Syncona Limited Nightstar reports Q2 2018 financial results

 
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: [phone] 
 
Tulchan Communications 
Martin Robinson 
Lisa Jarrett-Kerr 
Tel: [phone] 
 
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 
 

(END) Dow Jones Newswires

August 13, 2018 06:11 ET (10:11 GMT)