MeiraGTx (MGTX) Corporate presentation summary

Pipeline and clinical programs

• Four pivotal programs: radiation-induced xerostomia, X-linked retinitis pigmentosa, Parkinson's disease, and AIPL1 retinal dystrophy, all at late-stage or near BLA/MAA filing.

• Broad preclinical pipeline includes ALS, neuropathic pain, obesity, diabetes, and large ophthalmology indications.

• AAV-AQP1 for xerostomia shows transformative, durable improvements in both patient-reported and objective endpoints, with Phase 2 pivotal trial enrolling and BLA filing expected in 2027.

• Bota-vec for X-linked retinitis pigmentosa completed Phase 3 with robust efficacy and safety, supporting global regulatory filings.

• AAV-GAD for Parkinson's disease demonstrated significant, durable motor improvements and evidence of disease modification in multiple controlled trials; Phase 3 ready.

Technology and manufacturing

• Proprietary end-to-end GMP manufacturing with two cGMP vector facilities, in-house plasmid production, QC, and fill/finish, supporting clinical and commercial supply.

• Commercial licenses in Ireland and UK for viral vector production and QC.

• Advanced vector engineering platforms: >250k promoter library, AI-driven optimization, proprietary capsids, and riboswitch gene regulation for precise in vivo protein control.

• Riboswitch platform enables oral, titratable control of therapeutic protein production, validated in animal models for multiple modalities.

Market opportunity and commercial strategy

• Persistent radiation-induced xerostomia is a severe, lifelong, untreatable condition affecting 165k in the US and 435k globally, with a projected peak global revenue of ~$3.7B.

• Bota-vec targets >20,000 patients in US/EU, with projected peak sales of ~$1.7B and strong KOL and patient advocacy support.

• AAV-AQP1 therapy is minimally invasive, outpatient, and could become standard of care for xerostomia, with high clinician preference and payer coverage estimates (~90%).

• Strategic hospital targeting can reach >60% of US head and neck cancer population with 15 major metro sites.