Nightstar’s mission is to maintain and restore vision in blinding inherited retinal diseases. To that end, the company has seven retinal programs under license. Its Phase III STAR registration trial on NSR-REP1 for choroideremia is slated to begin during the first half of 2018. The Phase I/II trials showed a “durable and robust signal,” CEO David Fellows said, with some patients out to 5 years. Each of the disease states Nightstar is concentrating on affect between 10,000 and 20,000 people between Europe and the US. The STAR pivotal trial will deliver the therapy through a transvitreal subretinal procedure using optical coherence tomography to guide surgical placement. Because a maintenance trial would be lengthy, the company is looking at “hyperresponders,” those who have gained 15 letters, as an FDA-accepted endpoint. In the Phase I/I studies, 4 of 19 patients (21%) who had 20/40 to 20/200 baseline vision were able to show and maintain vision gains of 15 letters at 12 months, Fellows stated. Those results are comparable to approved anti-VEGF therapies for wet age-related macular degeneration. NSR-REP1 has completed enrollment in the Phase II REGENERATE study, and the GEMINI study to evaluated safety/efficacy of bilateral treatment is expected to start by the end of 2017. Other programs are investigating treatments for X-linked retinitis pigmentosa, in Phase I/II with preliminary data expected in 2018; best vitelliform macular dystrophy, with a Phase I/I trial to start in 2019; and Stargardt disease, which is still in preclinical trials.

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