MeiraGTx Announces Nine-Month Data from Phase 1/2 Trial of AAV-RPGR Demonstrating Significant and Sustained Vision Improvement in X-Linked Retinitis Pigmentosa (XLRP)

• Data presented at EURETINA 2020 Virtual Congress show sustained improvements in retinal sensitivity at nine months
  
• Significant improvements were also demonstrated in the time taken to walk through a vision-guided mobility maze at nine months

LONDON and NEW YORK, Oct. 03, 2020 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced nine-month data from the ongoing Phase 1/2 clinical trial (NCT03252847) of AAV-RPGR, an investigational gene therapy in development for the treatment of patients with X-linked retinitis pigmentosa (XLRP). Data presented today at the EURETINA 2020 Virtual Congress demonstrated significant improvement in vision-guided mobility and retinal sensitivity in treated eyes compared to untreated eyes nine months after treatment.

MeiraGTx and Janssen Pharmaceuticals, Inc. (Janssen), one of the Janssen Pharmaceutical Companies of Johnson & Johnson, are jointly developing AAV-RPGR as part of a broader collaboration to develop and commercialize gene therapies for the treatment of inherited retinal diseases.

In July 2020, MeiraGTx and Janssen announced six-month data from the ongoing MGT009 clinical trial showing significant improvement in retinal sensitivity in the low (n=3) and intermediate (n=4) dose cohorts in the dose escalation phase of the trial.

Data at the nine-month time point continued to demonstrate significant improvement in retinal sensitivity in treated eyes in both the low and intermediate dose cohorts. In addition, data from the assessment of vision-guided mobility carried out at the nine-month timepoint demonstrated a significant improvement in walk time compared to baseline in treated eyes compared to untreated eyes in the low and intermediate dose cohorts (n=6).

“There are currently no treatment options for XLRP, and vision in patients suffering from this disease inevitably declines over time,” said Michel Michaelides¹, BSc MB BS MD(Res) FRCOphth FACS, MGT009 trial investigator, Consultant Ophthalmologist, Moorfields Eye Hospital and Professor of Ophthalmology, University College London. “Data from this clinical trial demonstrate that patients treated with AAV-RPGR had significant and sustained improvement in retinal sensitivity, as well as improved ability to navigate in low light conditions. These exciting results continue to suggest that AAV-RPGR has the potential to be a much-needed and important treatment option for those living with XLRP.”