Nacuity Pharmaceuticals achieves target enrollment for Phase 1/2 clinical trial of NPI-001 for the treatment of retinitis pigmentosa associated with Usher syndrome

Nacuity Pharmaceuticals has announced that the target enrollment of 48 patients has been reached for the SLO-RP Phase 1/2 clinical trial of NPI-001 tablets in patients with retinitis pigmentosa (RP) associated with Usher syndrome (USH). The results of an interim analysis, including efficacy data, are expected to be released later this year.

NPI-001 is a patented GMP-qualified formulation of N-acetylcysteine amide (NACA) tablets, being developed to address oxidative stress associated with severe eye diseases such as retinitis pigmentosa. Preclinical studies indicate that oral administration of NPI-001 protects the photoreceptor cones in the eye from oxidative damage. This is achieved by stimulating glutathione, the most powerful endogenous antioxidant in the body, to prevent chemically aggressive oxygen molecules from damaging the eye tissue.

The achievement of the target enrollment for the clinical trial of NPI-001 is a significant milestone in the development of a potential treatment for retinitis pigmentosa associated with Usher syndrome. The aim of the study is to evaluate the efficacy and safety of NPI-001 in patients with this condition. The interim analysis of the results will provide important insights into the effectiveness of the drug.

For more information about the clinical trial and NPI-001, please visit the website of Nacuity Pharmaceuticals:

June 2023

Juni 2023