First patient dosed in a global Phase 3 trial for a blinding disease with no therapy to slow it

Retinitis pigmentosa linked to Usher syndrome slowly takes away vision, and today there is no approved therapy that slows the loss. Nacuity Pharmaceuticals has dosed the first patient in SLO-RP Preserve, a global confirmatory Phase 3 trial of its tablet NPI-001, designed to protect light-sensing cells in the eye from oxidative stress — the chemical wear-and-tear implicated in the disease. The 24-month, double-masked study will enrol up to 80 adults at sites including the Retina Foundation of the Southwest in Dallas and centres in Australia; participants take the tablet or a placebo twice a day at home. The trial follows encouraging Phase 2 results. 'Our team is working diligently to move the study forward as quickly as possible, while maintaining our commitment to rigorous science and patient safety,' said chief clinical officer Jami Kern. For families watching vision fade with no options, a confirmatory trial is the step that decides whether hope becomes medicine.

SDG 3 · Good health and well-being — Mechanism: tech innovation