Nacuity Pharmaceuticals, Inc. announced it has reached target enrollment (48 patients) for its SLO-RP phase 1/2 clinical trial assessing NPI-001 tablets for the treatment of retinitis pigmentosa (RP) associated with Usher syndrome (USH).
Give me some background on the company.
Nacuity Pharmaceuticals is a clinical-stage pharmaceutical company focused on developing targeted therapies for oxidative stress.
Supported by Foundation Fighting Blindness and the RD (Retinal Degeneration) Fund, the company currently has several highly differentiated clinical programs for RP, cataract, and cystinosis.
Now talk about NPI-001.
Granted Orphan Drug status by the FDA, NPI-001 is a proprietary, Good Manufacturing Process (GMP) formulation of N-acetylcysteine amide (NACA) tablets designed to block oxidative stress associated with RP and other blinding eye diseases.
The tablet is developed to protect photoreceptor cones within the eye from oxidative damage via boosting glutathione (the strongest known endogenous antioxidant within the body) in order to prevent chemically-aggressive oxygen molecules from causing damage to ocular tissues.
Breakdown this SLO-RP study name.
The randomized, placebo-controlled, multicenter, double-masked, dose-escalation phase 1/2 trial (NCT04355689) is called the Safety and Efficacy of NPI-001 Tablets versus PLacebO for Treatment of Retinitis Pigmentosa Associated with Usher syndrome (SLO-RP).
And the actual study?
Divided into two phases, the trial is assessing the use of a 250 mg oral NPI-001 tablet twice a day (BID) compared to oral placebo tablets (BID) for 24 months in 48 patients (age 18+ years) diagnosed with RP associated with USH at four trial sites located in Australia.
Following the 24-month period, treatment will be extended for interested patients; according to the company, all eligible patients have chosen to continue.
What’s being measured?
Primary outcomes include the change from baseline for retinal sensitivity, as determined via microperimetry of active vs placebo, for 24 months.
This will also be evaluated for the extension period.
When can we expect results?
According to Halden Conner, chairman, CEO, and co-founder of Nacuity, the company is looking forward to “sharing results from an interim analysis including efficacy data by year end 2023.”
Per Clinical Trials, the study is expected to be completed by January 2025. Stay tuned for updates in the meantime!