botaretigene sparoparvovec — NDA/BLA submission
2026
Anticipate submission of global regulatory filings for approval of bota-vec in 2026 and potential BLA filing for AAV2-hAQP1 in mid-2027Source filing
MGTX biotech catalysts & pipeline
MEIRAGTX HOLDINGS PLC · Data as of 2026-09-08
Upcoming catalysts
native human leptin (Ribo-Leptin) — IND submission
2026
The Company is in iterative discussion with the FDA to open a Ribo-Leptin IND later this year.Source filing
botaretigene sparoparvovec — Regulatory decision
2027
An additional $50 million will be available at the Company's option tied to regulatory approval of bota-vec in 2027Source filing
AAV2-hAQP1 — Topline data
Q2 2027
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 and remains on track for a 12-month pivotal data readout in the second quarter of 2027, which, if positive, would support a BLA filing and potential approval targeted for the end of 2027, with U.S. launch in 2028.Source filing
AAV2-hAQP1 — NDA/BLA submission
mid-2027
Anticipate submission of global regulatory filings for approval of bota-vec in 2026 and potential BLA filing for AAV2-hAQP1 in mid-2027Source filing
AAV2-hAQP1 — NDA/BLA submission
Late 2027
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 and remains on track for a 12-month pivotal data readout in the second quarter of 2027, which, if positive, would support a BLA filing and potential approval targeted for the end of 2027, with U.S. launch in 2028.Source filing
AAV2-hAQP1 — Regulatory decision
2028
An additional $50 million will be available at the Company's option tied to regulatory approval of AAV2-hAQP1 in 2028Source filing