According to the company, the Phase 1/2 SLO-RP trial completed target enrollment of 48 patients diagnosed with RP associated with USH, with interim efficacy data anticipated by the end of the year.
Nacuity Pharmaceuticals Inc. announced target enrollment of 48 patients has been reached in the SLO-RP Phase 1/2 clinical trial (NCT04355689) of NPI-001 tablets in patients with retinitis pigmentosa (RP) associated with Usher syndrome (USH).
Halden Conner, chairman, CEO and co-founder of Nacuity Pharmaceuticals, said the company was pleased to reach its target enrollment, an important step toward its goal to realize the potential of NPI-001 for patients affected by RP.
“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,” Conner said in a news release. “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.”
According to the company, 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, PhD, 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.”