Nightstar Therapeutics To Present New Data in Choroideremia at the ARVO 2018 Annual Meeting

Presentations to Include Long-term Clinical Data on NSR-REP1 and Natural History Data in Choroideremia.


Nightstar Therapeutics plc (NASDAQ: NITE), a clinical-stage gene therapy company developing treatments for rare inherited retinal diseases, today announced that one oral presentation and four posters have been selected for presentation at the Association for Research in Vision and Ophthalmology (ARVO) 2018 Annual Meeting taking place April 29 to May 3, 2018, in Honolulu, HI. The presentations include clinical and nonclinical data on Nightstar’s lead product candidate, NSR-REP1, which is in Phase 3 clinical development for choroideremia, as well as from the existing Nightstar-sponsored natural history observational study (NIGHT study). Presentation abstracts can be found on the ARVO 2018 website. Updated and detailed results from these studies will be presented at the conference.

“We are pleased to be presenting such a comprehensive body of work related to our Phase 3 program for NSR-REP1 in choroideremia at the ARVO Annual Meeting,” said Dave Fellows, Chief Executive Officer of Nightstar.

“The breadth of data reflects our continued commitment to patients with inherited retinal diseases that lead to blindness, and we are excited to discuss our progress at this important medical meeting. We are also looking forward to presenting details from our Phase 1/2 trials and NIGHT natural history study in choroideremia later this year.”

Oral Presentation

Retinal Gene Therapy for Choroideremia in a Multicenter Dose Escalation Phase I/II Clinical Trial
Author: MacLaren, Oxford Eye Hospital, University of Oxford
Presentation #: 1195
Session # / Title: 209, Gene Therapy
Session Date/Times: April 30, 2018 from 9:30 AM to 9:45 AM

Poster Presentations

Natural History of Progression of Choroideremia (NIGHT) Study: Longitudinal Changes in Visual Acuity over 12 Months
Author: Pennesi, Casey Eye Institute
Poster #: 3898 - C0364
Session # / Title: 382, Profound Low Vision and Low-vision Clinical Trials
Session Date/Times: May 1, 2018 from 3:30 PM to 5:15 PM

Natural History of Progression of Choroideremia (NIGHT) Study: Cross-Sectional Analysis of Baseline Characteristics
Author: Lam, Bascom Palmer Eye Institute
Poster #: 3899 - C0365
Session # / Title: 382, Profound Low Vision and Low-vision Clinical Trials
Session Date/Times: May 1, 2018 from 3:30 PM to 5:15 PM

Biocompatibility and stability of an AAV vector for choroideremia gene therapy following passage through its surgical device
Author: Patrício, Oxford Eye Hospital, University of Oxford
Poster #: 4541 - A0056
Session # / Title: 442, Ocular gene therapies and chemical therapeutics
Session Date/Times: May 2, 2018 from 11:15 AM to 1:00 PM

Vector shedding and immunology results from a gene therapy clinical trial for choroideremia
Author: Barnard, Oxford Eye Hospital, University of Oxford
Poster #: 5654 - A0373
Session # / Title: 515, Gene therapy, implants
Session Date/Times: May 3, 2018 from 8:15 AM to 10:00 AM

About NSR-REP1 Gene Therapy

NSR-REP1 is comprised of an AAV2 vector containing recombinant human complementary DNA, or cDNA, that is designed to produce REP1 inside the eye. NSR-REP1 is administered surgically by injection into the sub-retinal space, which is between the outer layers of the retina. The introduction of a functional CHM gene into patients is intended to allow expression of REP1, thereby slowing or stopping the progression of CHM and the decline in vision. In addition, Nightstar believes that enhanced REP1 expression may also be able to slow or reverse the early stages of cell death in already damaged retinal cells, accounting for the substantial improvements in visual acuity that have been observed in some patients after treatment with NSR-REP1. Nightstar has received orphan drug designation for NSR-REP1 for the treatment of CHM from the U.S. Food and Drug Administration, or the FDA, in the United States and from the European Medicines Agency, or the EMA, in the European Union. The safety and efficacy of NSR-REP1 has not yet been established.

About Nightstar

Nightstar is a leading clinical-stage gene therapy company focused on developing and commercializing novel one-time treatments for patients suffering from rare inherited retinal diseases that would otherwise progress to blindness. Nightstar’s lead product candidate, NSRREP1, is currently in Phase 3 development for the treatment of patients with choroideremia, a rare, degenerative, genetic retinal disorder that has no current treatments and affects approximately one in every 50,000 people. Positive results from a Phase 1/2 trial of NSR-REP1 were published in The Lancet in 2014 and in The New England Journal of Medicine in 2016. Nightstar’s second product candidate, NSR-RPGR, is currently being evaluated in a Phase 1/2 clinical trial for the treatment of patients with X-linked retinitis pigmentosa, an inherited X-linked recessive retinal disease that affects approximately one in every 40,000 people. For more information about Nightstar or its clinical trials, please visit

Cautionary Language Concerning Forward-Looking Statements

This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. The words “believe,” “anticipate,” “intend,” “estimate,” “will,” “may,” “should,” “expect” or other similar expressions are intended to identify forward- looking statements, although not all forward-looking statements contain these identifying words. All statements contained in this press release other than statements of historical facts are forwardlooking statements, including, without limitation: statements about the potential safety and efficacy of our product candidates, including NSR-REP1, our planned clinical trials for NSR-REP1, including our STAR Phase 3 trial in choroideremia and the expansion of future clinical trials, the continued clinical development of our pipeline, the timelines associated with our research and development programs including the timing of patient enrollment and the release data from ongoing clinical trials and studies, the prevalence of patient populations for our targeted indications, and the utility of prior preclinical and clinical data in determining future clinical results. These forward-looking statements are based on management's current expectations of future events and are subject to a number of involve substantial known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to be materially different from the information expressed or implied by these forward-looking statements, including the risks and uncertainties set forth in Item 3.D. "Risk Factors" in our Annual Report on Form 20-F for the year ended December 31, 2017, and subsequent reports that we file with the U.S. Securities and Exchange Commission. We may not actually achieve the plans, intentions, estimates or expectations disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. Actual results or events could differ materially from the plans, intentions, estimates and expectations disclosed in the forward-looking statements we make. We anticipate that subsequent events and developments will cause our views to change. We are under no duty to update any of these forward-looking statements after the date of this press release to conform these statements to actual results or revised expectations, except as required by law. You should, therefore, not rely on these forward-looking statements as representing our views as of any date subsequent to the date of this press release.


Senthil Sundaram, Chief Financial Officer
Brian Luque, Sr. Manager, Investor Relations

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