SYNC Syncona Limited

 
TIDMSYNC 
 
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. 
 
 
 
END 
 

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March 05, 2018 06:33 ET (11:33 GMT)

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