Nightstar Announces Planned Initiation of Phase 2/3 Expansion Study in XIRIUS Trial for NSR-RPGR in XLRP

Nightstar Therapeutics (NASDAQ:NITE), a clinical-stage gene therapy company developing treatments for rare inherited retinal diseases, provided an update on recent achievements and upcoming clinical milestones and reported financial results for the quarter ended September 30, 2018.

As quoted in the press release:

“We are pleased with the upcoming initiation of the Phase 2/3 expansion study in line with our previous guidance after being the first sponsor to establish proof-of-concept in X-linked retinitis pigmentosa,” said Dave Fellows, Chief Executive Officer. “The modified Phase 2/3 expansion study is designed to be consistent with recommendations in FDA’s draft guidance on the development of gene therapy products for retinal disorders, which we believe will allow us to expedite the development of NSR-RPGR.”

XIRIUS is a clinical trial consisting of an open-label, dose escalation study followed by an expansion study. Enrollment of the Phase 1 dose escalation study in the XIRIUS trial was completed in August 2018, consisting of six cohorts of three patients each for a total of 18 adult patients. On September 22, 2018, Nightstar announced positive preliminary safety and efficacy data from the XIRIUS trial for the first five cohorts (combined n=15) of the dose escalation study at the EURETINA medical meeting. Six-month follow-up data on all 18 patients in the dose escalation study is expected to be available in the second quarter of 2019, with one-year follow-up data expected to be available in the fourth quarter of 2019.

Click here to read the full press release.