Syncona
Limited
Nightstar
initiates Pivotal Phase 3 trial in Choroideremia
5 March 2018
Syncona Ltd, a leading healthcare company focused on investing
in and building global leaders in life science, today notes the
announcement from its portfolio company, Nightstar Therapeutics Plc
(NASDAQ: NITE) (Nightstar).
Nightstar has initiated its STAR Phase 3 registrational trial
(“the STAR trial”) to study the safety and efficacy of NSR-REP1 in
patients with choroideremia. The STAR 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.
Patients in the STAR trial are expected to be recruited
primarily from the ongoing Nightstar-sponsored natural history
observational study (the NIGHT study) in order to accelerate Phase
3 enrolment from this well-characterized patient population. 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.[1] The primary endpoint
will be assessed at one-year by comparing patients in the high-dose
treatment arm with patients in the control arm.
Nightstar’s STAR trial represents the first ever Phase 3 pivotal
trial for choroideremia, a rare disease leading to total blindness
for which there is currently no available treatment. In data from
32 patients treated with NSR-REP1 across four open-label Phase 1/2
clinical trials, over 90 per cent of treated patients maintained or
improved their visual acuity over a one-year follow-up period.
The full Nightstar announcement can be found here:
http://ir.nightstartx.com/news-releases
[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
Forward-looking statements – this
announcement contains certain forward-looking statements with
respect to the portfolio of investments of Syncona Ltd. These
statements and forecasts involve risk and uncertainty because they
relate to events and depend upon circumstances that may or may not
occur in the future. There are a number of factors that could cause
actual results or developments to differ materially from those
expressed or implied by these forward-looking statements and
forecasts. Nothing in this announcement should be construed as a
profit forecast.
About Syncona:
Syncona is a leading FTSE250 healthcare company focussed 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 market leading funds portfolio seeks to generate superior
returns by investing in long only and alternative investment funds.
This represents 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.
About Nightstar:
Nightstar is a 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 most advanced programme is in clinical trials for
the treatment of choroideremia, a rare disease which causes
permanent loss of eyesight for which there is no treatment.
Nightstar’s treatment would deliver a gene therapy by injection
into the retina, providing a working copy of the disease-causing
faulty gene locally in the eye. The disease modifying technology
has the potential to maintain and restore sight in patients, from a
single administration.
There are multiple inherited forms of blindness that are
addressable with Nightstar’s gene therapy technology. Nightstar is
building a pipeline of products based on its manufacturing, gene
therapy and retinal surgery capability. Nightstar is listed on
NASDAQ under the ticker ‘NITE’.
Syncona launched Nightstar in 2014 with Professor Robert MacLaren, Professor of Ophthalmology at
the University of Oxford.
[1] ETDRS is a standardised, widely used measure of visual
acuity which requires subjects to read letters on a chart.