Key Takeaways

  • The first patient has been dosed in Nacuity Pharmaceuticals’ global, confirmatory phase 3 clinical trial of NPI-001 tablets for the treatment of RP associated with Usher Syndrome
  • NPI-001 is a proprietary, GMP-grade formulation of N-acetylcysteine amide (NACA) tablets being developed to address diseases driven by oxidative stress.
  • There are currently no approved therapies that slow photoreceptor degeneration in patients with RP associated with Usher syndrome

Nacuity Pharmaceuticals announced that the first patient has been dosed in the global, confirmatory phase 3 SLO-RP Preserve clinical trial of NPI-001 (N-acetylcysteine amide) tablets for the treatment of retinitis pigmentosa (RP) associated with Usher Syndrome.

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. The SLO-RP Preserve trial is designed to evaluate whether NPI-001 can slow disease progression by reducing photoreceptor loss.

The 24-month, randomized, double-masked study (NCT07710196) will enroll up to 80 adult participants who will self-administer an NPI-001 or a placebo oral tablet twice daily and complete nine in-clinic visits over approximately 2 years.

“Building on the encouraging findings observed to date, we look forward to further evaluating the potential of NPI-001 to slow disease progression and preserve vision in this phase 3 trial," said Mark Pennesi, MD, PhD, chief medical officer and director of the Inherited Retinal Degeneration Center at the Retina Foundation and principal investigator of the SLO-RP Preserve trial.

NPI-001 is a proprietary, GMP-grade formulation of N-acetylcysteine amide (NACA) tablets being developed to address diseases driven by oxidative stress.

“Dosing the first patient in our first phase 3 trial marks a significant milestone for Nacuity and, more importantly, for the Usher syndrome community,” said Halden Conner, chairman, CEO, and cofounder of Nacuity. “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.”

The SLO-RP Preserve trial is now enrolling patients at the Inherited Retinal Degeneration Clinic at the Retina Foundation of the Southwest in Dallas and other sites across the United States and Australia.