MeiraGTx : Corporate Deck May 2025 Website
MGTX
Published on 05/13/2025 at 08:07
Diverse Program Pipeline
Broad pipeline across neuro, salivary gland, and ophthalmology
4 pivotal stage programs: Parkinson's disease, radiation-induced xerostomia, AIPL1 retinal dystrophy, X-linked retinitis pigmentosa1
Diverse preclinical pipeline: ALS, obesity, Stargardt's, wet AMD
Broad Ophthalmology pipeline
End-to-End GMP Manufacturing
In-house manufacturing and
industry-leading process
2 GMP viral vector manufacturing facilities, each with multiple production suites - flexible and scalable
In-house GMP plasmid production
Commercially-licensed QC facility for release and stability
In-house Fill C Finish, warehouse
and supply chain
Next-Generation Vector Optimization
Improved potency s safety,
lower dose and lower COGS
Novel Promoters/Enhancers
Proprietary intravitreal capsids
Vector sequence optimization -durability, immune protection, manufacturability
Translational models - human retinal organoids, patient samples, iPSCs
Transformative Riboswitch Technology
Proprietary platform for titratable control of gene expression
Gene expression is activated via oral small molecule inducers in a dose responsive manner
Precise dose response with unprecedented dynamic range (>5000-fold)
Broad applications: cell therapy, gene editing, genetic medicine
Expected near-term global BLA
filings based on current studies:
AIPL1 congenital blindness (2025)
RPGR X-linked RP (2025)
Radiation induced xerostomia (2026)
Parkinson's disease (2027)
Avoid CDMO bottlenecks C quality
failures
Saves up to 3 years in development timeline, significantly increasing ROI
Significantly reduced Cost of Goods (COGS)
Improvements in potency > 3 logs
Enables lower AAV dose with
improved safety and efficacy
Vast reduction in COGS -
improving patient access
Highly modular - any transgene can be controlled by riboswitch, delivered by any vector
New approach to metabolic disease -combinations of vectorized metabolic peptides (e.g. GLP1, GIP, leptin, myokines)
1 Remaining interests in program sold to Janssen in December 2023; MeiraGTx to receive up to an aggregate of $350.0 million upon achievement of milestones and will manufacture and supply commercial product for Janssen.
MeiraGTx: late-stage clinical pipeline and comprehensive end-to-end capabilities & technologies in genetic medicine
01 02 03 04
Phase 3 ready
Potential BLA filing in 2027
Large patient population inadequately controlled by dopamine therapy
Pivotal Phase 2
Potential BLA filing in 2026
Large patient population with no effective therapies available
'Pipeline in a product'
Developed under 'specials' license
Potential UK approval in 2025
Potential FDA
expedited approval
Transformative effect (blind to seeing)
Completed Phase 3
BLA filing in 2025
Acquired by Janssen; MeiraGTx manufactures commercial product
Four pivotal stage programs in prevalent and rare indications
Best in class end-to end internal capabilities: Process development facility, Plasmid production facility, two flexible scalable Vector Production facilities and internal QC facility with commercial license for release and stability
Commercial process at IND: Saves 2-3 years
for an AAV clinical development timeline from IND to commercial, allows faster move to pivotal with expedited time to market. Increases ROI
on each Product
Global Regulatory buy-in and extensive experience pre-IND through BLA/commercial
Commercial grade manufacturing with scalable and flexible capacity for full internal pipeline and commercial partners
Unique end-to-end offering of innovative technology, commercial quality and capacity
Unique end-to-end in-house manufacturing infrastructure and production process fit for clinical & commercial supply
Disclaimer
MeiraGTx Holdings plc published this content on May 13, 2025, and is solely responsible for the information contained herein. Distributed via Public Technologies (PUBT), unedited and unaltered, on May 13, 2025 at 12:06 UTC.