Meiragtx Holdings Plc · NASDAQ:MGTX
Meiragtx — Investor Presentation (August 2026)
97 slides from Meiragtx's investor presentation, August 2026
Slide 1: Corporate Presentation
Corporate Presentation August 2026 1
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this presentation that do not relate to matters of historical fact should be considered forward-looking statements,
Slide 3: Company Overview
Company Overview G MEIRAGx
Slide 4: MeiraGTx: late-stage clinical pipeline and end-to-end capabilities in genetic medicine
MeiraGTx: late-stage clinical pipeline and comprehensive end-to-end capabilities & technologies in genetic medicine Diverse Program End-to-End GMP Next-Generation Transformative in vivo Pipeline Manufacturing Vector Optimization production Technology Broad pipeline across neuro, In-house manufacturi
Slide 5: Broad Pipeline of Transformative Genetic Medicines
Broad Pipeline of Transformative Genetic Medicines Advanced clinical programs across multiple therapeutic areas Phase 3 / Product Indication Preclinical Phase 1 Phase 2 Registrational Salivary Gland Radiation-induced xerostomia Breakthrough Designation, RMAT, Orphan Drug Drug AAV-AQP1 Sjögren’s dise
Slide 6: 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: End-to-end internal Two flexible & scalable cGMP vector production facilities, London, UK a
Slide 7: Comprehensive vector engineering technologies
Comprehensive vector engineering technologies Potency & safety optimization and precise control of gene expression Promoter engineering & discovery In-house vector engineering platforms >250k promoter library Combine rational design, massive high throughput screening, and AI to optimize Extensive in
Slide 8: Riboswitch platform: precise in vivo production of therapeutic proteins
Riboswitch platform: precise in vivo production of therapeutic proteins using oral small molecule inducers Riboswitch technology enables unprecedented Riboswitch technology can be applied precise in vivo delivery of biologic therapeutics across many therapeutic areas and with orally administered ind
Slide 9: Broad Applicability of Riboswitch Technology
Broad A Applicability broad of Riboswitch range of therapeutic Technology proteins encoded by Riboswitch-containing transgenes show tight control via oral small molecule dosing, in vivo Therapeutic Cell Therapy Therapeutic Gene/RNA Editing Antibodies Hormones/Cytokines Nucleases / Peptides • Anti-PC
Slide 10: Four pivotal stage programs in prevalent and rare indications
Four pivotal stage programs in prevalent and rare indications 01 02 03 04 Radiation-Induced X-Linked Retinitis AIPL1-Associated Parkinson’s Xerostomia Pigmentosa (RPGR) Congenital Blindness Disease • Completed Enrollment of • Completed Phase 3 • Developed under ‘specials’ • Phase 3 ready Pivotal Pha
Slide 11: Strong industry partnerships
Strong industry partnerships In November 2025, MeiraGTx entered into In October 2023, MeiraGTx received a $30 MeiraGTx entered into a strategic a broad strategic collaboration with Eli million strategic investment from Sanofi collaboration with Hologen AI to Lilly to develop and commercialize throug
Slide 12: Late-stage clinical programs
Late-stage clinical programs AAV-AQP1 for treatment of grade 2/3 xerostomia: Pivotal Study enrollment completed in 2Q 2026 Bota-vec for the treatment of X-Linked Retinitis Pigmentosa (XLRP): Phase 3 complete, BLA and MAA filing preparation under way AAV-AIPL1 for treatment of LCA4: potential near-te
Slide 13: AAV-AQP1 for treatment of xerostomia - pivotal Phase 2 enrollment complete
AAV-AQP1 for treatment of xerostomia - pivotal Phase 2 enrollment complete Large patient population with no effective treatment options MECHANISM OF ACTION Radiation-Induced Xerostomia (RIX) Pivotal Phase 2 study completed enrollment in 2Q • Saliva-producing cells are vulnerable to Thalamus Ionizing
Slide 14: AAV-GAD for treatment of Parkinson's disease - Phase 3 ready
AAV-GAD for treatment of Parkinson’s disease - Phase 3 ready Meaningful clinical improvements with evidence of disease modification MECHANISM OF ACTION AAV injected into The only CNS gene therapy program Subthalamic • AAV-GAD delivers a functional copy of the Glutamic Acid Nucleus with two randomize
Slide 15: AAV-AIPL1 for treatment of LCA4 congenital blindness
AAV-AIPL1 for treatment of LCA4 congenital blindness Restoring vision in children who are born legally blind MECHANISM OF ACTION AIPL1 Retinal Dystrophy (LCA4)1 • AAV8-AIPL1 delivers the wild-type human AIPL1 Thalamus • Children with AIPL1-associated retinal gene under control of a photoreceptor-spe
Slide 16: AAV-AQP1: a pipeline in a product for treatment of xerostomia
AAV-AQP1: a pipeline in a product for treatment of xerostomia Pivotal Phase 2 Study enrollment complete in radiation induced xerostomia Granted RMAT and Orphan Drug designations Alignment with FDA on pivotal clinical trial design and CMC for BLA filing
Slide 17: Pipeline in a product: multiple prevalent indications with high unmet need
Pipeline in a product: multiple prevalent indications with high unmet need 01 02 03 04 Radiation-induced Xerostomia associated Sjögren’s-related Prevention of xerostomia with Radioligand xerostomia radiation-induced Therapy xerostomia • Pivotal Phase 2 enrollment • Xerostomia is the most • Prevalent
Slide 18: AAV-AQP1 Long Term Data and Market Review
AAV-AQP1 Long Term Data and Market Review April 16, 2026 18
Slide 19: Patient experience and disease burden: How bad is RIX really?
Patient experience and disease burden: How bad is RIX really? Very bad indeed. Persistent, late, RIX is a severe, untreatable, lifelong condition with devastating consequences for >30% of survivors of head and neck cancer. ➢ Extreme persistent dry mouth, inability to swallow or chew, loss of sense o
Slide 20: Etiology of Persistent RIX and Population Size
Etiology of Persistent RIX and Population Size Persistent RIX is the most frequent and severe consequence of curative radiation treatment for head & neck cancer ➢ Almost all patients treated with radiation for H&N cancer experience acute xerostomia at the time of radiation ➢ In 60%-70% of patients,
Slide 21: Mechanism of Action of AAV-AQP1 for treatment of Late RIX
Mechanism of Action of AAV-AQP1 for treatment of Late Radiation-induced Xerostomia (RIX) AAV-AQP1 Delivery Procedure: ➢ Salivary glands are particularly vulnerable to radiation ➢ Small dose delivered locally directly to salivary gland ➢ Damage to salivary glands during radiation leads to acute xeros
Slide 22: Phase 1 (n=24): Transformative Clinical Improvements in Xerostomia PRO and Saliva Flow
Phase1 (n=24): Transformative Clinical Improvements in Xerostomia PRO (XQ) as well as Objective Saliva Flow (UWSFR) Endpoints Xerostomia Clinical Definition: Xerostomia Endpoints: ➢ Xerostomia is a purely patient reported condition ➢ Xerostomia Questionnaire (XQ) is the ➢ The level of xerostomia sym
Slide 23: Phase 1 AQUAx Clinical study of AAV-AQP1
Phase 1 AQUAx Clinical study of AAV-AQP1 for treatment of radiation induced xerostomia
Slide 24: AQUAx: Phase 1 Clinical Study Design
AQUAx: Phase 1 Clinical Study Design • Open-label, multi-center, dose- Primary endpoint escalation study (4 sites, US/Canada) Cohort Dose • One-time administration of AAV-AQP1 • Safety Unilateral treatment to one (unilateral) or both (bilateral) parotid glands 1 1 × 1011 vg/gland Secondary endpoint
Slide 25: SAFETY: Primary Endpoint of Phase 1 Study
SAFETY: Primary Endpoint of Phase 1 Study ➢ AAV2-hAQP1 was generally safe and well-tolerated at all doses tested ➢ No treatment-related serious adverse events ➢ No dose-limiting toxicities ➢ No participant discontinued from the study ➢ 6 mild, treatment-emergent treatment-related adverse events (TEA
Slide 26: AQUAx: Xerostomia Questionnaire 12-month change from baseline
AQUAx: Xerostomia Questionnaire (XQ) 12 month change from baseline ❑ 8 symptom-specific xerostomia PRO answered by patients with a total Average change in XQ score maximum score of 80 points (higher is worse) ❑ An improvement (decrease) of ≥8 points is considered clinically Mean change from baseline
Slide 27: AQUAx: Unstimulated whole saliva flow rate average percent change
AQUAx: Unstimulated whole saliva flow rate average percent change from baseline at 12 months pooled unilateral and bilateral cohorts At Month 12, the Unstimulated Whole Average percent change from baseline Saliva Flow Rate increased by 112.5% from baseline 120% Average % change from baseline 100% 11
Slide 28: Biopsies indicate that AAV2-hAQP1 persists in the salivary gland
Biopsies indicate that AAV2-hAQP1 persists in the salivary gland Dose Participant Cohort per Visit of Biopsy Copy #/ng DNA Copy #/Cell • Core needle biopsies were obtained in 7 participants who gland enrolled in a NIH Phase 1 study of AAV2-hAQP1 (MGT001). AAV001 1 1E10 18 Months 160 0.96 • 6/7 biops
Slide 29: AAV-AQP1: Phase 1 data out to 3 years
AAV-AQP1: for treatment of xerostomia Phase 1 data out to 3 years on all cohorts
Slide 30: XQ improvements maintained out to 3 years
Improvements in Xerostomia Questionnaire (XQ) were maintained out to 3 years, demonstrating significant durability of AAV-AQP1 The transformative Average change in XQ score from baseline improvement in the XQ PRO observed in Month 12 were maintained through Month 36 Bilaterally-treated participants
Slide 31: Waterfall Plots of individual subject XQ scores at 12 months
Waterfall Plots of individual subject XQ scores at 12 months: Bilateral and Unilateral: Absolute change and Percentage change Absolute Change from Baseline Percent Change from Baseline 31
Slide 32: Individual patient data demonstrate durable clinical response over 3 years
Individual patient data demonstrate durable clinical response over 3 years Bilateral cohort: Change from baseline (XQ) Unilateral cohort: Change from baseline (XQ) Robust Clinical Response: almost all patients experienced a significant improvement in xerostomia symptoms, with some patients reporting
Slide 33: Waterfall Plot of individual XQ score at each visit over 36 months
Waterfall Plot of individual subject XQ score at each visit over 36 months showing the consistency of individual patient responses Individual Patient XQ score at each visit out to 36 months: ❖ The graphs shows the XQ scores for each patient at 12,18, 24 and 36 months and illustrates the consistency
Slide 34: Waterfall Plots of individual XQ scores: Bilateral and Unilateral Combined
Waterfall Plots of individual subject XQ scores at 12 months: Bilateral and Unilateral Combined Individual Patient XQ score at each visit out to 36 months: the graphs shows the XQ scores for each patient grouped together at 12,18, 24 and 36 months and illustrates the consistency in XQ response for e
Slide 35: UWSFR: Average Change from Baseline out to Month 36
Unstimulated Whole Saliva Flow Rate (UWSFR) : Average Change from Baseline out to Month 36 in all treated patients Visit Baseline Day 2 Day 8 Day 15 Day 30 Day 60 Day 90 Day 180 Month 12 Month 18 Month 24 Month 36 N_Overall 15 13 13 14 15 15 15 15 15 13 13 13 N_Bilateral 11 11 10 10 11 11 11 11 11 1
Slide 36: Waterfall Plot of each individual subject UWSFR out to 36 months
Waterfall Plot of each individual subject UWSFR at each visit out to 36 months with subjects showing the overall consistency of individual patient responses ➢ Individual Patient UWSFR score at each visit out to 36 months: the graphs shows the UWSFR for each patient r at 12,18, 24 and 36 months and i
Slide 37: Pivotal MGT-AQP1-201 Study (AQUAx2)
Pivotal MGT-AQP1-201 Study (AQUAx2) Written alignment with FDA on AQUAx2 design to support BLA filing Dose 1 (0.4×1012 vg/mL), n=46 Cohort 1 Dose 2 (1.2×1012 vg/mL), n=46 Placebo (diluent), n=46 Randomization Dose 3 (2.2×1012 vg/mL), n=46 Cohort 2 Dose 4 (5.0×1012 vg/mL), n=46 Placebo (diluent), n=4
Slide 38: Study Investigator Discussion of Disease Burden, Patient Experience, and Treatment Administration
Study Investigator Discussion of Disease Burden, Patient Experience, and Treatment Administration David Owens, MBCHB, FRCS, MPHIL, PGDME, FFST(Ed) Consultant Otolaryngologist, University Hospital of Wales, Cardiff, UK.
Slide 39: David Owens: Impact of Radiation-induced Xerostomia and Limitations of Current Therapies
David Owens: Impact of Radiation-induced Xerostomia and Limitations of Current Therapies Severe Clinical Consequences Grade 2/3 radiation-induced xerostomia causes constant dryness, pain, sores, swallowing difficulties, and altered taste. • Lifelong condition with profound impact on both patient and
Slide 40: Clinical Experience, Feasibility, and Implications for Practice
Clinical Experience, Feasibility, and Implications for Practice Clinical Trial Outcomes Minimally Invasive Procedure AQUAx Phase 1 trial showed promising efficacy, safety, and long-term Retrograde intraductal delivery of durable benefits for patients. AAV-hAQP1 is straightforward, tolerable, and typ
Slide 41: Study Investigator discussion of disease burden, patient experience, and treatment administration
Study Investigator discussion of disease burden, patient experience, and treatment administration Michael Brennan, DDS, MHS Professor and Chair, Department of Oral Medicine/Oral & Maxillofacial Surgery, Wake Forest University School of Medicine Atrium Health Carolinas Medical Center, Charlotte, NC
Slide 42: Dr. Brennan: Transformative, Disease-Modifying Potential
Dr. Brennan: Transformative, Disease-Modifying Potential for A Serious, Lifelong Debilitating Disorder Debilitating Side Effect of Radiotherapy One-Time Gene Therapy Targeting the Root Cause Radiation-induced xerostomia occurs in virtually all patients receiving of the Disorder radiation treatment f
Slide 43: Global Survey of Physicians and Payors with 3 year durability data
Global Survey of Physicians and Payors with 3 year durability data Commercial Opportunity
Slide 44: Clinicians view strong response rates and durability as highly meaningful
Clinicians view the strong response rates, curative treatment, and durability of effect as highly meaningful in this unmet need Clinician Preference Share: ~78% global adoption based on clinician preference Physicians highlight: Clinician-stated preference ~78% ➢ Transformative benefit in meaningful
Slide 45: Late moderate to severe RIX: unmet need and large potential market
Late moderate to severe RIX: A completely unmet need and large potential market Peak global revenue is estimated to be ~$3.7B, with a steady state of ~$3.2B • Strong responses in meaningful measures: PRO and salivary flow, long-lasting benefit following a simple one-time treatment Clinician perspect
Slide 46: Access to >60% of the U.S. population >55 years old with HNC
Access to >60% of the U.S. population >55 years old with HNC by targeting a concentrated set of 15 major metro areas with a 3-hour driving catchment radius U.S. Population and HNC hospitals Key assumptions and inputs • HNC is distributed evenly across the U.S. population • Population >55 years consi
Slide 47: MeiraGTx can optimize reach across leading head and neck cancer centers
Of the leading head and neck cancer centers, MeiraGTx can optimize reach based on the cumulative population addressed; diminishing incremental reach is seen after ~17 hospitals in the top 25 Optimized number of sites that 30% 40% 50% 60% >60% account for ~X% of the population: Note: *>80% of HNC is
Slide 48: AAV-AQP1: Program highlights
AAV-AQP1: Program highlights • One-time, minimally-invasive, local delivery of a single, small dose delivered through an outpatient cannulation procedure with which AAV-AQP1 has the potential to become the standard of ENTs and dentists trained in oral medicine are familiar with care for patients wit
Slide 49: Significant commercial opportunity for a first-in-class treatment of persistent RIX
Significant commercial opportunity for a first-in-class treatment for large population of persistent RIX with severely debilitating unmet need 435K JP SIGNIFICANT PATIENT POPULATION 19% Annual peak sales (late RIX only) 435k patients currently of $3.7 billion globally US ANNUAL PEAK living with pers
Slide 50: 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 51: Bota-vec Opportunity Overview
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 52: 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 53: Strong physician and patient advocacy group 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 54: 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 55: 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 56: 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 57: Bota-vec Phase 3: benefit in multiple 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 58: Low luminance visual acuity: 46% vs 7% achieving ≥10 letter gain
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 59: Illustrative Depiction of Effect of bota-vec Treatment
Illustrative Depiction of Effect of bota-vec Treatment: 15 letters = 3 lines on the ETDRS chart 59
Slide 60: 40% of Treated Patients Showed Improvement in ≥2 Endpoints vs 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 61: 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 62: 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
Slide 63: AAV-GAD: a first-in-class genetic medicine for treatment of Parkinson's disease
AAV-GAD: a first-in- class genetic medicine for treatment of Parkinson’s disease Phase 3 ready | evidence of disease modification | RMAT designation
Slide 64: Parkinson's Disease: AAV-GAD overview
Parkinson’s Disease AAV-GAD overview • • Parkinson’s disease (PD) is the second most common neurodegenerative disease Approximately one million patients in the U.S. currently living with 10M Parkinson's disease Parkinson’s patients worldwide • Individuals with PD initially respond to dopamine replac
Slide 65: AAV-GAD: a first-in-class therapy for Parkinson's Disease
AAV-GAD: a first-in-class therapy for Parkinson’s Disease Therapeutic Approach The Glutamic Acid Decarboxylase (GAD) gene is delivered locally to the STN. Approach AAV-GAD delivers the Glutamic Acid Decarboxylase (GAD) Conversion of glutamate to GABA via GAD gene locally into the sub-thalamic nucleu
Slide 66: Mechanism of action
Mechanism of action AAV-GAD circumvents need for dopamine input to suppress STN hyperactivation, resulting in improved motor function PD Brain AAV-GAD Dopaminergic pathway Cortex Cortex Glutamate Glutamate GABA GAD GABA Thalamus Thalamus Striatum SNPc Striatum SNPc Gpi AAV-GAD Gpi SNpr SNpr Restorat
Slide 67: Results from a Phase 1, dose escalation study of AAV-GAD
Results from a Phase 1, dose escalation study of AAV-GAD Study design Single-arm, open-label, dose escalation study of unilateral subthalamic administration of AAV-GAD in patients with PD (n=12) Safety • AAV-GAD was generally safe and well tolerated, with no adverse events related to the therapy • N
Slide 68: Results from a Phase 2 randomized, sham-controlled study of AAV-GAD
Results from a Phase 2, randomized, double-blind, sham-controlled, multi-center study of AAV-GAD Study design Efficacy findings (summary): • Randomized (n=45, 1:1) double-blind study of bilateral STN AAV-GAD against • Study met primary endpoint: UPDRS 3 motor score sham control in patients with adva
Slide 69: Phase 2: significant improvements following AAV-GAD vs sham surgery
Results from the Phase 2 study: significant improvements following AAV-GAD treatment compared to sham surgery control Significant improvements in UPDRS 3 Significantly greater responder rate Reduction in duration of motor scores ≥9 points UPDRS levodopa-induced dyskinesia AAV-GAD SHAM p=0.023 62.50%
Slide 70: AAV-GAD treatment is safe & well tolerated
AAV-GAD treatment is safe & well tolerated Worsening of Parkinson’s observed in sham group but not in AAV-GAD treated group Adverse events over 12 months (20% or Worsening of Parkinson’s Disease Headache greater frequency) Number of subjects ***p<0.005; Fisher Exact Test Number of subjects Worsening
Slide 71: AAV-GAD therapy shows evidence of disease modification
AAV-GAD therapy shows evidence of disease modification FDG-PET data from AAV-GAD clinical trials shows disease- Disease modification in preclinical models1 modifying effects in the basal ganglia circuitry, including the substantia nigra – the site of dopamine-producing neurons • Preclinical studies
Slide 72: Positive topline data from AAV-GAD sham surgery-controlled Bridging Study
Positive topline data summary from AAV-GAD sham surgery-controlled Bridging Study Summary of results: A 6-month, three-arm, randomized, double-blind, sham-controlled study AAV-GAD was generally safe and well tolerated with no serious adverse events related using material manufactured in- to AAV-GAD
Slide 73: Summary: AAV-GAD for treatment of Parkinson's disease
Summary: AAV-GAD for treatment of Parkinson’s disease • AAV-GAD is the only CNS gene therapy program tested in three human trials – meeting a prespecified efficacy Large market with high primary endpoint unmet need in patients no • A total of 58 patients in 3 independent multicenter longer respondin
Slide 74: AAV-AIPL1 for treatment of AIPL1-associated retinal dystrophy
AAV-AIPL1 for treatment of AIPL1-associated retinal dystrophy1 Restoring vision in children who are born legally blind Potential Path to Marketing Authorization under exceptional circumstances | RPDD, Orphan Drug Designation in US & EU 1 Partnered with Eli Lilly
Slide 75: AIPL1 video
AIPL1 video Click to play video 75
Slide 76: AAV-AIPL1 for treatment of LCA4 retinal dystrophy
AAV-AIPL1 for treatment of LCA4 retinal dystrophy1 Potential path to marketing authorization in UK and US A novel treatment for AIPL1 congenital blindness Data published in The Lancet • Children with AIPL1-associated retinal dystrophy (LCA4) are blind from birth. Watch the MeiraGTx webcast By age 4,
Slide 77: Riboswitch Gene Regulation Platform
Riboswitch Gene Regulation Platform In vivo production of vectorized therapeutic proteins and peptides with oral inducers
Slide 78: Riboswitch platform: in vivo production of therapeutic proteins via oral inducers
Riboswitch platform: precise in vivo production of therapeutic proteins via oral small molecule inducers mRNA formation is controlled by alternative splicing cassette via binding of a small molecule inducer to the Riboswitch cassette No small molecule (OFF state) With small molecule (ON state) mRNA
Slide 79: Riboswitch provides significant advantages over other inducible systems
Riboswitch provides significant advantages over other inducible systems • The native form of any biologic therapeutic can be encoded by the DNA template and produced in vivo, controlled by bespoke oral small molecules • Extremely precise dosing of biologic therapeutics with the production of the the
Slide 80: Protein Expression
Riboswitch splicing cassette drives precise control of therapeutic protein production with unprecedented dynamic range and precision Expression level of the 1800.0 Riboswitch-regulated Unregulated gene construct without the 1600.0 Maximal expression of Riboswitch regulated gene regulated gene Ribosw
Slide 81: Broad Applicability of Riboswitch Technology
Broad A Applicability broad of Riboswitch range of therapeutic Technology proteins encoded by Riboswitch-containing transgenes show tight control via oral small molecule dosing, in vivo Therapeutic Cell Therapy Therapeutic Gene/RNA Editing Antibodies Hormones/Cytokines Nucleases /Peptides • Anti-PCS
Slide 82: Precise regulation of multiple therapeutic hormones and peptides with riboswitch, in vitro
Precise regulation of multiple therapeutic hormones and peptides with riboswitch, in vitro The riboswitch platform can be applied to any transgene and delivered by any vector - achieving in vivo production of the therapeutic protein or peptide in a precise dose response to a bespoke orally administe
Slide 83: Riboswitch regulation of metabolic peptides, alone or in combination
Riboswitch regulation of metabolic peptides, alone or in combination Single Peptide Combination Peptide In vivo production of natural gut peptides Constructs Constructs • Delivery of high effective levels of active peptides can be challenging. MeiraGTx has achieved high expression of natural gut pep
Slide 84: Riboswitch regulation of combinations of gut peptides in vitro
Riboswitch regulation cassette controls the expression of combinations of gut peptides GLP-1, GIP and PYY, in vitro GLP-1 expression GIP expression PYY expression GLP-1 activity specifically on GLP-1 receptor GIP activity specifically on GIP receptor Inducer (uM) 84
Slide 85: In vivo delivery of GLP1-GIP via oral inducer improves weight loss and glucose control
In vivo delivery of GLP1-GIP via daily oral inducer dosing significantly improves weight loss and glucose control compared to constitutively expressed GLP1-GIP GLP1-GIP: comparison of constitutive expression of the dual peptides compared to daily in vivo delivery induced by oral small molecule Weigh
Slide 86: In vivo delivery of GLP1-GIP-Glucagon via oral inducer improves weight loss
In vivo delivery of GLP1-GIP-Glucagon via daily oral inducer dosing significantly improves weight loss & glucose control vs. constitutive GLP1-GIP-Glucagon Weight loss in DIO mice Glucose Control in DIO mice Start of oral inducer dosing • Untreated DIO mice show persistent weight gain over 10 weeks
Slide 87: In vivo peptide production addresses challenges in current treatment of metabolic disorders
In vivo production of peptide therapeutics addresses several challenges in current pharmacological treatment of metabolic disorders Efficacy Safety & Tolerability • Native/natural short acting peptides can • Lower levels of short acting native Short lived agonists in responsive be delivered and prec
Slide 88: Riboswitch-regulated leptin therapy (riboLeptin) - Summary
Riboswitch-regulated leptin therapy (riboLeptin) - Summary MeiraGTx is developing a new leptin therapy using its proprietary Riboswitch • Leptin is a key hormone regulating energy technology - to deliver native leptin to patients with a daily dose of an oral pill balance and metabolic homeostasis. I
Slide 89: Riboswitch-regulated leptin demonstrates dose-dependent expression in vitro
Riboswitch-regulated leptin demonstrates dose-dependent expression in vitro in mammalian cells P Leptin CDS Leptin CDS Dose-dependent leptin expression in HEK 293 cells • Riboswitch gene regulation cassette was inserted in the coding sequence (CDS) of human leptin gene • The construct was transfecte
Slide 90: Riboswitch control of leptin demonstrates dose-dependent expression in vivo in ob/ob mice
Riboswitch-small molecule control of leptin demonstrates dose-dependent expression in vivo in leptin deficient ob/ob mice Leptin In leptin-deficient mice, leptin is expressed in a dose secretion dependent manner in response to oral dosing with AAV Inducer (PO) Riboswitch inducer, MXU-001, achieving
Slide 91: riboLeptin demonstrates dose-dependent weight loss with oral inducer dosing
riboLeptin demonstrates dose-dependent weight loss in response to oral inducer dosing, preventing morbid obesity I.M. PBS AAV9.RS.Leptin riboLeptin normalizes body weight and prevents Oral M310 0 mg/kg 0 mg/kg 10 mg/kg 30 mg/kg 100 mg/kg morbid obesity in leptin-deficient mice: • AAV9 vector contain
Slide 92: Durable riboswitch-controlled leptin production in ob/ob mice over a year
Durable riboswitch-controlled production of leptin in ob/ob mice effectively treats leptin deficiency with daily oral small molecule dosing over a year after one-time IM injection of RiboLeptin • 5.5 weeks old ob/ob mice were injected with PBS or 1E11 vg AAV9.RS.Leptin (RiboLeptin) into the muscle o
Slide 93: Riboswitch-regulated leptin therapy (RiboLeptin) - Summary
Riboswitch-regulated leptin therapy (RiboLeptin) - Summary RiboLeptin resolved leptin deficiency following oral inducer treatment in ob/ob mice: ❑ Dose dependent expression of leptin to physiological levels ❑ Significant reduction in excessive food intake behavior (hyperphagia) ❑ Significant and dur
Slide 94: AAV-BDNF: small dose, locally delivered to hypothalamus
AAV-BDNF: small dose, locally delivered to hypothalamus to change dysregulated MC4R circuitry in the brain IND Enabling: clear evidence of strong efficacy in animal models
Slide 95: AAV-BDNF leads to significant weight loss in obese mice without muscle loss
Treatment with AAV-BDNF leads to significant weight loss in obese mice, without muscle loss. Works downstream of MC4R to control weight. Meaningful and durable weight loss in Significant fat reduction without Optimized, high expressing vector DIO mice affecting lean muscle mass • Vector optimization
Slide 96: MeiraGTx is leading the next wave of genetic medicines
MeiraGTx is leading the next wave of genetic medicines Powered by best-in-class genetic medicine Advanced and technologies and unique industry leading end-to-end diverse pipeline of in-house GMP manufacturing: genetic medicines Vector Design & ☑ Novel intravitreal capsids ☑ Proprietary non-coding ☑
Slide 97: Seeeceeecse
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MeiraGTx Holdings — Investor Presentation (August 2026) on 6ix