REG-Syncona Limited: Nightstar Reports 2017 Financial Results

SynconaAnnouncement

04/04/2018 07:05

Syncona Limited

Nightstar reports 2017 financial results and business highlights

4 April 2018

Syncona Ltd, a leading healthcare company focused on investing in and building
global leaders in life science, today notes the announcement that its
portfolio company, Nightstar Therapeutics Plc (NASDAQ: NITE) (Nightstar),
reported its 2017 Financial Results and Business Highlights yesterday, April 3
2018.

The Annual Report and financial accounts can be accessed on Nightstar’s
investor website at http://ir.nightstartx.com and full text of the summary
announcement from Nightstar is contained below.

 ENDS 

Enquiries
Syncona Ltd
Siobhan Weaver
Tel: +44 (0) 20 7611 2031
+44 (0) 7921 822 994

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. Our current
investment portfolio consists of seven high quality companies in life science
and a leading range of fund investments. 

We seek to partner with the best, brightest and most ambitious minds in
science to build globally competitive businesses. We are established leaders
in gene therapy, cell therapy and advanced diagnostics, and focus on
delivering dramatic efficacy for patients in areas of high unmet need.

Our fund investments represent a productively deployed evergreen funding base
which enables us to take a long-term approach to investing in life sciences as
we target the best new opportunities and support our existing portfolio
companies to grow and succeed.

Syncona is aligned with two of the premium charitable funders in UK science,
the Wellcome Trust, original founder of Syncona, and Cancer Research UK, both
of which are significant shareholders in our business.  We make a donation of
0.3% of Net Asset Value to a range of charities each year.

Nightstar Therapeutics Reports 2017 Financial Results and Business Highlights

- Recent initiation of STAR phase 3 trial in Choroideremia -
- Begins 2018 with a cash position over $129 million -

LEXINGTON, Mass. and LONDON – April 3, 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 year ended December 31, 2017 and provided an update
on recent corporate and clinical highlights.

“2017 was an exceptional year for the company,” said Dave Fellows, Chief
Executive Officer. “We set the stage for the recent initiation of the
first-ever Phase 3 trial in choroideremia and today announced further data
supporting the durability of the treatment effect for NSR-REP1 in
choroideremia. Elsewhere in our pipeline, we initiated our Phase 1/2 gene
therapy trial for X-linked retinitis pigmentosa and in-licensed our program
for Stargardt disease. We also raised more than $130 million of gross proceeds
from our successful Series C financing and IPO to continue our pioneering
research and development activities.”

Business Highlights Include
* Initiated First-Ever Phase 3 Trial for Choroideremia (NSR-REP1).  The STAR
Phase 3 registrational trial is expected to enroll approximately 140 patients
across 18 clinical sites in the United States, Europe, Canada and South
America, of which six sites will be surgical centers. Eligible patients will
be randomized into one of three study arms: 56 patients receiving a high-dose
of NSR-REP1 in one-eye; 28 patients receiving a low-dose of NSR-REP1 in
one-eye; and 56 patients receiving no treatment (no-sham, parallel control
arm). The primary endpoint of the STAR trial is the proportion of patients
with an improvement of at least 15 ETDRS letters from baseline in visual
acuity at 12 months post-treatment. The primary endpoint will compare patients
in the high-dose treatment arm with patients in the control arm. We anticipate
that the STAR trial will be fully enrolled by the first half of 2019.
* NSR-REP1 Data Supporting the Durability of Treatment Effect to Be Presented
at ARVO 2018
(https://ep70.eventpilot.us/web/page.php?page=IntHtml&project=ARVO18&id=2909906)
(NSR-REP1).  After two years of follow-up in the Oxford Trial, the median
visual acuity (n=14) improved by 4.5 letters in the treated eyes compared with
a loss of 1.5 letters in the untreated eyes. At the last follow up (ranging
from two to five years), visual acuity had been maintained or had increased in
all 12 treatment eyes that received gene therapy as per protocol, compared to
four of the 12 untreated eyes. (link to abstract
(https://ep70.eventpilot.us/web/page.php?page=IntHtml&project=ARVO18&id=2909906))
* Natural History Data Increases Understanding of 2.5x Greater Risk of
Untreated Disease Progression in Choroideremia.  Preliminary top-line data
from the NIGHT natural history observational study at 20 months follow-up (the
final time point in the NIGHT study) indicated that 22% of choroideremia eyes
experienced a loss in visual acuity of five or more ETDRS letters, compared to
less than 8% for patients treated with the high-dose of NSR-REP1.  This data
further supports the importance of developing new treatments for choroideremia
to reverse or maintain the loss of visual acuity due to the natural
progression of disease.
* Initiated Phase 1/2 Gene Therapy Clinical Trial for X-Linked Retinitis
Pigmentosa (XLRP).  Our second retinal gene therapy product candidate,
NSR-RPGR, is in a dose-ranging Phase 1/2 clinical trial for the treatment of
XLRP in the United Kingdom and United States.  We are evaluating multiple
doses and have completed dosing of the first few cohorts of three patients
each. We expect the initial data on safety and tolerability from the dose
escalation cohorts of this trial to be available towards the end of 2018; this
data will determine the dose for study in an expansion cohort anticipated to
include up to 30 patients.
* Expanded Inherited Retinal Disease Pipeline with Novel Gene Therapy for the
Treatment of Stargardt Disease. We recently in-licensed NSR-ABCA4 for the
treatment of Stargardt disease, utilizing a novel technology developed by the
University of Oxford. Preclinical proof-of-concept studies in the Abca4-/-
murine model of Stargardt disease have demonstrated the expression,
localization and function of the ABCA4 protein. Stargardt disease is the most
common form of inherited juvenile macular dystrophy with a prevalence of one
in 10,000 people. There are no treatments currently available for Stargardt
disease. This program will leverage Nightstar’s capabilities, physician
relationships and clinical experience with NSR-REP1 and NSR-RPGR.
Financial Results

Research and development expenses for the year ended December 31, 2017 were
$20.5 million versus $10.2 million for the year ended December 31, 2016. The
increase of $10.3 million resulted from increases in program-related expenses
of $3.4 million for NSR-REP1, and $1.5 million for NSR-RPGR, as well as a $1.9
million increase in personnel-related costs, and a $1.3 million expense to
reduce research and development tax relief claims receivable from HM Revenue &
Customs in the United Kingdom. Research and development personnel costs
increased due to additional employees hired in 2017 to support our growing
company and to assist in the further development of our product candidates and
pipeline.  This increase in research and development personnel-related costs
includes $0.5 million of additional non-cash stock-based compensation compared
to 2016.

General and administrative expenses for the year ended December 31, 2017 were
$7.0 million, compared to $2.1 million for the year ended December 31, 2016.
The increase of $4.9 million is mainly due to a $2.1 million increase in
consulting and professional fees, including increased legal, accounting and
audit fees associated with our corporate reorganization and non-capitalizable
costs incurred in connection with our IPO, and a $1.8 million increase in
personnel-related costs.  General and administrative personnel costs
increased to support our increased research and development activities and our
status as a public company. This increase in general and administrative
personnel-related costs includes $0.7 million of additional non-cash
stock-based compensation compared to 2016.

Net loss for the year ended December 31, 2017 was $29.7 million, or ($1.63)
basic and diluted net loss per ordinary share, as compared to $12.2 million
for the year ended December 31, 2016, or ($1.36) basic and diluted net loss
per ordinary share.

As of December 31, 2017, our cash and cash equivalents totaled $129.4 million,
compared to $10.1 million at December 31, 2016.  In October 2017, we sold
6,164,000 American Depositary Shares (representing the same number of ordinary
shares), including the full exercise by the underwriters of their option to
purchase additional ADSs, at $14.00 per ADS in our IPO for net proceeds of
approximately $77.4 million.  In June 2017, we completed a $45 million Series
C financing with Syncona, NEA, Wellington and Redmile.  As of December 31,
2017, we had 28.9 million ordinary shares outstanding.

Upcoming Conference Participation

Nightstar will participate in the Bloomberg Biotech Innovations Conference on
Monday, April 9(th), 2018 at 3:30pm ET in New York City.  Additional
information is available at http://b.bloomberg.com/reg-BiotechInnovations. The
link to the live audio webcast and replay of the presentation will be
available through Bloomberg terminals at Live  go .

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 current treatments and
affects approximately one in every 50,000 people. Positive results from a
Phase 1/2 trial of NSR-REP1 were published in The Lancet
(http://thelancet.com/journals/lancet/article/PIIS0140-6736(13)62117-0/fulltext)
in 2014 and in The New England Journal of Medicine
(http://www.nejm.org/doi/full/10.1056/NEJMc1509501) in 2016.  Nightstar’s
second product candidate, NSR-RPGR, is currently being evaluated in a Phase
1/2 clinical 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 full-year 2017, cash position and
sufficiency of capital resources to fund our operating requirements, our
planned clinical trials for NSR-REP1 and NSR-RPGR, including our STAR Phase 3
trial in choroideremia and the expansion of future clinical trials, 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 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 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
the "Risk Factors" section of our prospectus filed pursuant to Rule 424(b)(4)
under the U.S. Securities Act of 1933, as amended, on September 28, 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.



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