MeiraGTx Completes Bota-vec MAA Ahead of December EMA Filing

The Tip Desk · November 12, 2026 · 4 min read

Cash and cash equivalents and restricted cash stood at $186.8 million as of September 30, 2026, as the company prepared a full FDA biologics license application for bota-vec in the first quarter of 2027.

MeiraGTx Holdings plc (MGTX), a vertically integrated, clinical-stage genetic medicines company, said the marketing authorisation application dossier for botaretigene sparoparvovec (bota-vec) in X-linked retinitis pigmentosa is complete and in publishing, with submission to the European Medicines Agency intended for December 2026, subject to EMA confirmation of the date.

The company anticipates a full biologics license application to the U.S. Food and Drug Administration in the first quarter of 2027. President and chief executive Alexandria Forbes said the MAA dossier is complete and awaiting submission and that the BLA is expected to follow early next year. In parallel, she said, the company has been preparing market access and commercial teams ahead of an anticipated launch of bota-vec in late 2027.

MeiraGTx holds all rights and interests in bota-vec following its May 2026 acquisition from Johnson & Johnson. The Phase 3 LUMEOS study was a global, randomized study (n=95) in which all patients were treated bilaterally. The company said data highlight the potential of bota-vec to improve each of the domains of vision, with a safety profile as expected and manageable, no new safety signals, and an improved inflammatory profile relative to the Phase 1/2 study. As commercial manufacturer, MeiraGTx completed process performance qualification, with Module 3 of the MAA complete. It holds a commercial license for its London manufacturing facility and a commercial license for its QC facility in Shannon, Ireland. Bota-vec has Fast Track and orphan drug designation from the FDA, and in the EU has received PRIME, ATMP classification, and orphan drug designation.

For the three months ended September 30, 2026, net loss attributable to ordinary shareholders was $51.3 million, or $0.53 a share, compared with $50.5 million, or $0.62 a share, a year earlier.

Total revenue was $4.8 million. Service revenue was $1.6 million, versus nil a year earlier, from contract manufacturing and support services for Eli Lilly under the AAV-AIPL1 collaboration. Service revenue – related party was $3.2 million, versus $0.4 million, primarily from development and transition services to Reogen versus PPQ services to J&J in 2025. Cost of service revenue was $0.6 million and cost of service revenue – related party was $2.8 million.

Research and development expenses were $39.3 million, versus $32.5 million. The company said the $6.8 million increase was primarily attributable to increased clinical trial activities and manufacturing of clinical trial batch material related to AAV2-hAQP1, higher bota-vec expenses after the Janssen reacquisition, and increased AAV-GAD costs, primarily from allocation of manufacturing batch costs not recognized in cost of service revenue – related party. Manufacturing costs also increased on scheduled maintenance, an increase the company said was fully offset by lower expenses on other preclinical ocular programs. General and administrative expenses were $12.0 million, versus $13.6 million.

Financial related costs, net reflected a $1.6 million gain, versus a $3.1 million expense, a $4.7 million favorable swing the company attributed primarily to a gain from the change in fair value of the royalty notes and lower interest expense after terminating the Perceptive notes purchase agreement in the second quarter of 2026. Interest income was $1.2 million, versus $0.2 million. Foreign currency loss was de minimis, versus a $1.6 million loss. Loss on equity method investee was $3.9 million, versus nil, reflecting the company’s share of Reogen’s research and development expenses. Income tax expense was $0.6 million.

Cash, cash equivalents and restricted cash were $186.8 million as of September 30, 2026, compared with $17.1 million a year earlier. Cash and cash equivalents were approximately $184.7 million. With an additional $95.0 million upfront payment due from Hologen and associated reimbursements, the company said it believes it will have sufficient capital to fund operating expenses and capital expenditure into the second half of 2028. That estimate excludes $135.0 million of potential near-term cash consideration from Lilly upon achievement of certain development and regulatory approval milestones, or any subsequent tranches under the Oberland royalty note purchase agreement.

The pivotal Phase 2 AQUAx2 study of AAV2-hAQP1 for radiation-induced xerostomia completed enrollment in the second quarter of 2026 and remains on track for a 12-month pivotal data readout in mid-2027, which, if positive, could support a BLA submission by the end of 2027, with potential U.S. approval in 2028. The FDA granted Breakthrough Therapy Designation to AAV2-hAQP1 in March 2026. The joint venture with Hologen, Reogen, intends to initiate a Phase 3 study of AAV-GAD in Parkinson’s disease in the first quarter of 2027. A single ascending dose/multiple ascending dose study of the company’s lead riboswitch small molecule is expected to initiate in December 2026.