MeiraGTx Holdings plc MGTX
MeiraGTx Holdings plc (Nasdaq: MGTX) is an ophthalmology drug developer. Its lead program, Botaretigene sparoparvovec (bota-vec), is in Phase 3 for X-linked retinitis pigmentosa (RPGR). Its next catalyst on record is AAV2-hAQP1: Pivotal Phase 2 AQUAx2 12-month data readout (Q2 2027).
Upcoming catalysts
No catalyst is dated to the next 30 days or to an FDA action date.
Next further out: AAV2-hAQP1 — Pivotal Phase 2 AQUAx2 12-month data readout (Q2 2027). 5 catalysts on record beyond the next 30 days; subscribers see each with its filing sentence.
Pipeline
| Program | Indication | Modality | Stage |
|---|---|---|---|
| Bota-vec | X-linked retinitis pigmentosa (Phase 3 LUMEOS completed; rights reacquired from Janssen May 2026) | gene therapy | Phase 3 |
| AAV-AIPL1 (medu-vec) | Leber congenital amaurosis 4 (out-licensed to Lilly) | gene therapy | Phase 3 |
| AAV2-hAQP1 | Radiation-induced xerostomia (pivotal Phase 2 AQUAx2, enrollment complete) | gene therapy | Phase 2 |
| AAV-GAD | Parkinson's disease (RMAT; Phase 3 to start) | gene therapy | Phase 2 |
| AAV-BDNF | Genetic obesity disorders (in Reogen JV) | gene therapy | Pre-clinical |
| Ribo-Leptin (riboswitch) | Metabolic disease | gene therapy | Pre-clinical |
| Riboswitch neuropathic pain program | Long-term intractable pain | gene therapy | Pre-clinical |
Recent results and decisions
- Aug 13, 2026completed enrollment Pivotal Phase 2 AQUAx2 study of AAV2-hAQP1 completed enrollment in Q2 2026.
The pivotal Phase 2 AQUAx2 study (NCT05926765), a randomized, double-blind, placebo-controlled study at 30 sites in the U.S., Canada, and the U.K. completed enrollment in the second quarter of 2026
Filing - May 14, 2026positive Phase 1 AQUAx (n=24) 3-year data showed sustained, clinically meaningful improvement in patient-reported outcomes and salivary flow from 12 to 36 months, well tolerated.
In April 2026, MeiraGTx reported positive three-year data from its Phase 1 AQUAx clinical trial (n=24) evaluating AAV2-hAQP1 for the treatment of moderate to severe grade 2/3 radiation-induced xerostomia.
Filing - Apr 16, 2026missed Phase 3 LUMEOS primary endpoint (VMA maze) not statistically significant (treated 2.4x more likely to respond) though secondary endpoints significant, e.g. LLVA p=0.003; data previously presented in 2025.
The novel primary endpoint to assess the effect of bilaterial treatment with bota-vec on functional vision as measured by a Visual Mobility Assessment (VMA), or maze, did not meet statistical significance.
Filing
1 more on record.
For subscribers
- Cash runway against each catalyst, with the company's own runway statement
- Track record: every Phase 3 readout, FDA decision and guidance slip since 2014
- Expected move for each dated catalyst, from comparable past events
- Phase 2 evidence behind each Phase 3 readout
- Companies on the same target or class
- Full catalyst list with filing sentences, deals, registered trials, Excel export
Other phase 3 ophthalmology companies
The free Monday brief
Last week's readouts and FDA decisions and the dated catalysts in the week ahead, each with its filing sentence.