Meiragtx Holdings Plc · NASDAQ:MGTX
Meiragtx — Investor Presentation (October 2026)
24 slides from Meiragtx's investor presentation, October 2026
Slide 1: Botaretigene Sparoparvovec (bota-vec): A BLA-ready Program for RPGR XLRP
Botaretigene Sparoparvovec (bota-vec): A BLA-ready Program for treatment of RPGR XLRP
Slide 2: Bota-vec for the treatment of X-Linked Retinitis Pigmentosa (XLRP)
Bota-vec for the treatment of X-Linked Retinitis Pigmentosa (XLRP) • BILATERAL Phase 3 complete (n=95): conducted at 32 of the leading global sites for Inherited Retinal Disease (IRD) in North America, Europe, and Japan • Unilateral Phase 1/2 dose escalation followed by expansion cohort (n=45) • Bot
Slide 3: XLRP: Severe, Progressive Vision Loss with High Unmet Need
XLRP: Severe, Progressive Vision Loss with High Unmet Need >20,000 patients in US and EU X-linked retinitis pigmentosa (XLRP) is one of the XLRP represents a significant portion of the most severe forms of RP, characterized by early onset broader Retinitis Pigmentosa population being night blindness
Slide 4: Program status: Potential approvals in 2027 | In-house commercial manufacturing established
Program status: Potential approvals in 2027 | In-house commercial manufacturing established REGULATORY FILING COMMERCIAL-READY IN-HOUSE MANUFACTURING In-house commercial manufacturing at MeiraGTx Filing now facilities in London MeiraGTx was J&J's selected commercial manufacturer • EU: MAA filing sch
Slide 5: Unique end-to-end, in-house GMP manufacturing infrastructure and production platform
Unique end-to-end, in-house GMP manufacturing infrastructure and production platform process, scalable and flexible, fit for clinical & commercial supply • Best-in-class, fully end-to-end internal capabilities: Two End-to-end internal flexible & scalable cGMP vector production facilities, London, UK
Slide 6: Bota-vec Commercial End-to-End Supply Chain & CMC Capability
Bota-vec Commercial End-to-End Supply Chain & CMC Capability Strong internal MeiraGTx commercial manufacturing foundation established for successful bota-vec launch LONDON & SHANNON PROCESS GMP MANUFACTURING Integrated AAV vector Licensed production and aseptic VALIDATION DS & DP batches INFRASTRUCT
Slide 7: Strong support from KOLs and patient groups and concentrated market
Strong support from KOLs and patient groups and concentrated market • MeiraGTx is recognized as a global leader in ophthalmology gene therapy – close relationship with Moorfields/UCL Institute of Ophthalmology, one of the leading centers for IRD research and patient care globally • KOLs in US and EU
Slide 8: 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 Fighting Blindness (FFB) Open Letter Patient Voices Quoted in FFB Letter: In June 2025, the FFB published a public letter signed by 30 leading retinal specialists urging J&J to seek approval for bota-vec “T
Slide 9: Phase 3 LUMEOS study of botaretigene sparoparvovec (bota-vec)
Phase 3 LUMEOS study of botaretigene sparoparvovec (bota-vec) Summary of Data
Slide 10: Bota-vec clinical development summary
Bota-vec clinical development summary Largest global development program in an Inherited Retinal Disease (IRD) Bota-vec is the first and only gene therapy for XLRP to be Largest global development program for an IRD - completed with tested bilaterally in a randomized controlled global phase 3 compel
Slide 11: 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 12: Demographics and Baseline Characteristics by Treatment Group in Adults
Demographics and Baseline Characteristics by Treatment Group in Adults *Excludes 6 pediatric patients, adult patients only for this analysis. Baseline values were acquired during ≥1 visit prior to treatment in study MGT-RPGR-021. BCVA, best-corrected visual acuity; dB, decibels; LLVA, low-luminance
Slide 13: Safety & Tolerability
Safety & Tolerability • Expected, acceptable, and manageable safety profile consistent with other ocular AAV gene therapies • Most ocular-related AEs (86.2%) were of mild or moderate severity • Favorable safety profile with no new safety signals were reported compared with the phase 1/2 study • AAV5
Slide 14: 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 P value = 0.247 P value = 0.006 P value = 0.001 P value = 0.001 P value = 0.001 P value = 0
Slide 15: Bota-vec Phase 3 Pivotal Study Results: Highly statistically significant benefit in secondary endpoints
Bota-vec Phase 3 Pivotal Study Results: Highly statistically significant and clinically meaningful benefit in multiple secondary endpoints across every domain of vision Significant improvements in Patient Reported Outcomes (PRO) questions related to everyday function and quality of life: • IVI-A: si
Slide 16: Bota-vec Phase 3 Pivotal Study Results: Highly statistically significant benefit in secondary endpoints
Bota-vec Phase 3 Pivotal Study Results: Highly statistically significant and clinically meaningful benefit in multiple secondary endpoints across every domain of vision 16
Slide 17: Individual Results for Low Luminance Visual Acuity (LLVA) Endpoint
Individual Results for Low Luminance Visual Acuity (LLVA) Endpoint ≥10 letters difference is considered clinically meaningful based on patient and KOL feedback (45% vs. 7%) 15 letters 10 letters 5-6 letters 5-6 letters
Slide 18: Illustrative Depiction of Effect of bota-vec Treatment: 15 letters = 3 lines on the ETDRS chart
Illustrative Depiction of Effect of bota-vec Treatment: 15 letters = 3 lines on the ETDRS chart 18
Slide 19: Significant treatment benefit demonstrated in multiple perimetry measures
Significant treatment benefit demonstrated in multiple perimetry measures Pointwise responder analysis | Static perimetry | Mesopic microperimetry Retinal sensitivity, measured by perimetry, provides a quantitative assessment of the retina’s functional ability to detect light The data demonstrate re
Slide 20: Bota-vec showed significant improvements in Patient Reported Outcome (PRO) questions
Bota-vec showed significant improvements in Patient Reported Outcome (PRO) questions related to everyday function and quality of life Statistically significant improvements in mobility and function under dim-light conditions, as well as independence and emotional well being Strong concordance betwee
Slide 21: 40% (22/55) 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 22: Multi-Endpoint Responder Analysis: Comprehensive Results
Multi-Endpoint Responder Analysis: Comprehensive Results 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 PERIMETRY ENDPOINT COMBINATION TREATED CONTROL DIFF ENDP
Slide 23: Summary: Compelling Totality of Evidence Supporting Global Regulatory Approval
Summary: Compelling Totality of Evidence Supporting Global Regulatory Approval • LUMEOS – Bilateral Phase 3 study (n=95) conducted across 32 leading global clinical sites • Following presentation of the positive UNILATERAL randomized controlled Phase 1/2 study demonstrating safety and benefit in end
Slide 24: Bota-vec Opportunity Overview: High-Value, De-Risked Program with Potential Near-Term Approval and Launch
Bota-vec Opportunity Overview: High-Value, De-Risked Program with Potential Near-Term Approval and Launch TOTALITY OF PHASE 3 DATA DEMONSTRATES HIGH UNMET NEED COMMERCIAL POTENTIAL BENEFIT OF TREATMENT Bota-vec addresses a severe unmet need with no Significant market >20,000 patients in US and EU Me