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
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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.
