Nacuity Pharmaceuticals has dosed the first patient in its global confirmatory Phase III SLO-RP Preserve study, evaluating NPI-001 for retinitis pigmentosa (RP) associated with Usher syndrome.
The study will assess whether the oral treatment can reduce photoreceptor loss and slow the progression of the rare, progressive inherited retinal condition, which causes irreversible sight loss and lacks approved treatments to slow degeneration.
The double-masked, 24-month, randomised trial plans to recruit up to 80 adult patients.
They will receive either an NPI-001 or placebo tablet orally twice daily and attend nine clinical visits across a two-year timeframe.
Recruitment is under way in Australia and the US, with active sites including the Inherited Retinal Degeneration Clinic at the Retina Foundation of the Southwest in Dallas, Texas.
Nacuity chairman, CEO and co-founder Halden Conner said: “Dosing the first patient in our first Phase III trial marks a significant milestone for Nacuity and, more importantly, for the Usher syndrome community.
“We look forward to advancing NPI-001 through this confirmatory study as we work toward delivering a potential disease-modifying therapy for patients with RP associated with Usher syndrome.
“We’re grateful for our continued collaboration with the Retina Foundation of the Southwest and to the investigators, patients and families making this study possible.”
Formulated as an oral tablet manufactured to good manufacturing practice (GMP) standards, NPI-001 delivers N-acetylcysteine amide (NACA) to treat illnesses triggered by oxidative stress.
The therapy holds fast track, breakthrough therapy, and orphan drug designations from the US Food and Drug Administration for RP, the last of which grants seven years of regulatory exclusivity in the US following potential regulatory approval.
Beyond RP, Nacuity operates clinical programmes focusing on cataract and cystinosis.
