LEXINGTON, Mass. and LONDON, March
05, 2018 (GLOBE NEWSWIRE) -- 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
Officer
investors@nightstartx.com
Alicia Davis, THRUST IR
910-620-3302
alicia@thrustir.com