intravitreal, Implant
01

Overview

NPI-001 + NPI-002 (also known as NP-4) is a preclinical combination therapy program developed by Nacuity Pharmaceuticals for the treatment of Age-related Macular Degeneration (AMD) and Stargardt disease. The program consists of two small molecule antioxidants: NPI-001, a proprietary formulation of N-acetylcysteine amide (NACA), and NPI-002, which is di-N-acetylcysteine amide (diNACA). Delivered via a sustained-release intravitreal implant, the combination is designed to protect the retinal pigment epithelium (RPE) from oxidative stress, a primary driver of retinal degeneration. The mechanism of action involves boosting endogenous levels of glutathione, the body's most potent antioxidant, to neutralize chemically aggressive oxygen molecules and prevent cellular damage. Preclinical studies have demonstrated that both components protect retinal cells from oxidative stressors that mimic disease pathology, with the combination showing superior activity compared to N-acetylcysteine (NAC) alone.

Other names
N-acetylcysteine amide + di-N-acetylcysteine amide
02

Targets

Cys/CySS (Cystine/thiol-disulfide redox pool)

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