Nightstar Therapeutics Receives Regenerative Medicine Advanced Therapy (RMAT) Designation for NSR-REP1 in Choroideremia
14 June 2018 - 8:05PM
- First gene therapy RMAT
designation for an inherited retinal disease
- STAR Phase 3 registrational
trial ongoing and FDA interactions planned
- RMAT designation enables closer
and more frequent multidisciplinary interaction with FDA with all
of the benefits of breakthrough therapy designation
WALTHAM, Mass. and LONDON, June 14, 2018 (GLOBE
NEWSWIRE) -- Nightstar Therapeutics plc (NASDAQ:NITE), a
clinical-stage gene therapy company developing treatments for rare
inherited retinal diseases, today announced that the U.S. Food and
Drug Administration (FDA) has granted Regenerative Medicine
Advanced Therapy (RMAT) designation to NSR-REP1, the company's lead
product candidate currently in Phase 3 development for the
treatment of choroideremia, a rare, degenerative, genetic retinal
disorder that leads to blindness.
"Receiving RMAT designation for NSR-REP1
highlights the potential of this gene therapy to maintain and
improve visual acuity in choroideremia," said Dave Fellows, Chief
Executive Officer. "This designation further underscores a
recognition of the serious nature of choroideremia and the urgent
need to develop new treatments for those affected by inherited
retinal diseases that would otherwise lead to blindness. We look
forward to working closely with the FDA to discuss the NSR-REP1
development program and to determine how we can accelerate the
pathway for making NSR-REP1 available to choroideremia
patients."
Established under the 21st Century Cures Act, the
RMAT designation is an expedited program for the advancement and
approval of regenerative medicine products. A regenerative
medicine is eligible for the designation if it is intended to
treat, modify, reverse or cure a serious or life-threatening
disease or condition, and preliminary clinical evidence indicates
that the drug has the potential to address unmet medical needs for
such a disease or condition. RMAT allows companies developing
regenerative medicine and gene therapies to work more closely and
frequently with the FDA, and grants all of the benefits of
Breakthrough Therapy Designation, including eligibility for
priority review, rolling review and accelerated approval. In
November 2017, the FDA expanded the RMAT designation to include
gene therapies.
RMAT designation for NSR-REP1 was based on
clinical data supporting the maintenance and improvement of visual
acuity from completed Phase 1/2 trials in choroideremia patients
treated with NSR-REP1 and disease progression in untreated patients
in the ongoing NIGHT natural history observational study.
About
Choroideremia
CHM is a rare, degenerative, X-linked genetic
retinal disorder primarily affecting males, with no treatments
currently available and represents a significant unmet medical
need. CHM presents in childhood as night blindness, followed by
progressive constriction of the visual fields, generally leading to
vision loss in early adulthood and total blindness thereafter. CHM
is caused by mutations in the CHM gene, which encodes REP1, a
protein that plays a key role in intracellular protein trafficking
and the elimination of waste products from retinal cells. Absence
of functional REP1 leads to death of the RPE cells and degeneration
of the overlying retina, which contains the retinal photoreceptors
required to convert light into visual signals. Thus, the loss of
REP1 function in retinal cells caused by CHM results in progressive
vision loss and blindness.
About Nightstar and
NSR-REP1
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
comprised of an AAV2 vector containing recombinant human
complementary DNA, or cDNA, that is designed to produce REP1 inside
the eye. 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
Phase 1/2 trials of NSR-REP1 were published in The
Lancet in 2014 and in The New England Journal of
Medicine in 2016. In data from 32 patients treated with
NSR-REP1 across four open-label Phase 1/2 clinical trials, over 90%
of treated patients maintained their visual acuity over a two-year
follow-up period. In some cases, substantial improvements in visual
acuity were also observed. Nightstar's second product
candidate, NSR-RPGR, is currently being evaluated in a clinical
trial known as the XIRIUS 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 our planned and ongoing clinical
trials for NSR-REP1, including our Phase 3 STAR trial in
choroideremia, 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 of data
from ongoing clinical trials and studies, whether the receipt of
regenerative medicines advanced therapy designation for NSR-REP1 in
choroideremia will meaningfully impact the development and review
of NSR-REP1 by the FDA or the likelihood that the product candidate
will be found safe and effective, 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" section of 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.
Investors:
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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