MeiraGTx — Third Quarter 2026 Earnings Summary

Financial Performance:

• Total revenue for the three months ended September 30, 2026, was $4.8 million, compared to $0.4 million for the same period in 2025.
• Service revenue was $1.6 million for the quarter ended September 30, 2026, compared to nil in 2025, driven by contract manufacturing services for Lilly.
• Service revenue – related party was $3.2 million for the quarter ended September 30, 2026, compared to $0.4 million in 2025, primarily due to development and transition services provided to Reogen.
• Cost of service revenue was $0.6 million for the quarter ended September 30, 2026, compared to nil in 2025.
• Cost of service revenue – related party was $2.8 million for the quarter ended September 30, 2026, compared to $0.3 million in 2025.
• General and administrative expenses were $12.0 million for the quarter ended September 30, 2026, a decrease of $1.6 million from $13.6 million in 2025.
• Research and development expenses were $39.3 million for the quarter ended September 30, 2026, an increase of $6.8 million from $32.5 million in 2025.
• Net loss attributable to ordinary shareholders was $51.3 million for the quarter ended September 30, 2026, compared to $50.5 million in 2025.
• Net loss per ordinary share was $0.53 (basic and diluted) for the quarter ended September 30, 2026, compared to $0.62 in 2025.
• Cash, cash equivalents, and restricted cash were $186.8 million as of September 30, 2026, compared to $17.1 million as of September 30, 2025.
• Deferred revenue, current was $0.5 million as of September 30, 2026, compared to nil in 2025.
• Deferred revenue – related party, current was $4.7 million as of September 30, 2026, compared to $1.8 million in 2025.
• Note payable, net, current was nil as of September 30, 2026, compared to $24.6 million in 2025.
• Note payable, net (non-current) was $121.9 million as of September 30, 2026, compared to $49.7 million in 2025.
• Total liabilities were $196.5 million as of September 30, 2026, compared to $250.2 million as of December 31, 2025.
• Shareholders' equity was $215.2 million as of September 30, 2026, compared to a deficit of $5.8 million as of December 31, 2025.

Guidance and Future Outlook:

• The Company expects to have sufficient capital to fund operating expenses and capital expenditure requirements into the second half of 2028.
• The MAA dossier for bota-vec is complete and expected to be submitted to the EMA in December 2026.
• The full BLA for bota-vec is expected to be submitted to the FDA in the first quarter of 2027.
• The pivotal Phase 2 AQUAx2 study for AAV2-hAQP1 is on track for a 12-month data readout in mid-2027, with a potential BLA submission by the end of 2027 and U.S. approval in 2028 if positive.
• The JV with Hologen (Reogen) intends to initiate a Phase 3 study of AAV-GAD in the first quarter of 2027.
• A single ascending dose/multiple ascending dose (SAD/MAD) study of the lead small molecule for the riboswitch platform is expected to initiate in December 2026.
• First-in-human combination studies for the prioritized riboswitch programs (Ribo-Leptin and neuropathic pain) are planned for 2027.
• Commercial launch of bota-vec is anticipated in late 2027.

Business Segments and Product Lines:

• bota-vec (XLRP): MeiraGTx holds all rights following a May 2026 acquisition from Johnson & Johnson. The program has received Fast Track and orphan drug designation from the FDA, and Priority Medicines (PRIME), ATMP classification, and orphan drug designation in the EU. Process performance qualification (PPQ) was completed, and commercial licenses are held for facilities in London and Shannon, Ireland.
• AAV2-hAQP1 (Radiation-induced xerostomia): Received Breakthrough Therapy Designation from the FDA in March 2026. The pivotal Phase 2 AQUAx2 study completed enrollment in Q2 2026. A commercial in-vitro biological activity potency assay was presented at the ESGCT 2026 Annual Congress in October 2026.
• AAV-GAD (Parkinson's Disease): Received Regenerative Medicine Advanced Therapy (RMAT) designation in 2025 based on data from Phase 2 and Phase 1/2 studies.
• AAV-AIPL1 (LCA4): Granted Eli Lilly worldwide exclusive rights; MeiraGTx received a $75 million upfront payment and is eligible for over $400 million in milestone payments plus tiered royalties.
• Riboswitch Platform: Prioritized for Ribo-Leptin (metabolic disease) and a neuropathic pain program. The platform allows precise dose control of biologic therapeutics via oral small molecules.
• AAV-BDNF: An oral presentation on this program for MC4R/BDNF genetic obesity is scheduled for ObesityWeek in November 2026.
• Other Programs: Advancing dry AMD and Geographic Atrophy programs using proprietary intravitreal capsids and cell-specific promoters.

Market and Competitive Landscape:

• XLRP is a rare inherited retinal disease with no currently approved treatments, affecting over 20,000 patients in the U.S. and EU.
• The riboswitch platform is described as gene-agnostic and applicable to various vectors including lentivirus, AAV, CRISPR, and LNPs.
• MeiraGTx has built comprehensive in-house manufacturing capabilities, including two GMP viral vector production facilities and a GMP QC facility with commercial licensure.

Risks and Challenges:

• The press release includes a standard "Forward Looking Statement" warning that actual results may differ materially due to risks such as incurrence of significant losses, inability to achieve profitability, failure to obtain regulatory approval, clinical trial failures, manufacturing issues, and competition.
• Specific risks include dependence on third parties, intellectual property risks, changes in tax policy, and litigation risks.

Management Commentary and Tone:

• CEO Alexandria Forbes, Ph.D., expressed pleasure at completing work toward global regulatory filings for bota-vec and noted preparations for market access and commercial teams ahead of the late 2027 launch.
• Management highlighted active preparation for the mid-2027 data readout for AAV2-hAQP1 and the advancement of several programs toward IND submissions.
• Dr. Forbes emphasized the use of internal and partnered AI technology to develop proprietary intravitreal capsids for dry AMD programs.

Other Key Points:

• Acquisition: MeiraGTx acquired bota-vec from Johnson & Johnson in May 2026.
• Collaboration: MeiraGTx granted Eli Lilly exclusive rights to AAV-AIPL1 and related technologies, receiving a $75 million upfront payment.
• Joint Venture: MeiraGTx has a JV with Hologen Limited (Reogen) for AAV-GAD, with a $95.0 million upfront payment due from Hologen.
• Royalty Note: MeiraGTx terminated a notes purchase agreement with Perceptive Credit Holdings III, LP in Q2 2026 and issued new royalty notes to Oberland Capital Management LLC.
• Leadership Appointments: John Knighton was appointed Executive Vice President of Global Manufacturing and Supply Chain in July 2026; Alan M. Sebulsky was appointed to the Board of Directors in September 2026.
• Capital Position: The Company believes it has sufficient capital to fund operations into the second half of 2028, excluding potential near-term cash consideration from Lilly milestones and subsequent tranches from the Oberland Capital royalty note.