MeiraGTx Holdings plc · NASDAQ:MGTX

MeiraGTx — Technical Presentation (April 2024)

16 slides from MeiraGTx's technical presentation, April 2024

  1. Slide 1: Results of a Phase 1 Study of AAV2-hAQP1 – The AQUAx Study

    Results of a Phase 1, Open-label, Dose-escalation Study of Gene Therapy with AAV2-hAQP1 as Treatment for Grade 2 and 3 Radiation-induced Late Xerostomia and Parotid Gland Hypofunction – The AQUAx Study Oral Abstract I Michael T Brennan, DDS, MHS [email protected]

  2. Slide 2: Disclosures

    Disclosures • I have received financial support from MeiraGTx, LLC for research studies • I served as an investigator in the completed MGT016 (AQUAx) study, sponsored by MeiraGTx, LLC • I currently serve as an investigator in the ongoing MGT-AQP1-201 (AQUAx2) study, sponsored by MeiraGTx, LLC 2

  3. Slide 3: Radiation-Induced Xerostomia (RIX)

    Radiation-Induced Xerostomia (RIX) • RIX is one of the most frequent complications of radiation treatment for head and neck cancer • IMRT has reduced the incidence of RIX, but it still affects >50% of those completing radiotherapy for head and neck cancer • Persistent Grade 2/3 (Moderate/Severe) RIX

  4. Slide 4: Significant Unmet Medical Need for an Effective RIX Treatment

    Significant Unmet Medical Need for an Effective RIX Treatment Prevalence (US) Incidence (US) 170K 15K • >170,000 patients with long-term (i.e., at least 2 years post radiation treatment) grade 2/3 RIX in the US alone1,2,3 Grade 2/3 RIX patients in New cases of grade 2/3 the US RIX annually in the US

  5. Slide 5: AAV2-hAQP1 Mechanism of Action

    AAV2-hAQP1 Mechanism of Action • Acinar cells are particularly vulnerable to radiation treatment • Acinar cell death and disorganization of gland epithelium following Ionizing radiation radiation results in hyposalivation causes irreversible damage to acinar cells, • Expression of the water channel,

  6. Slide 6: AQUAx: Phase 1 Clinical Study Design

    AQUAx: Phase 1 Clinical Study Design • Open-label, multi-center, dose-escalation study (4 sites, US/Canada) • One-time administration of AAV-hAQP1 to one (unilateral) or both (bilateral) parotid glands Cohort Dose • Four dose-escalating cohorts with 3 participants per cohort (n=12 for unilaterally t

  7. Slide 7: AQUAx: Demographics and Baseline Characteristics

    7 AQUAx: Demographics and Baseline Characteristics • 24 Participants • 20 Male, 4 Female • 23 White, 1 Black/African American • Average Age: 63.5 years (range 48-79) • 5+ years out from final radiotherapy treatment (2+ years for HPV+ tumors) • Average baseline Total Xerostomia Questionnaire (XQ) Sco

  8. Slide 8: AQUAx: Safety

    AQUAx: Safety Treatment-Related Treatment-Emergent Adverse Events in the AQUAx study All Participant • AAV2-hAQP1 was safe and well-tolerated at all System Organ Class N=24 doses tested Preferred Term N (%) • No treatment-related serious adverse events Participants with >1 treatment-related TEAE 6 (

  9. Slide 9: Average Change in XQ Score

    AQUAx: Xerostomia Questionnaire1 • 8 symptom-specific questions which Average Change in XQ Score the participant answers using a scale from 0 (not present) to 10 (worst possible) • Responses to individual questions are summed to provide the Total Score (0-80), an overall measure of disease burden •

  10. Slide 10: Average Change in DM Score

    AQUAx: MD Anderson Symptom Inventory Dry Mouth Question Average Change in DM Score • Question #10 from MD Anderson Symptom Inventory – Head and Neck1 • During the last 24 hours, please rate “Your dry mouth at its WORST” • Scale from 0 (not present) to 10 (as bad as you can imagine) Average Dry Mouth

  11. Slide 11: Average Change in GRCQ Score

    AQUAx: McMaster Global Rate of Change Questionnaire Score • Participants are asked, Average Change in GRCQ Score “Overall, has there been any change in your Dry Mouth since you received study treatment?” • Potential answers are “Better”, “About the Same”, or “Worse” • If they answer "Better" or "Wor

  12. Slide 12: Consistent Improvements across Patient Reported Outcome Measures

    AQUAx: Consistent Improvements across Patient Reported Outcome Measures Percent Change in PRO Score CONFIDENTIAL 12

  13. Slide 13: Unstimulated Whole Saliva Flow Rate

    AQUAx: Unstimulated Whole Saliva Flow Rate Average Percent Change from Baseline 120% 112.5% 100% 86.5% 80% Average % Change from Baseline 74.5% 62.7% 60% 54.5% 56.2% 40% 20% 7.3% 0% -13.0% -20% -40% 0 2 4 6 8 10 12 Months Post Treatment At Month 12, the Unstimulated Whole Saliva Flow Rate increased

  14. Slide 14: AQUAx - Summary of Findings

    AQUAx - Summary of Findings • No treatment-related serious adverse events or dose-limiting toxicities were reported, and all participants completed the study • The 3 different PRO instruments showed statistically significant improvements by Day 30 that were maintained through Month 12 • At Month 12,

  15. Slide 15: AQUAx2: Phase 2 Study Design and Endpoints

    AQUAx2: Phase 2 Study Design and Endpoints The Phase 2 randomized, double-blind, placebo-controlled study is actively enrolling Study Design Primary Efficacy Endpoint • Randomized, double-blind, placebo-controlled • Change from Baseline to Month 12 in Xerostomia Questionnaire (XQ) Total Score • 120

  16. Slide 16: Q & A

    Q&A https://aquax2study.com/

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