Nightstar Therapeutics Receives Regenerative Medicine Advanced Therapy (RMAT) Designation for NSR-REP1 in Choroideremia
June 14 2018 - 6:05AM
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
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