Report of Foreign Issuer (6-k)
14 June 2018 - 9:37PM
Edgar (US Regulatory)
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM
6-K
REPORT OF
FOREIGN PRIVATE ISSUER
PURSUANT TO RULE
13a-16
OR
15d-16
UNDER THE SECURITIES EXCHANGE ACT OF 1934
For the Month of June 2018
Commission File Number:
001-38217
Nightstar Therapeutics plc
(Translation of registrants name into English)
215 Euston
Road
London NW1 2BE United Kingdom
(Address of principal executive office)
Indicate by check mark whether
the registrant files or will file annual reports under cover of Form
20-F
or Form
40-F:
☒ Form
20-F ☐ Form
40-F
Indicate by check mark if the registrant is submitting the Form
6-K
in paper as permitted by Regulation
S-T
Rule 101(b)(1): ☐
Indicate by check mark if the registrant is submitting the Form
6-K
in paper as permitted by Regulation
S-T
Rule 101(b)(7): ☐
Nightstar Therapeutics Receives Regenerative Medicine Advanced Therapy (RMAT) Designation for
NSR-REP1
in Choroideremia
On June 14, 2018, Nightstar Therapeutics plc issued a press release announcing
that it has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration for
NSR-REP1
in choroideremia. The press release is attached as Exhibit 99.1 and is
incorporated by reference herein.
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Exhibit
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Description
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99.1
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Press Release dated June 14, 2018.
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SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned,
thereunto duly authorized.
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NIGHTSTAR THERAPEUTICS PLC
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Date: June 14, 2018
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By:
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/s/ Bryan Yoon
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Name:
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Bryan Yoon
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Title:
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General Counsel and Secretary
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Exhibit 99.1
Nightstar Therapeutics Receives Regenerative Medicine Advanced Therapy (RMAT) Designation for
NSR-REP1
in Choroideremia
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First gene therapy RMAT designation for an inherited retinal disease
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STAR Phase 3 registrational trial ongoing and FDA interactions planned
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RMAT designation enables closer and more frequent multidisciplinary interaction with FDA with all of the benefits of breakthrough therapy designation
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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 companys 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. Nightstars 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. Nightstars 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 managements 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.
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Investors:
Senthil Sundaram, Chief Financial Officer
Brian Luque, Sr. Manager, Investor Relations
investors@nightstartx.com
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