Syncona
Limited
Nightstar reports
Q2 2018 financial results
13 August 2018
Syncona Ltd, a leading healthcare company focused on investing
in and building global leaders in life science, notes the
announcement that its portfolio company, Nightstar Therapeutics Plc
(NASDAQ: NITE) (Nightstar), reported its Second Quarter 2018
Financial Results today, 13 August
2018.
The announcement can be accessed on Nightstar’s investor website
at http://ir.nightstartx.com and full text of the announcement from
Nightstar is contained below.
[ENDS]
Enquiries
Syncona Ltd
Annabel Clay
Tel: +44 (0) 20 7611 2031
Tulchan Communications
Martin Robinson
Lisa Jarrett-Kerr
Tel: +44 (0) 207 353 4200
Copies of this press release and other corporate information
can be found on the company website at: www.synconaltd.com
About Syncona:
Syncona is a leading FTSE250 healthcare company focused on
investing in and building global leaders in life science. Our
vision is to deliver transformational treatments to patients in
truly innovative areas of healthcare while generating superior
returns for shareholders.
We seek to partner with the best, brightest and most ambitious
minds in science to build globally competitive businesses.
We take a long-term view, underpinned by a deep pool of capital,
and are established leaders in gene and cell therapy. We focus on
delivering dramatic efficacy for patients in areas of high unmet
need.
Nightstar
Therapeutics Reports Second Quarter 2018 Financial Results and
Business Highlights
Regenerative
Medicine Advanced Therapy (RMAT) Designation received in
June 2018 for NSR-REP1 in
Choroideremia
Preliminary data
from Phase 1/2 XIRIUS trial for X-Linked Retinitis Pigmentosa
expected at EURETINA 2018 in Vienna,
Austria in September 2018
R&D Day
planned for September 24, 2018 in
New York City
WALTHAM, Mass. and
LONDON, UK – August 13, 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
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.
Consolidated Statements of Operations and Comprehensive
Loss |
(In
thousands, except per share amounts) |
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended June 30, |
|
Six Months Ended June 30, |
|
|
2018 |
|
2017 |
|
2018 |
|
2017 |
Operating
expenses: |
|
|
|
|
|
|
|
|
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): |
|
|
|
|
|
|
|
|
Interest and other
income |
|
722 |
|
2 |
|
1,089 |
|
6 |
Other Income
(expense), net |
|
2,224 |
|
(4) |
|
(3,661) |
|
— |
Total other income
(expense), net |
|
2,946 |
|
(2) |
|
(2,572) |
|
6 |
Loss before benefit
for income taxes |
|
(8,430) |
|
(4,221) |
|
(22,788) |
|
(7,693) |
Benefit for income
taxes |
|
(334) |
|
— |
|
(334) |
|
— |
Net loss |
|
(8,096) |
|
(4,221) |
|
(22,454) |
|
(7,693) |
|
|
|
|
|
|
|
|
|
Other comprehensive
income (loss) |
|
(2,681) |
|
1,579 |
|
3,665 |
|
1,722 |
Total comprehensive
loss |
$ |
(10,777) |
$ |
(2,642) |
$ |
(18,789) |
$ |
(5,971) |
Basic and diluted
net loss per ordinary share |
$ |
(0.29) |
$ |
(0.18) |
$ |
(0.80) |
$ |
(0.33) |
Weighted average
basic and diluted ordinary shares |
|
28,053 |
|
23,688 |
|
27,957 |
|
23,336 |
Consolidated Balance Sheets |
(In
thousands) |
|
|
|
|
|
|
|
June
30, |
|
December 31, |
|
|
2018 |
|
2017 |
Assets |
|
|
|
|
Current
assets: |
|
|
|
|
Cash and cash
equivalents |
$ |
41,576 |
$ |
129,404 |
Marketable
securities |
|
69,785 |
|
— |
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 |
|
— |
Total
assets |
$ |
121,188 |
$ |
135,197 |
|
|
|
|
|
Liabilities and
shareholders’ equity |
|
|
|
|
Current
liabilities: |
|
|
|
|
Accounts
payable |
$ |
3,369 |
$ |
3,196 |
Accrued expenses
and other liabilities |
|
8,752 |
|
6,189 |
Total current
liabilities |
|
12,121 |
|
9,385 |
Total
liabilities |
|
12,121 |
|
9,385 |
|
|
|
|
|
Total shareholders’
equity |
|
109,067 |
|
125,812 |
Total liabilities
and shareholders’ equity |
$ |
121,188 |
$ |
135,197 |