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, 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
OfficerBrian Luque, Sr. Manager, Investor
Relationsinvestors@nightstartx.com
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