Indicate by check mark whether
the registrant files or will file annual reports under cover of Form
20-F
or Form
40-F:
On June 6, 2018, Nightstar Therapeutics plc issued a press release announcing financial results for quarter ended March 31, 2018. The press release
is attached as Exhibit 99.1 hereto and is incorporated by reference herein.
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 6, 2018
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By:
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/s/ Bryan Yoon
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Name: Bryan Yoon
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Title: General Counsel and Secretary
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Exhibit 99.1
Nightstar Therapeutics Reports First Quarter 2018 Financial Results and Acceleration of Preliminary
NSR-RPGR
Readout to Third Quarter 2018
Preliminary data from dose escalation study in the Phase
1/2 XIRIUS trial for
X-Linked
Retinitis Pigmentosa expected ahead of schedule in Q3 2018
Dose expansion study in the XIRIUS trial expected to begin enrollment in Q4 2018
WALTHAM, Mass. and LONDON June
6, 2018
(GLOBE NEWSWIRE)
Nightstar Therapeutics plc (NASDAQ: NITE), a
clinical-stage gene therapy company developing treatments for rare inherited retinal diseases, today reported financial results for the quarter ended March 31, 2018 and provided an update on recent achievements and upcoming clinical milestones.
We started 2018 with a steadfast commitment to execution, said Dave Fellows, Chief Executive Officer. Were pleased to announce
that our program for the treatment of
X-Linked
Retinitis Pigmentosa (XLRP) is ahead of schedule. In the third quarter of this year, we expect to announce preliminary data from the dose escalation study of the
Phase 1/2 XIRIUS trial and, in the fourth quarter of this year, begin enrollment in the expansion study in XIRIUS. This follows our achievement earlier this year of initiating our Phase 3 STAR trial in choroideremia ahead of schedule. We look
forward to continuing our mission to cure inherited retinal diseases.
Anticipated Milestones for 2018 and 2019
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NSR-RPGR
for
X-Linked
Retinitis Pigmentosa
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Q3 2018: Preliminary Data from Dose Escalation Study.
Preliminary efficacy and safety data of
NSR-RPGR
from the dose escalation study in the XIRIUS trial is expected to be
presented at an upcoming medical meeting.
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Q4 2018: Initiation of Expansion Study.
The expansion study in the XIRIUS trial is intended to enroll approximately 30 patients at a therapeutic dose informed by the dose escalation study. It is anticipated that
the expansion study will include a
low-dose
control group of approximately 15 patients.
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Mid 2019: Preliminary Data from Expansion Study
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2H 2019:
One-Year
Follow-up
Data from Dose Escalation Study
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2020:
One-Year
Follow-up
Data from Expansion Study
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NSR-REP1
for Choroideremia
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1H 2019: Completion of Enrollment for Phase 3 STAR Registrational Trial for Choroideremia
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2020:
One-year
Follow-up
Data from Phase 3 STAR Trial
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First Quarter 2018 Financial Results
Research and
development expenses for the quarter ended March 31, 2018 were $6.1 million versus $2.8 million for the quarter ended March 31, 2017. The increase of $3.3 million resulted from increases in program-related expenses of
$1.0 million for
NSR-REP1
and $1.4 million for
NSR-RPGR,
as well as a $1.4 million increase in personnel-related costs, partially offset by an
increase of $0.7 million of research and development tax credit from HM Revenue & Customs in the United Kingdom. Research and development personnel-related costs increased due to an
increase in headcount in the first quarter of 2018 to support our growth and to assist in the further development of our product candidates and pipeline. The increase in research and development personnel-related costs includes $0.2 million of
additional
non-cash
share-based compensation compared to the same period in 2017.
General and administrative
expenses for the quarter ended March 31, 2018 were $2.8 million, compared to $0.7 million for the quarter ended March 31, 2017. The increase of $2.1 million was mainly due to a $0.7 million increase in consulting and
professional fees including increased legal, accounting and audit fees, and a $1.1 million increase in personnel-related costs. General and administrative personnel-related costs increased due to an increase in employees to support our
increased research and development activities and our status as a public company. The increase in general and administrative personnel-related costs included $0.5 million of additional
non-cash
share-based compensation compared to 2017.
Net loss for the quarter ended March 31, 2018 was $14.4 million, or $0.52 basic and diluted net loss
per ordinary share, as compared to $3.5 million for the quarter ended March 31, 2017, or $0.30 basic and diluted net loss per ordinary share.
As of March 31, 2018, our cash and cash equivalents totaled $122.3 million, compared to $129.4 million at December 31, 2017. As of
March 31, 2018, we had approximately 28.9 million ordinary shares outstanding.
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. Nightstars 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 trials of
NSR-REP1
were published in
The Lancet
in 2014 and in
The New England Journal of Medicine
in 2016. 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
results of
operations for the first quarter of 2018, cash position and sufficiency of capital resources to fund our operating requirements, our planned and ongoing clinical trials for
NSR-REP1
and
NSR-RPGR,
including our Phase 3 STAR trial in choroideremia, the dose escalation study in the XIRIUS trial for
X-linked
Retinitis Pigmentosa and the planned expansion study in the XIRIUS trial, 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, 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.
###
Investors:
Senthil Sundaram, Chief Financial Officer
Brian Luque, Sr.
Manager, Investor Relations
investors@nightstartx.com
NIGHTSTAR THERAPEUTICS PLC
Consolidated Statements of Operations and Comprehensive Loss
(In thousands, except share and per share amounts)
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Three Months Ended March 31,
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2018
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2017
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Operating expenses:
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Research and development
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$
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6,064
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$
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2,750
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General and administrative
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2,776
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730
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Total operating expenses
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8,840
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3,480
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Other income (expense):
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Interest and other income
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367
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4
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Other expense, net
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(5,885
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)
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4
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Total other income (expense), net
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(5,518
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)
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8
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Net loss
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(14,358
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)
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(3,472
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)
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Other comprehensive loss:
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Foreign exchange translation adjustment
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6,346
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143
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Total comprehensive loss
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$
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(8,012
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)
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$
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(3,329
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Basic and diluted net loss per ordinary share
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$
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(0.52
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)
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$
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(0.30
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Weighted average basic and diluted ordinary shares
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27,861,709
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11,679,707
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NIGHTSTAR THERAPEUTICS PLC
Consolidated Balance Sheets
(In thousands)
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March 31,
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December 31,
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2018
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2017
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Assets
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Current assets:
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Cash and cash equivalents
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$
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122,277
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$
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129,404
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Prepaid expenses and other assets
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6,135
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5,438
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Total current assets
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128,412
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134,842
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Property and equipment, net
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352
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355
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Total assets
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$
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128,764
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$
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135,197
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Liabilities and shareholders equity
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Current liabilities:
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Accounts payable
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$
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5,148
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$
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3,196
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Accrued expenses and other liabilities
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4,980
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6,189
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Total current liabilities
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10,128
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9,385
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Total liabilities
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10,128
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9,385
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Total shareholders equity
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118,636
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125,812
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Total liabilities and shareholders equity
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$
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128,764
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$
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135,197
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