Press release
MeiraGTx Announces Positive Top-Line Data from the MGT009 Phase 1/2 Clinical Study Demonstrating Safety and Improvement in Multiple Domains of Vision in X-Linked Retinitis Pigmentosa Patients treated with Botaretigene Sparoparvovec (AAV-RPGR) compared...
MeiraGTx Announces Positive Top-Line Data from the MGT009 Phase 1/2 Clinical Study Demonstrating Safety and Improvement in Multiple Domains of Vision in
About this update from Meiragtx Holdings Plc
[{"type":"text","content":"MeiraGTx Announces Positive Top-Line Data from the MGT009 Phase 1/2 Clinical Study Demonstrating Safety and Improvement in Multiple Domains of Vision in X-Linked Retinitis Pigmentosa Patients treated with Botaretigene Sparoparvovec (AAV-RPGR) compared to Untreated Randomized Control \n • The primary outcome of the MGT009 study is safety, and botaretigene sparoparvovec treatment was found to be generally safe and well-tolerated • Significant improvements were demonstrated in multiple different endpoints in botaretigene sparoparvovec-treated patients across each of the three domains of vision: retinal function, visual function and functional vision • MeiraGTx to host a conference call on Tuesday, June 28, 2022, at 8am ET LONDON and NEW YORK, June 28, 2022 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced top-line data from the Phase 1/2 clinical study MGT009 (NCT03252847) of botaretigene sparoparvovec (formerly referred to as AAV-RPGR), an investigational gene therapy in development for the treatment of patients with X-linked retinitis pigmentosa (XLRP) with disease-causing variants in the RPGR gene. Treatment with botaretigene sparoparvovec was found to be generally safe and well-tolerated, and significant improvements were demonstrated in multiple endpoints across each of the three domains of vision - retinal function, visual function, and functional vision - in participants treated with botaretigene sparoparvovec when compared to the randomized untreated control arm of the study at 6 months post-treatment. MeiraGTx and Janssen Pharmaceuticals, Inc. (Janssen), one of the Janssen Pharmaceutical Companies of Johnson & Johnson, are jointly developing botaretigene sparoparvovec as part of a broader collaboration to develop and commercialize gene therapies for the treatment of inherited retinal diseases. In the dose escalation and expansion phases of the MGT009 study, improvements in vision were seen in subjects treated with low and intermediate doses of botaretigene sparoparvovec compared to the randomized concurrent control arm at 6 months. Improvements were demonstrated in (i) retinal sensitivity on static perimetry, using both pointwise responder analysis as well as mean retinal sensitivity analysis; (ii) functional vision in a vision-guided mob...