TIDMSYNC
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.
[S]
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 GBP1.5 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 Six Months Ended June
30, 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 $ (0.29) $ (0.18) $ (0.80) $ (0.33)
share
Weighted average basic and diluted 28,053 23,688 27,957 23,336
ordinary shares
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
END
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August 13, 2018 06:11 ET (10:11 GMT)
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