Meiragtx Holdings Plc · NASDAQ:MGTX
Meiragtx — Investor Presentation (October 2026)
13 slides from Meiragtx's investor presentation, October 2026
Slide 1: Bota-vec for the treatment of X-Linked Retinitis Pigmentosa (XLRP)
Bota-vec for the treatment of X-Linked Retinitis Pigmentosa (XLRP) • Phase 3 complete (n=95) • Phase 3 conducted at 32 of the leading global sites for Inherited Retinal Disease (IRD) in North America and Europe • Granted PRIME and Fast Track designations, and US and EU Orphan Drug Designations • BLA
Slide 2: Bota-vec Opportunity Overview: High-Value, De-Risked Program
Bota-vec Opportunity Overview: High-Value, De-Risked Program with Potential Near-Term Approval and Launch Totality of Phase 3 data High Unmet Need Commercial Potential demonstrates benefit of treatment • Significant market >20,000 US and EU • Bota-vec addresses a severe unmet need • Significant impr
Slide 3: Bota-vec status: awaiting BLA and MAA filings
Bota-vec status: awaiting BLA and MAA filings Largest global development program in a severe Inherited Retinal Disease (IRD) completed with compelling data indicating statistically significant and clinically meaningful benefits across every domain of vision supporting global approval: • Phase 3 comp
Slide 4: Strong physician and patient advocacy group (FFB) support for approval of bota-vec
Strong physician and patient advocacy group (FFB) support for approval of bota-vec Foundation for Fighting Blindness (FFB) Open Letter Patient Voices from LUMEOS Trial Quoted in FFB Letter In June 2025, the FFB published a public letter signed by 30 leading retinal specialists urging J&J to seek app
Slide 5: Bota-vec commercial opportunity
Bota-vec commercial opportunity: Launch into concentrated, educated KOL market with commercial supply manufactured in-house at MeiraGTx Projected cost-effective launch into a concentrated group of educated physicians and patients globally ➢ Most IRD patients are seen by a small community of connecte
Slide 6: Phase 3 LUMEOS study of botaretigene sparoparvovec (bota-vec)
Phase 3 LUMEOS study of botaretigene sparoparvovec (bota-vec) Summary of Data Source: Oral presentation at FFB ARVO 2025 meeting and publication in preparation
Slide 7: Bota-vec Phase 3 Pivotal Study Results
Bota-vec Phase 3 Pivotal Study Results Consistent and Robust Efficacy Across Objective & Subjective Measures These Endpoints Reflect Significant and Clinically Meaningful Improvement in Every Domain of Vision *VMA is a novel assessment first used in this study. †Results trending in anticipated direc
Slide 8: Bota-vec Phase 3 Pivotal Study Results: secondary endpoints in every domain of vision
Bota-vec Phase 3 Pivotal Study Results: Highly statistically significant and clinically meaningful benefit in multiple secondary endpoints every domain of vision Significant improvements in Patient Reported Outcomes (PRO) questions related to everyday function and quality of life: • IVI-A: significa
Slide 9: Low light visual acuity (LLVA) results at week 52
Impressive results for Visual Acuity measured in low light levels (LLVA) with 46% of treated patients achieving ≥10 letter gain on ETDRS chart compared to 7% for untreated controls ≥10 letters difference is considered clinically meaningful based on patient and KOL feedback (46% vs. 7%) Change in Low
Slide 10: Illustrative Depiction of Effect of bota-vec Treatment
Illustrative Depiction of Effect of bota-vec Treatment: 15 letters = 3 lines on the ETDRS chart 10
Slide 11: 40% of Treated Patients Showed Improvement in ≥2 Endpoints Compared to 0% in Controls
40% (22/55) of Treated Patients Showed 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 (
Slide 12: Multi-Endpoint Responder Analysis: Comprehensive Results
Multi-Endpoint Responder Analysis: Comprehensive Results FF = Full Field; Pt = Pointwise; binoc = binocular. Consistent 25–40% treatment benefit across ALL endpoint combinations with most combinations showing 0% response in control arm SECTION 1: VMA + LLVA COMBINATIONS SECTION 3: LLVA + STATIC PERI
Slide 13: Conclusions
Conclusions • Large Phase 3 study, 32 leading global sites (n=95). Although the novel primary endpoint was not statistically significant, there was a positive trend with the treated group 2.4x more likely to respond than untreated. • Unprecedented clinically meaningful and statistically significant
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