MeiraGTx Holdings plc · NASDAQ:MGTX
MeiraGTx — ARVO Annual Meeting 2022
13 slides from MeiraGTx's conference presentation, May 4, 2022
Slide 1: AAV5-RPGR Gene Therapy for X-Linked Retinitis Pigmentosa Demonstrates Localized Improvements in Static Perimetry
AAV5-RPGR (Botaretigene Sparoparvovec) Gene Therapy for X-Linked Retinitis Pigmentosa Demonstrates Localized Improvements in Static Perimetry Michel Michaelides,1* Jialin Xu,2 Dai Wang,2 Peggy Wong,2 Albert Fung,2 Alexandra Forbes,3 Stuart Naylor,3 Robert Zeldin,3 Maria A. Parker,4 Thales Antonio Ca
Slide 2: MGT009: Phase 1/2 Trial of AAV5-RPGR
MGT009: Phase 1/2 Trial of AAV5-RPGR Open-label study of an AAV5-RPGR gene therapy (NCT03252847) conducted at 5 sites in the US and UK Dose Dose-escalation Phase Expansion Cohort Confirmation Adults Children Adults and Children Followed 12 months Low Intermediate dose Intermediate dose Low dose (n=8
Slide 3: Improvement in Mean Retinal Sensitivity and V30 in Low- and Intermediate-dose Cohorts
Improvement in Mean Retinal Sensitivity and V30 in Low- and Intermediate-dose Cohorts1 Dose-escalation Phase Treated-Untreated Eye Difference @ 12 months Parameter (90% CI adjusted for baseline) Mean Retinal Sensitivity (dB) Low (n=2) ꝉ 0.76 (–0.14, 1.66) Intermediate (n=4) 1.05 (0.81, 1.29)* High (
Slide 4: Improvement in Retinal Sensitivity in Low- and Intermediate-dose Cohorts (n=6)
Improvement in Retinal Sensitivity in Low- and Intermediate-dose Cohorts (n=6)1 Dose-escalation Phase Static Perimetry V30 Change From Baseline (Mean ± 90% CI) Eye M3 M6 M9 M12 Treated 0.38 (0.07, 0.69) 0.67 (0.13, 1.20) 0.86 (0.17, 1.55) 0.95 (0.17, 1.74) Untreated –0.22 (–0.44, 0.01) –0.39 (–.76,
Slide 5: Retinal Sensitivity Improvements on Mesopic Microperimetry: Intermediate-dose Cohort at Month 12
Retinal Sensitivity Improvements on Mesopic Microperimetry: Intermediate-dose Cohort at Month 121 Dose-escalation Phase 12 mo Δ Treated Eye Untreated Eye Treated – Baseline Month 3 Month 6 Month 9 Month 12 12 mo Δ Baseline Month 3 Month 6 Month 9 Month 12 12 mo Δ Untreated 1-004 ND –1.2 dB ND –2.9 d
Slide 6: Functional Vision Assessment: Mobility Maze
Functional Vision Assessment: Mobility Maze1 Dose-escalation Phase Patient 01-007 Maze assessment shown at 9-month timepoint. • Light level: 1 lux • Baseline performance: 61.7 seconds with 2 errors • 9-month performance: 16.4 seconds with no errors 1. Michaelides, M et al. Presentation at the Americ
Slide 7: Bleb Overlay Subanalysis Methods
Bleb Overlay Subanalysis Methods • Location/extent of bleb(s) was (were) documented with intraoperative video and delineated by the surgeon • Hill of vision (HOV) surface was flipped 90 degrees vertically along the horizontal axis (for proper alignment) and superimposed onto the color fundus image •
Slide 8: Example of V30 Bleb Overlay: 6 months
Example of V30 Bleb Overlay: 6 months Patient 01-005 Escalation Cohort https://www.congresshub.com/Retina/ARVO2022/ The QR code is intended for use in the US, Canada, Vietnam, Australia, Indonesia, and India only; it is intended to provide scientific information for individual reference, and the inf
Slide 9: Example of V30 Bleb Overlay: 6 months
Example of V30 Bleb Overlay: 6 months Patient 02-022 Expansion Cohort V30, 30-degree hill-of-vision. https://www.congresshub.com/Retina/ARVO2022/ The QR code is intended for use in the US, Canada, Vietnam, Australia, Indonesia, and India only; it is intended to provide scientific information for ind
Slide 10: Example of V30 Bleb Overlay: 6 months
Example of V30 Bleb Overlay: 6 months Patient 02-028 Expansion Cohort V30, 30-degree hill-of-vision. https://www.congresshub.com/Retina/ARVO2022/ The QR code is intended for use in the US, Canada, Vietnam, Australia, Indonesia, and India only; it is intended to provide scientific information for ind
Slide 11: Conclusions
Conclusions • Patients treated with the low- and intermediate-dose of AAV5-RPGR (botaretigene sparoparvovec) in MGT-009 achieved improvements in retinal sensitivity • Individual patients demonstrated improved visual mobility at low light levels • Exploration of the association between the location o
Slide 12: Acknowledgments
Acknowledgments • Participants and families • Referring clinicians and collaborators • Trial managers, coordinators, and technicians • Reading centers in Belfast, OHSU, and MCW • Trial funding: MeiraGTx and Janssen OHSU, Oregon Health and Science University; MCW, Medical College of Wisconsin. https:
Slide 13: To View a Copy of Today's Presentation, Please Use the QR Code Below
To View a Copy of Today’s Presentation, Please Use the QR Code Below https://www.congresshub.com/Retina/ARVO2022/ The QR code is intended for use in the US, Canada, Vietnam, Australia, Indonesia, and India only; it is intended to provide scientific information for individual reference, and the infor