MeiraGTx Reacquires Bota-vec, AAV2-hAQP1 Gets FDA BTD; Raises $100M
MeiraGTx Holdings plc announced strong operational and financial results for Q1 2026, highlighted by the reacquisition of full rights to botaretigene sparoparvovec (bota-vec) for X-linked retinitis pigmentosa from Johnson & Johnson. The company also received FDA Breakthrough Therapy Designation for AAV2-hAQP1 for radiation-induced xerostomia, supported by positive three-year Phase 1 data. MeiraGTx strengthened its balance sheet with $100 million in gross proceeds from an equity offering, extending its cash runway into the second half of 2028. Despite a net loss of $46.3 million for the quarter, these strategic moves position the company for potential regulatory approvals and launches of two wholly-owned therapies within the next two years.
Key Highlights
- Reacquired full rights to bota-vec for X-linked retinitis pigmentosa from Johnson & Johnson for $25M upfront.
- AAV2-hAQP1 received FDA Breakthrough Therapy Designation for radiation-induced xerostomia in March 2026.
- Reported positive three-year data from Phase 1 AQUAx study of AAV2-hAQP1, showing sustained improvements.
- Strengthened balance sheet with $100M gross proceeds from an underwritten offering of ordinary shares.
- Q1 2026 net loss attributable to ordinary shareholders was $46.3M, or $0.57 per share.
- Cash and cash equivalents were $71.5M as of March 31, 2026.
- Appointed Penny Fleck as Chief Development Officer and Stuart Naylor as Chief Scientific Officer, Ophthalmology.
- AAV-GAD for Parkinson's disease received FDA RMAT designation in 2025, with Phase 3 study initiating soon.