Exhibit 99.1
Nightstar Reports Positive Proof of Concept Data from Dose Escalation Study in XIRIUS Trial for
NSR-RPGR
in XLRP Patients
NSR-RPGR
data presented at
EURETINA 2018 Congress demonstrated proof of concept with durable dose-related improvements seen as early as month 1 across multiple microperimetry analyses
Preliminary efficacy signals observed in 3/3 patients in cohort 3 and 2/6 patients in cohorts 4 and 5, including durable improvements in
overall macula sensitivity, central 16 macula sensitivity and number of improved macula loci
NSR-RPGR
was well-tolerated with no dose limiting toxicities or serious treatment-related adverse
events
XIRIUS expansion study planned to initiate in Q4 2018
Detailed results to be presented at R&D day on September 24, 2018
WALTHAM, Mass and LONDON, UK September
22, 2018
(GLOBE NEWSWIRE)
Nightstar Therapeutics plc (NASDAQ:NITE), a
clinical-stage gene therapy company developing treatments for rare inherited retinal diseases, today announced that positive preliminary safety and efficacy data of
NSR-RPGR
from the dose escalation study in
the Phase 1/2 XIRIUS trial were presented today at the EURETINA 2018 Congress.
We initiated this study last year, with the anticipation of
demonstrating safety and stabilization of disease with
NSR-RPGR,
our codon-optimized gene therapy for XLRP, said Tuyen Ong, M.D., chief development officer of Nightstar. 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.
XIRIUS is a Phase 1/2, open-label, dose-ranging,
single-eye
clinical trial consisting of a dose escalation study and an expansion study with sites in both the United States and the United Kingdom. The XIRIUS trial is intended to evaluate the safety, tolerability
and efficacy of
NSR-RPGR
for the treatment of XLRP in patients with the RPGR mutation.
Enrollment of the dose
escalation study in the XIRIUS trial was completed in August 2018, consisting of six cohorts of three patients each for a total of 18 adult patients. Each patient in the trial received a single
sub-retinal
injection of
NSR-RPGR.
Doses ranged from 5x10^9 genome particles (gp) in cohort 1 up to 5x10^11gp in cohort 6.
One-year
follow-up
data on all 18 patients in the dose escalation study is expected to be available in the second half of 2019.
Preliminary
NSR-RPGR
Data from the Dose Escalation Study of XIRIUS Trial in XLRP
Safety and efficacy data were presented from the
one-month
follow-up
for the
first five cohorts. As of September 4, 2018, data through varying timepoints up to 12 months was available for the earlier cohorts. However, the
one-month
follow-up
was the common timepoint for which the