MeiraGTx's gene therapy shows sustained benefit in inherited retinal disorder

• MeiraGTx (MGTX +2.5%) has announced 12-month data from the ongoing Phase 1/2 trial of AAV-RPGR, an investigational gene therapy for the treatment of X-linked retinitis pigmentosa (XLRP). Data were presented at the American Academy of Ophthalmology.
• The primary endpoint of the trial is safety, with secondary endpoints assessing changes in visual function at pre-specified timepoints post-treatment.
• In the dose escalation phase of the trial, data at the 12-month time point demonstrated statistically significant improvement in retinal sensitivity in treated eyes in both the low (n=3) and intermediate (n=4) dose cohorts, with six of seven patients demonstrating improved or stable vision in the treated eye one year after treatment.
• Efficacy signals were observed at the first post-treatment assessments at three months, with improvements sustained or increased at 12 months.
• AAV-RPGR is well-tolerated till data, with no dose-limiting events occurred.
• MeiraGTx and development partner Janssen are preparing to initiate the pivotal Phase 3 Lumeos clinical trial of AAV-RPGR for the treatment of patients with XLRP.
• XLRP is the most severe form of retinitis pigmentosa, a group of inherited retinal diseases characterized by progressive retinal degeneration and vision loss. The most frequent cause of XLRP is disease-causing variants in the RPGR gene.
• AAV-RPGR is designed to deliver functional copies of the RPGR gene to the subretinal space in order to improve and preserve visual function.
• https://seekingalpha.com/news/3636738-meiragtxs-gene-therapy-shows-sustained-benefit-in-inherited-retinal-disorder