Nacuity Pharmaceuticals announced that the first patient has been dosed in the global, confirmatory phase 3 SLO-RP Preserve clinical trial of NPI-001 for the treatment of retinitis pigmentosa (RP) associated with Usher syndrome. The Inherited Retinal Degeneration Clinic at the Retina Foundation of the Southwest in Dallas, and other sites in the United States and Australia are actively enrolling additional patients, the company said in a press release.

RP associated with Usher syndrome is a rare, progressive inherited retinal disease that leads to irreversible vision loss and currently has no approved therapies that slow photoreceptor degeneration. According to the company, SLO-RP Preserve is designed to evaluate whether NPI-001 can slow disease progression by reducing photoreceptor loss. The 24-month, randomized, double-masked study will enroll up to 80 adult participants who will self-administer an NPI-001 or a placebo tablet twice a day orally and complete nine in-clinic visits over approximately two years.

According to the company, NPI-001 is a proprietary formulation of N-acetylcysteine amide tablets being developed to target oxidative stress associated with diseases such as RP. Preclinical studies suggest that NPI-001 increases glutathione to stop chemically aggressive oxygen molecules from damaging retinal cells.

NPI-001 has also received Fast Track, Breakthrough Therapy, and Orphan Drug designations for RP. If approved, the treatment may qualify for 7 years of orphan drug exclusivity in the United States, subject to applicable regulatory requirements. RP