Meiragtx Holdings Plc · NASDAQ:MGTX
Meiragtx — Foundation Fighting Blindness (FFB) Retinal Therapeutics Innovation Summit 2025
14 slides from Meiragtx's conference presentation, May 2, 2025
Slide 1: Update on Bota-vec Efficacy and Safety Data in Patients With RPGR-XLRP
Update on Bota-vec Efficacy and Safety Data in Patients With RPGR-XLRP Michael Clark,1 Cagri Besirli,1 Peggy Wong,1 Jialin Xu,1 Leslie Patch,1 Saskia Houwing,1 Pansy Minnick,1 Colleen Peluso,1 Marla Sultan,1 Rachel Huckfeldt,2 James Bainbridge,3,4 Michel Michaelides3,4 1Johnson & Johnson, Raritan, N
Slide 2: Financial Disclosures
Financial Disclosures Michael Clark, MD (presenter) • Employee: Johnson & Johnson • Equity ownership: Johnson & Johnson 2 2 Presented by M Clark at Foundation Fighting Blindness (FFB) Retinal Therapeutics Innovation Summit; May 2, 2025; Salt Lake City, UT, United States
Slide 3: Background
Background • XLRP is a severe form of retinitis pigmentosa that leads to progressive vision loss1 – XLRP is characterized by early-onset and rapid symptom progression – The most common form of XLRP is caused by variants in the RPGR gene (RPGR-XLRP) • AAV5-hRKp.RPGR (botaretigene sparoparvovec; bota-
Slide 4: Phase 3 Randomized, Controlled Study of Bota-Vec Followed by Long-Term Follow-Up
Phase 3 Randomized, Controlled Study of Bota-Vec Followed by Long-Term Follow-Up Phase 3 MGT021 • 95 participants were randomized to low dose, high dose or deferred treatment with bota-vec • Primary objective: to assess the effect of bilateral treatment with bota-vec on functional vision as measured
Slide 5: Demographics and Baseline Characteristics by Treatment Group in Adults
Demographics and Baseline Characteristics by Treatment Group in Adults Control (deferred treatment) Pooled treated group group Characteristic (n = 58)* (n= 31)* Age, mean (range), y 28.7 (18, 53) 25.6 (18, 45) 96.6% male 96.8% male Sex, % 3.4% female 3.2% female BCVA, mean (SD) 66.6 (9.6) 70.9 (7.8)
Slide 6: Bota-vec Phase 3 MGT021 Pivotal Study Results
Bota-vec Phase 3 MGT021 Pivotal Study Results Category Functional Vision Retinal Function Visual Function Safety Assessment VMA* PRO: LLQ Static Static Retinal BCVA Low Intraocular (binocular) Extreme lighting perimetry perimetry sensitivity (ETDRS luminance inflammation PRIMARY domain score respon
Slide 7: Functional Vision: VMA-Binocular Responders at Week 52
Functional Vision: VMA-Binocular Responders at Week 52 Subgroup Analysis by MRS Strata • Similar % responders* in pooled treated group between two MRS strata levels • Fewer responders in control group in MRS >10 dB, compared to MRS ≤10 dB Perc entage of VM A respon ders overa ll an d 40 by MR S stra
Slide 8: Retinal Function: Mean Retinal Sensitivity in Static Perimetry
Retinal Function: Mean Retinal Sensitivity in Static Perimetry (2nd Secondary) Cha nge from baseline in MR S10 overall and by M RS strata levels at Week 52 2.0 LS mean (95% CI) change from baseline in MRS10 excluding scotoma (dB) 1.5 1.0 0.88 0.87 0.76 0.5 0.0 ‒0.5 ‒0.36 ‒0.36 ‒0.32 ‒1.0 ‒1.5 Overal
Slide 9: LLVA Responders (≥15 Letters) Present at Week in Study MGT021
LLVA Responders (≥15 Letters) Present at Week 12 in Study MGT021 (Worse-Seeing Eye) Perc entage of LLVA respon ders 40 (≥15 letters) over tim e change ≥15 letters in worse-seeing eye Percentage of participants with LLVA 35 29% 30% 30 27% 25 20% 20 15 10 5 3% 14/48 0% 15/55 0% 16/54 0% 11/55 1/30 0 W
Slide 10: LLVA Responders (≥10 Letters) Present at Week in Study MGT021
LLVA Responders (≥10 Letters) Present at Week 12 in Study MGT021 (Worse-Seeing Eye) Perc entage of LLVA respon ders change ≥10 letters in worse-seeing eye 60 Percentage of participants with LLVA (≥10 letters) over tim e 50% 49% 50 46% 43% 40 30 20 10 7% 7% 7% 3% 24/48 1/29 27/55 2/30 23/54 2/29 25/5
Slide 11: 40% (22/55) of Treated Patients Show Improvement in ≥2 Endpoints Compared to 0% in Controls
40% (22/55) of Treated Patients Show Improvement in ≥2 Endpoints Compared to 0% in Controls Pooled treated group (n = 55) Control group (n = 30) VMA LLVA ≥10 VMA LLVA ≥10 binocular worse eye binocular worse eye (n=15) Functional 3 Visual (n=25) Functional 0 Visual Vision Function Vision Function (5%
Slide 12: Clinical Safety and Adverse Events in Adults
Clinical Safety and Adverse Events in Adults • Overall, 100% of treated participants had ≥1 TEAE, Pooled most (86%) of which were mild or moderate Control treated group • 53% had ≥1 TEAE considered to be related to group (n = 31) bota-vec, and 98% had ≥1 TEAE related to the Participants*, n (%) (n =
Slide 13: Conclusions
Conclusions • Primary endpoint (VMA*) was not met but was directionally supportive • Majority of secondary endpoints, including data from all three visual domains, support that bota-vec leads to clinical improvements in vision • Response between the two doses (RPGR2e11 group [low dose] and RPGR4e11
Slide 14: Acknowledgments
Acknowledgments Big Thank You to: Participants and families Referring clinicians and collaborators Trial managers, coordinators, and technicians Reading centers: Boston Image Reading Center and Casey Eye Institute Other supporting institutions: Amsterdam University Medical Center, Careggi University