May, 31 2023 • Articles

Nacuity Pharmaceuticals Achieves Target Enrollment for Phase 1/2 Clinical Trial of NPI-001 for the Treatment of Retinitis Pigmentosa Associated with Usher Syndrome

Phase 1/2 SLO-RP trial completed target enrollment of 48 patients diagnosed with RP associated with USH

Interim efficacy data anticipated by year end 2023

FORT WORTH, Texas, May 31, 2023 (GLOBE NEWSWIRE) -- Nacuity Pharmaceuticals, Inc., a clinical stage biopharmaceutical company developing treatments for retinitis pigmentosa, cataracts and other diseases caused by oxidative stress, today announced that target enrollment of 48 patients has been reached in the SLO-RP Phase 1/2 clinical trial of NPI-001 tablets in patients with retinitis pigmentosa (RP) associated with Usher syndrome (USH).

“We are pleased to reach our target enrollment, an important step toward our goal to realize the potential of NPI-001 for patients affected by RP,” said Halden Conner, Chairman, CEO and Co-Founder of Nacuity Pharmaceuticals. “We extend our gratitude to the principal investigators, Usher patient advocacy organizations and clinical sites who supported our recruitment efforts, working through challenges including the early days of a global pandemic. We are especially appreciative of the patients who have made this work possible. We look forward to sharing results from an interim analysis including efficacy data by year end 2023.”

The SLO-RP trial is a randomized, placebo-controlled, multicenter, double-masked, dose-escalation trial designed to assess the clinical safety, tolerability and efficacy of NPI-001 tablets versus placebo in patients with RP associated with USH. The trial enrolled 48 male and female patients, ages 18 years and older, at its four trial sites in Australia. Patients will be followed for two years in the core study to assess efficacy. The protocol has been amended to allow continued treatment beyond two years for interested participants while the trial is ongoing. To date, all eligible participants have elected to continue on treatment.

“The incredible compliance and enthusiasm of participants in the trial is a promising demonstration of the potential of NPI-001 for the treatment of RP,” said Jami Kern, Ph.D., Senior Vice President and Chief Clinical Officer of Nacuity. “We hope to confirm and build upon the encouraging preclinical and Phase 1 results to generate a robust package of data supporting the promise of an oral treatment option for patients with RP, while validating the potential for broader applications in other diseases caused by oxidative stress.”

For more information, visit (NCT04355689).

About NPI-001
NPI-001 is a proprietary, GMP-grade formulation of N-acetylcysteine amide (NACA) tablets being developed to target oxidative stress associated with blinding eye diseases, such as retinitis pigmentosa. Preclinical studies indicate that oral NPI-001 protects photoreceptor cones in the eye from oxidative damage by boosting glutathione, the body’s most powerful endogenous antioxidant, to stop chemically aggressive oxygen molecules from damaging eye tissue. NPI-001 and related small molecules have been shown to be potent antioxidants in several preclinical models that may lead to additional indications. NPI-001 has been granted orphan drug designation, which provides seven years of U.S. FDA regulatory exclusivity for the product upon regulatory approval.

About Nacuity Pharmaceuticals
Nacuity Pharmaceuticals is a clinical-stage leader in innovative treatments for oxidative stress. The company’s powerful, targeted therapies aim to stop oxidative tissue damage, a driver of blinding eye diseases and a broad spectrum of serious chronic conditions. Nacuity has three highly differentiated clinical programs ongoing in retinitis pigmentosa, cataracts and cystinosis with the potential to be first-of-a-kind therapies and gateways to wider applications. Based in Fort Worth, TX, the company has extensive managerial and scientific domain expertise as well as backing from Foundation Fighting Blindness ( and its venture arm RD Fund ( For more information, please visit

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6 Degrees PR