Presentations to Include Long-term Clinical Data
on NSR-REP1 and Natural History Data in Choroideremia
LEXINGTON, Mass. and LONDON, April 23, 2018 (GLOBE
NEWSWIRE) -- 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: PatrĂcio,
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, NSR-REP1, 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 www.nightstartx.com.
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 forward-looking 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
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.
Contacts:
Senthil Sundaram, Chief Financial Officer
Brian Luque, Sr. Manager, Investor Relations
investors@nightstartx.com
This
announcement is distributed by Nasdaq Corporate Solutions on behalf
of Nasdaq Corporate Solutions clients.
The issuer of this announcement warrants that they are solely
responsible for the content, accuracy and originality of the
information contained therein.
Source: Nightstar Therapeutics via Globenewswire
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