MeiraGTx Holdings plc · NASDAQ:MGTX
MeiraGTx — EURETINA Virtual Meeting 2021
9 slides from MeiraGTx's conference presentation, Sep 12, 2021
Slide 1: AAV5-RPGR Gene Therapy for RPGR-Associated X-Linked Retinitis Pigmentosa Reverses Natural Disease Progression
EURETINA Virtual Meeting – 9-12 September 2021 AAV5-RPGR Gene Therapy for RPGR-Associated X-Linked Retinitis Pigmentosa Reverses Natural Disease Progression Michel Michaelides,1,2* Cagri Besirli,3 Kamron Khan,4 Yesa Yang,1,2 Chien Wong,1,2,5,6 Jose Sahel,7 Mahmood Shah,7 James Tee,1,2 Neruban Kumara
Slide 2: Financial Disclosures
Financial Disclosures Michel Michaelides, MD (presenter) • Consultant: Acucela, Stargazer Pharmaceuticals, 2C Tech, MeiraGTx, Janssen Pharmaceuticals • PI: Acucela, ProQR, MeiraGTx • Equity ownership: MeiraGTx 2
Slide 3: Ten Participants From Natural History Study MGT011 Enrolled in MGT009 Phase 1/2 Trial
Ten Participants From Natural History Study MGT011 With RPGR-associated X-linked Retinitis Pigmentosa (RPGR-XLRP) Enrolled in MGT009, a Phase 1/2 Trial of an AAV5-RPGR Gene Therapy (Botaretigene Sparoparvovec) MGT011 (NCT03349242): MGT009 (NCT03252847): Multicenter open-label Phase 1/2 trial of Natu
Slide 4: Baseline Characteristics of MGT011 Participants Enrolling in MGT009 (Dose-escalation Phase)
Baseline Characteristics of MGT011 Participants Enrolling in MGT009 (Dose-escalation Phase) Mean age (range), Mean visual acuity Cohort Patients Ethnic Origin years (range) 8 White Total 24 (18, 30) 69 (52, 83) 10 1 Black 1 Other Low dose 27 (24, 30) 62 (52,70) 3 3 White 3 White Intermediate dose 25
Slide 5: Mean Retinal Sensitivity in Intermediate Dose Patients Improved Post-intervention, Reversing a Downward Trajectory
Mean Retinal Sensitivity in Intermediate Dose Patients Improved Post-intervention, Reversing a Downward Trajectory dB, decibels. At Month 12 post-treatment, the treated eye in patients at the intermediate dose demonstrated improvements in mean retinal sensitivity derived from Octopus perimetry, achi
Slide 6: V30 in Intermediate Dose Patients Improved Post-intervention, Reversing a Downward Trajectory
V30 in Intermediate Dose Patients Improved Post-intervention, Reversing a Downward Trajectory dB-sr, decibel-steradians; V30, 30-degree hill of vision. At Month 12 post-treatment, the treated eye in patients at the intermediate dose demonstrated improvements in perimetry- derived V30, achieving leve
Slide 7: V30 in Untreated Eye in Study MGT009 Decreases at a Similar Rate to Natural History
V30 in Untreated Eye in Study MGT009 Decreases at a Similar Rate to Patients in Natural History Study MGT011 Number of participants by treatment and visit Group Baseline Month 3 Month 6 Month 9 Month 12 Month 18 Month 24 Treated eye (low and intermediate doses) 6 6 5 5 6 NA NA in study MGT009 Untrea
Slide 8: Conclusions
Conclusions • At 12 months post-intervention, treatment with the low and intermediate doses of AAV5-RPGR resulted in clinically meaningful improvements in retinal sensitivity across multiple metrics and modalities • For the intermediate dose cohort, intervention with AAV5-RPGR therapy in the poorer-
Slide 9: 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. 9