24th Sep 2018 08:44
LONDON (Alliance News) - FTSE 250-listed healthcare investor Syncona Ltd said on Monday its portfolio company Nightstar Therapeutics PLC reported positive results from the dose escalation study in its XIRIUS trial for NSR-RPGR in XLRP patients.
The general results from the trial was that NSR-RPGR was well tolerated by patients, along with positive efficacy results, which has provided the basis for an early clinical proof of concept for the trial to move to an expansion study.
Enrolment for the expansion is expected to start in the forth quarter of 2018, and to enrol 30 adult and paediatric patients, with efficacy data anticipated to be available in mid-2019.
XLRP is a rare inherited genetic retinal disorder that primarily affects men, in which 70% of cases is due to changes in genes responsible for the production of RPGR, used for the transport of proteins needed for maintaining photoreceptor cells.
The XIRIUS trial is a single-eye clinical trial intended to evaluate the safety, tolerability and efficacy of NSR-RPGR, a vector designed to produce human RPGR inside the eye.
The trial consisted of 18 adult patients, which received a single retinal injection of NSR-RPGR at different doses.
"Based on the preliminary findings of improved visual function as measured by microperimetry, we have established early proof of concept in XLRP, our second clinical program. As we move forward with the expansion study, we look forward to continuing to execute our clinical programs and sharing additional data on our XLRP program at future medical meetings," said Nightstar Chief Development Officer Tuyen Ong.
Shares in Syncona were up 0.8% at 280.28 pence on Monday.
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