Nacuity Pharmaceuticals Announces Initiation of SLO-RP (Safety and Efficacy of NPI-001 Tablets versus PLacebO for Treatment of Retinitis Pigmentosa Associated with Usher Syndrome) Phase 1/2 Clinical Trial

NPI-001 Being Evaluated for the Treatment of Retinitis Pigmentosa in Patients with Usher Syndrome

FORT WORTH, TX, May 18, 2020 – Nacuity Pharmaceuticals, Inc., a clinical stage biotech company focused on the treatment of ocular diseases involving oxidative stress, today announced the initiation of a Phase 1/2 study of NPI-001 tablets in patients with retinitis pigmentosa (RP) associated with Usher syndrome (USH). The study, named SLO-RP, is designed to demonstrate greater retinal sensitivity in RP/USH patients treated with NPI-001 Tablets versus placebo. Previously, NPI-001 was evaluated in a Phase 1 study conducted in healthy volunteers, demonstrating its safety profile. The SLO-RP study will enroll patients diagnosed with RP associated with USH who are early in disease progression.

Preclinical studies indicate that oral NPI-001 protects photoreceptor cones in the eye from oxidative damage, which may preserve visual function.0 Furthermore, NPI-001 and related small molecules have been shown to be potent antioxidants in several preclinical models that may lead to additional indications.0 “Initiation of the SLO-RP trial is an important milestone in our efforts to understand the potential of this treatment for RP and a prelude to treating other conditions of the eye involving oxidative stress,” said G. Michael Wall, PhD, senior vice-president and chief scientific officer at Nacuity.

Nacuity Pharmaceuticals has partnered with the Foundation Fighting Blindness on a variety of initiatives related to the development of NPI-001 including funding through the Foundation’s venture arm the RD Fund, trial design, awareness and recruitment, and additional strategic guidance. “We are happy on behalf of the many patients around the globe who are affected by RP to see this promising treatment reach the clinic.” said Dr. Ben Yerxa, chief executive officer of Foundation Fighting Blindness, “We remain committed to seeing this potential treatment through to the next milestone.”

About the SLO-RP Trial

The randomized, placebo-controlled, multicenter, double-masked, dose escalation trial is designed to assess the clinical safety, tolerability and efficacy of NPI-001 Tablets versus placebo in patients with USH (NCT04355689). The Australian study will target enrollment of at least 48 male and female USH patients, ages 18 years and older, who will be followed for 2 years. The goals are to assess the safety of chronic dosing up to 500mg/day and generate clinical proof of concept for the treatment of RP. The key clinical endpoint is change from baseline in retinal sensitivity of patients treated with NPI-001 versus placebo.

Retinitis Pigmentosa

RP refers to a group of inherited diseases causing retinal degeneration and vision loss that worsen over time including USH, Leber congenital amaurosis, rod-cone disease, Bardet-Biedl syndrome, Refsumdisease, and others. The vision loss in USH is due to RP, presenting in the same manner. By showing a slowing of disease progression in the SLO-RP study, Nacuity aims to begin to address RP as a whole.

The National Eye Institute (NEI) estimates that approximately 100,000 people in the United States have RP. About 1.5 million people are afflicted worldwide. Night blindness is one of the earliest and most frequent symptoms. RP is a progressive disorder typically diagnosed in adolescents and young adults. The rate of progression and degree of visual loss varies by case, with the majority of patients legally blind by age 40. The only FDA-approved treatment for RP is LUXTURNA®, a gene therapy applicable for about 1% of RP patients (rpe65). (Trademarks are property of their owners.)

Nacuity Pharmaceuticals, Inc. ( is a clinical stage biotech company focused on advancing treatments for ocular conditions involving oxidative stress. Nacuity is funded by Foundation Fighting Blindness grants and direct investment, as well as private investors.

Foundation Fighting Blindness ( Established in 1971, the Foundation Fighting Blindness is the world’s leading private funding source for retinal degenerative disease research. The Foundation has raised more than $760 million toward its mission of accelerating research for preventing, treating, and curing blindness caused by the entire spectrum of retinal degenerative diseases including: retinitis pigmentosa, age-related macular degeneration, Usher syndrome, and Stargardt disease. Visit for more information.

Forward Looking Statements
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