Regenerative Medicine Advanced Therapy (RMAT)
Designation received in June 2018 for NSR-REP1 in Choroideremia
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
June 30, 2018 and provided an update on recent achievements and
upcoming clinical milestones.
“The highlight of the second quarter was the
U.S. Food and Drug Administration’s granting of the Regenerative
Medicine Advanced Therapy designation to NSR-REP1 for
choroideremia,” said Dave Fellows, Chief Executive Officer. “I’m
also pleased to report we are on track with the guidance provided
last quarter for our NSR-RPGR program. We look forward to the
presentation of preliminary data from the dose escalation study of
the XIRIUS trial for XLRP at the EURETINA Congress in September and
plan to commence the expansion study in the XIRIUS trial in the
fourth quarter of this year.”
Business Highlights Include
- Innovate UK grant for AAV manufacturing of NSR-RPGR
started April 2018. Secured £1.5 million in funding
from Innovate UK, the UK’s innovation agency, to lead an industrial
research project for the commercial manufacture of NSR-RPGR.
- RMAT designation for NSR-REP1 in June 2018.
This is the first gene therapy RMAT designation for an inherited
retinal disease. The FDA granted RMAT designation for NSR-REP1
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.
Anticipated Milestones for 2018 and
2019
- NSR-RPGR for X-Linked Retinitis Pigmentosa
- Q3 2018: Preliminary Data from Dose Escalation
Study. Preliminary safety and efficacy data of NSR-RPGR
from the first five cohorts (n=15) out of a total of six cohorts
(n=18) in the dose escalation study in the XIRIUS trial is expected
to be available for presentation at EURETINA 2018. Session
Date/Time: Saturday, September 22, 2018 at 4:30 p.m. CEST
Presenter: Dr. Robert MacLaren, Oxford Eye Hospital, University of
Oxford Session title: Main Session 8: Research, Gene therapy for
retinitis pigmentosa (link)
- 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 and a low-dose control group of approximately
15 patients.
- Mid 2019: Preliminary Data from Expansion
Study
- 2H 2019: One-Year Follow-up Data from Dose Escalation
Study
- 2020: One-Year Follow-up Data from Expansion
Study
- NSR-REP1 for Choroideremia
- 1H 2019: Completion of Enrollment for Phase 3 STAR
Registrational Trial for Choroideremia
- 2020: One-year Follow-up Data from Phase 3 STAR
Trial
Nightstar Therapeutics’ R&D Day on
September 24, 2018
Nightstar will host an R&D Day on Monday,
September 24, with presentations beginning at 8:00 a.m., Eastern
Time. The R&D Day will feature presentations from Nightstar’s
management team and physicians specializing in the field of retinal
diseases covering the NSR-RPGR data presented at EURETINA 2018 and
the Company’s other pipeline programs.
The R&D Day event will be webcast live under
the investor relations section of Nightstar’s website at
ir.nightstartx.com. A conference call will also be provided for
those who opt to listen to the event by telephone. An archived
webcast will be available on Nightstar’s website for at least 2
weeks following the event.
Second Quarter 2018 Financial
Results
Three Months Ended June 30, 2018 and 2017
Research and development expenses were $8.1
million for the three months ended June 30, 2018, compared to $3.5
million for the three months ended June 30, 2017. The increase of
$4.5 million resulted primarily from increases in program-related
expenses of $2.4 million for NSR-REP1 and $1.1 million for
NSR-RPGR, as well as a $1.3 million increase in personnel-related
costs, and a $0.7 million increase in the indirect research and
development and preclinical expenses. The increased expenses were
partially offset by an increase of $1.0 million of research and
development tax credits from Her Majesty’s Revenue & Customs,
or HMRC. Research and development personnel-related costs increased
due to an increase in headcount during 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 $0.5 million of additional
non-cash share-based compensation compared to the same period in
2017.
General and administrative expenses were
$3.3 million for the three months ended June 30, 2018,
compared to $0.7 million for the three months ended June 30, 2017.
The increase of $2.6 million is mainly due to a $2.0 million
increase in personnel-related costs and a $0.6 million increase in
consulting and professional fees, including increased legal,
accounting and audit fees. General and administrative
personnel-related costs increased due to an increase in headcount
to support our increased research and development activities,
growth of our company and our status as a public company. The
increase in general and administrative personnel-related costs
includes $0.6 million of additional non-cash share-based
compensation compared to the same period in 2017.
Net loss for the three-month period ended June
30, 2018 was $8.1 million, or $0.29 basic and diluted net loss per
ordinary share, as compared to $4.2 million, or $0.18 basic and
diluted net loss per ordinary share for the three-month period
ended June 30, 2017.
Six Months Ended June 30, 2018 and 2017
Research and development expenses were $14.1
million for the six months ended June 30, 2018, compared to $6.3
million for the six months ended June 30, 2017. The increase of
$7.8 million resulted primarily from increases in program-related
expenses of $3.4 million for NSR-REP1 and $2.5 million for
NSR-RPGR, as well as a $2.7 million increase in personnel-related
costs and a $0.9 million increase in the indirect research and
development expenses. The increased expenses were partially offset
by an increase of $1.6 million of research and development tax
credits from the HMRC. Research and development personnel-related
costs increased due to an increase in headcount 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 $0.8 million of additional
non-cash share-based compensation compared to the same period in
2017.
General and administrative expenses were
$6.1 million for the six months ended June 30, 2018, compared
to $1.4 million for the six months ended June 30, 2017. The
increase of $4.7 million is mainly due to a $3.7 million increase
in personnel-related costs and $1.0 million increase in consulting
and professional fees, including increased legal, accounting and
audit fees and insurance costs. General and administrative
personnel-related costs increased due to an increase in headcount
to support our increased research and development activities,
growth of our company, and our status as a public company. The
increase in general and administrative personnel-related costs
includes $1.1 million of additional non-cash share-based
compensation compared to the same period in 2017.
Net loss for the six-month period ended June 30,
2018 was $22.5 million, or $0.80 basic and diluted net loss per
ordinary share, as compared to $7.7 million, or $0.33 basic and
diluted net loss per ordinary share for the six-month period ended
June 30, 2017.
As of June 30, 2018, our cash, cash equivalents
and marketable securities totaled $111.4 million, compared to
$129.4 million at December 31, 2017. As of June 30, 2018, there
were 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.
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 treatments
currently available 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. 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,” “could,”
“intend,” “estimate,” “will,” “would,” “may,” “should,” “project,”
“target,” “track,” “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 cash position and sufficiency of
capital resources to fund our operating requirements, trends and
other factors that may affect our financial results, our planned
and ongoing clinical trials for NSR-REP1 and NSR-RPGR, including
our Phase 3 STAR trial in choroideremia and Phase 1/2 XIRIUS trial
in X-linked retinitis pigmentosa, potential results and timelines
relating to the dose escalation study in the XIRIUS trial 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 management's current expectations of future
events as of the date of this release 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 those related to the timing and costs
involved in commercializing any product candidate that receives
regulatory approval; the initiation, timing and conduct of clinical
trials; the availability of data from clinical trials and
expectations for regulatory submissions and approvals; our
scientific approach and general development progress; the
availability or commercial potential of the our product candidates;
the sufficiency of our cash resources, and other 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. Any reference to our
website address in this press release is intended to be an inactive
textual reference only and not an active hyperlink.
Investors:Senthil Sundaram,
Chief Financial OfficerBrian Luque, Sr. Manager, Investor
Relationsinvestors@nightstartx.com
NIGHTSTAR THERAPEUTICS PLC |
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Consolidated Statements of Operations and
Comprehensive Loss |
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(In thousands, except per share amounts) |
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Three Months Ended June 30, |
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Six Months Ended June 30, |
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2018 |
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2017 |
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2018 |
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2017 |
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Operating expenses: |
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|
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|
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Research
and development |
$ |
8,052 |
|
|
$ |
3,542 |
|
|
$ |
14,116 |
|
|
$ |
6,292 |
|
General
and administrative |
3,324 |
|
|
677 |
|
|
6,100 |
|
|
1,407 |
|
Total
operating expenses |
11,376 |
|
|
4,219 |
|
|
20,216 |
|
|
7,699 |
|
Other
income (expense): |
|
|
|
|
|
|
|
|
|
|
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Interest
and other income |
722 |
|
|
2 |
|
|
1,089 |
|
|
6 |
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Other
Income (expense), net |
2,224 |
|
|
(4 |
) |
|
(3,661 |
) |
|
— |
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Total
other income (expense), net |
2,946 |
|
|
(2 |
) |
|
(2,572 |
) |
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6 |
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Loss
before benefit for income taxes |
(8,430 |
) |
|
(4,221 |
) |
|
(22,788 |
) |
|
(7,693 |
) |
Benefit
for income taxes |
(334 |
) |
|
— |
|
|
(334 |
) |
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— |
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Net
loss |
(8,096 |
) |
|
(4,221 |
) |
|
(22,454 |
) |
|
(7,693 |
) |
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|
|
|
|
|
|
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Other
comprehensive income (loss) |
(2,681 |
) |
|
1,579 |
|
|
3,665 |
|
|
1,722 |
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Total
comprehensive loss |
$ |
(10,777 |
) |
|
$ |
(2,642 |
) |
|
$ |
(18,789 |
) |
|
$ |
(5,971 |
) |
Basic
and diluted net loss per ordinary share |
$ |
(0.29 |
) |
|
$ |
(0.18 |
) |
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$ |
(0.80 |
) |
|
$ |
(0.33 |
) |
Weighted
average basic and diluted ordinary shares |
28,053 |
|
|
23,688 |
|
|
27,957 |
|
|
23,336 |
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NIGHTSTAR THERAPEUTICS PLC |
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Consolidated Balance Sheets |
(In thousands) |
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June 30, |
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December 31, |
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2018 |
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2017 |
Assets |
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Current
assets: |
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Cash and
cash equivalents |
$ |
41,576 |
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$ |
129,404 |
Marketable securities |
69,785 |
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— |
Prepaid
expenses and other assets |
9,232 |
|
5,438 |
Total
current assets |
120,593 |
|
134,842 |
Property
and equipment, net |
380 |
|
355 |
Other
assets |
215 |
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— |
Total
assets |
$ |
121,188 |
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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 |
$ |
3,369 |
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$ |
3,196 |
Accrued
expenses and other liabilities |
8,752 |
|
6,189 |
Total
current liabilities |
12,121 |
|
9,385 |
Total
liabilities |
12,121 |
|
9,385 |
|
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Total
shareholders’ equity |
109,067 |
|
125,812 |
Total
liabilities and shareholders’ equity |
$ |
121,188 |
|
$ |
135,197 |
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