Meiragtx Holdings Plc · NASDAQ:MGTX
Meiragtx — Technical Presentation (December 2021)
22 slides from Meiragtx's technical presentation, Dec 7, 2021
Slide 1: Gene Therapy for Radiation-Induced Xerostomia: AAV-hAQP1 Program Update
CONFIDENTIAL Gene Therapy for the Treatment of Radiation-Induced Xerostomia: AAV-hAQP1 Program Update December 7, 2021
Slide 2: Forward-looking statements
CONFIDENTIAL 2 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, including,
Slide 3: Agenda and presenters
CONFIDENTIAL 3 1. Introduction Zandy Forbes, PhD President & CEO MeiraGTx 2. Radiation-Induced Xerostomia: Disease Background and Unmet Medical Need Robert K. Zeldin, MD Chief Medical Officer MeiraGTx 3. AQUAx Phase 1 Study: Preliminary Data for AQUAx Cohorts 1 to 3 Zandy Forbes, PhD President & CEO
Slide 4: Salivary Gland Gene Therapy Radiation-Induced Xerostomia
Salivary Gland Gene Therapy Radiation-Induced Xerostomia Robert K. Zeldin, MD
Slide 5: Radiation-induced xerostomia disease background
CONFIDENTIAL • 85% of radiation-treated patients experience reduced saliva production, 50% of whom have persistent Grade 2/3 RIX1 • >170,000 existing patients in the US with Grade 2/3 RIX (Orphan Status)1,2 • 54,000 new cases of head and neck cancer per year in the US; 650,000 worldwide3 • Progressi
Slide 6: Limited current treatment options for xerostomia
CONFIDENTIAL 6 ❑ Saliva substitutes • Carboxymethyl cellulose and mucin • Short term benefit ❑ Mechanical and Gustatory stimulation • Not all patients tolerate frequent gum chewing • May exacerbate temporomandibular disorder symptoms ❑ Sialogogues: Pilocarpine (indicated for RIX) and Cevimeline (off
Slide 7: FDA Patient Focused Drug Development meeting
CONFIDENTIAL 7 Xerostomia-focused meeting hosted by a consortium of advocacy and patient care groups with guidance from the US FDA on August 19th, 2021 • The goal of Patient Focused Drug Development meetings is to systematically obtain the patient perspective on specific diseases and their treatment
Slide 8: Patient voices on xerostomia
CONFIDENTIAL 8 “Sometimes the sticky saliva closes the back of my throat, and I can’t speak for up to a minute. My throat is just stuck, and I’m not able to get words out.” “Aside from painful swallowing, I’ve had a couple of incidents of almost choking due to food getting stuck in my throat.” “Alon
Slide 9: AAV-hAQP1 MGT016 AQUAx Phase 1 Study
AAV-hAQP1 MGT016 AQUAx Phase 1 Study
Slide 10: AQUAx study design
CONFIDENTIAL 10 Study Design Open label, multi-center, dose escalation study of a single administration of AAV- hAQP1 to one or both parotid glands in patients with radiation-induced parotid salivary hypofunction and xerostomia • Four unilateral treated escalating dose cohorts with a minimum of 3 su
Slide 11: Study status
CONFIDENTIAL 11 Study Status • All centers open for enrollment • All four unilateral dose cohorts treated (n=12) • One bilateral dose cohort treated (n=3) • Completion of enrollment of bilateral cohorts in the coming months 7 participants (3 each from Cohorts 1 & 2 and 1 from Cohort 3) have data ava
Slide 12: Phase 2 study design
CONFIDENTIAL 12 Study Design: • Randomized, double-blind, placebo-controlled study • Two active doses of AAV2-hAQP1 Primary Endpoint: • McMaster Global Rate of Change at 12 months after treatment Secondary Endpoint: • Symptom-specific Xerostomia Questionnaire Exploratory: • Whole saliva volume
Slide 13: AAV-hAQP1 Clinical Development Program: Preliminary Data for AQUAx Cohorts 1 to 3
AAV-hAQP1 Clinical Development Program: Preliminary Data for AQUAx Cohorts 1 to 3 Zandy Forbes, PhD
Slide 14: Mechanism of action and viral vector configuration
CONFIDENTIAL 14 • In normal salivary glands water flows through acinar cells into the duct. Duct cells are impermeable to water. • Impermeable duct cells generate an osmotic gradient (lumen > interstitium) • Acinar cells are particularly vulnerable to damage with Ionizing radiation used to treat hea
Slide 15: McMaster Global Rate of Change assessment
CONFIDENTIAL 15 • A validated Patient Reported Outcome measure wherein the patient rates the severity of their dry mouth • Patients are asked, “Overall, has there been any change in your Dry Mouth since you received the study treatment” • Patients may reply, “Better”, “Worse”, or “About the Same” •
Slide 16: McMaster results at Day 90 and beyond
CONFIDENTIAL 16 • 6 of the 7 participants to date reaching 90-day assessments reported their symptoms of dry mouth as better following treatment • All 6 of these participants rated changes in xerostomia scores that were important or very important (a score of 2 or more) • 3 participants rated the ch
Slide 17: Xerostomia Questionnaire results
CONFIDENTIAL 17 • A Patient Reported Outcome measure consisting of 8 symptom-specific questions wherein the patient rates each symptom from 0 (not present) to 10 (worst possible) • The responses are summed (0-80), providing an overall measure of disease burden • This is refined from the Xerostomia I
Slide 18: Safety and efficacy summary
CONFIDENTIAL 18 Safety • AAV-hAQP1 treatment appears safe and well tolerated at each dose tested • No DLT or SAEs Efficacy • Improvements in xerostomia symptoms and disease burden reported in two different PRO tools validated for xerostomia • McMaster – which has been the basis of approval of other
Slide 19: Clinical Perspective
Clinical Perspective Michael Brennan, DDS, MHS, FDS RCSEd
Slide 20: Treatment administration and patient qualification
CONFIDENTIAL 20 • Quick outpatient procedure • Non-invasive: allows local administration and avoids systemic exposure • Parotid gland is isolated and encapsulated, somewhat immune protected • Small volume of vector required Qualifying a patient for treatment with AAV-hAQP1 utilizes routine practices
Slide 21: Summary of AAV-hAQP1 opportunity
CONFIDENTIAL 21 • Serious debilitating condition with severe impact on health and daily living • Intractable disease with no treatment options • Large unmet need with >170,000 grade 2 and grade 3 xerostomia patients in the U.S. and 5,000-10,000 new patients annually • Patients are already in healthc
Slide 22: Audience Q&A
C) MEIRAGTx Audience Q&A
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