Nightstar Therapeutics plc (NASDAQ:NITE), a clinical-stage gene
therapy company developing treatments for rare inherited retinal
diseases, today announced the initiation of the company’s STAR
Phase 3 registrational trial to study the safety and efficacy of
NSR-REP1 in patients with choroideremia. In data from 32 patients
treated with NSR-REP1 across four open-label Phase 1/2 clinical
trials, over 90% of treated patients maintained or improved their
visual acuity over a one-year follow-up period.
The STAR trial is expected to enroll approximately 140 patients
across 18 clinical sites in the United States, Europe, Canada and
South America, of which six sites will be surgical centers.
Eligible patients will be randomized into one of three study arms:
56 patients receiving a high-dose of NSR-REP1 in one-eye (1.0 ×
10^11 genome particles, or gp); 28 patients receiving a low-dose of
NSR-REP1 in one-eye (1.0 × 10^10 gp); and 56 patients receiving no
treatment (no-sham, parallel control arm). Patients in the STAR
trial are expected to be recruited primarily from the existing
Nightstar-sponsored natural history observational study (NIGHT
study) in order to accelerate Phase 3 enrollment from this
well-characterized patient population. The primary endpoint of the
STAR trial is the proportion of patients with an improvement of at
least 15 ETDRS letters from baseline in visual acuity at 12 months
post-treatment. The primary endpoint will compare patients in the
high-dose treatment arm with patients in the control arm.
“The initiation of this first-ever Phase 3 trial for the
treatment of choroideremia is a major milestone for Nightstar and a
tremendous step forward for patients otherwise at risk of blindness
due to this devastating disease,” said Dave Fellows, chief
executive officer of Nightstar. “We are very encouraged by the
responses we have seen to-date following treatment with NSR-REP1.
This accomplishment demonstrates our team’s ability to successfully
advance important gene therapies. We are thankful to our academic
and advocacy partners, as well as the many patients who have
participated in our studies, all of whom have been instrumental in
helping us to achieve this milestone.”
“The Choroideremia Research Foundation is encouraged by the
advancement of this gene therapy and congratulates the Nightstar
team for their unrelenting commitment to serving patients,” said
Randy Wheelock, chief advisor for research and therapy development
for the Choroideremia Research Foundation (CRF,
http://curechm.org/).
Dr. Christopher Moen, president of the CRF commented, “Not only
is this important for choroideremia patients and their families,
but it is another important step toward developing therapies for
the many people affected by blinding inherited retinal diseases, of
which over 200 have been identified. The CRF is proud of its
contributions in helping Nightstar achieve this milestone,
including grants for initial research and preclinical studies. We
look forward to realizing the full potential NSR-REP1 could have
for patients with this challenging condition.”
About Choroideremia
Choroideremia, or 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.
Patients generally maintain good visual acuity until the
degeneration encroaches onto the fovea, or the central part of the
retina responsible for detailed vision. CHM is a degenerative
disease that, starting at an early age, affects the retinal pigment
epithelium, or RPE, which provides supportive biological functions
for the photoreceptors and the underlying choroid, or outer retinal
blood supply. Without a properly functioning RPE, the
photoreceptors and the choroid slowly begin to atrophy, leading to
vision loss. For CHM patients, it is often in middle age, when
people typically are at or near their peak productive years, that
visual impairment begins to limit independent activities of daily
living and working productivity, generally leading to vision loss
and total blindness thereafter. The prevalence of CHM is estimated
to be one in 50,000 people, implying a total population of
approximately 13,000 patients in the United States and the five
major European markets.
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 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.
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.
Forward-Looking Statements
This press release contains “forward-looking statements” within
the meaning of the Private Securities Litigation Reform Act of
1995, including, but not limited to: statements about our plans to
develop and commercialize our product candidates, our STAR trial
and other planned clinical trials, the clinical relevance and
utility of the endpoints to be studied in the STAR trial, 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 the "Risk Factors" section of our prospectus filed
pursuant to Rule 424(b)(4) under the U.S. Securities Act of 1933,
as amended, on September 28, 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. The forward-looking statements
in this press release represent our views as of the date hereof. We
anticipate that subsequent events and developments will cause our
views to change. However, while we may elect to update these
forward-looking statements at some point in the future, we have no
current intention of doing so except to the extent required by
applicable 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.
Contact:Senthil Sundaram, Chief Financial
Officerinvestors@nightstartx.com
Alicia Davis, THRUST IR910-620-3302alicia@thrustir.com
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