MeiraGTx Achieves Regulatory Milestones, Secures $200M Funding, and Extends Financial Runway

MeiraGTx achieved significant regulatory milestones in Q2 2025, including FDA alignment on its Phase 2 AQUAx2 study and RMAT designation for AAV-GAD in Parkinson's treatment. The company secured $200M in cash and potential additional funding of up to $230M through a strategic collaboration with Hologen. MeiraGTx reported $32.2M in cash as of June 30, 2025, with expected proceeds to fund operations into 2027.

MeiraGTx Holdings plc (Nasdaq: MGTX) reported significant regulatory milestones and secured substantial funding during the second quarter of 2025. The company announced alignment with the U.S. Food and Drug Administration (FDA) on its Phase 2 AQUAx2 study for radiation-induced xerostomia (RIX) and received Regenerative Medicine Advanced Therapy (RMAT) designation for AAV-GAD in the treatment of Parkinson’s disease. Additionally, MeiraGTx secured $200 million in cash and potential additional funding of up to $230 million through a strategic collaboration with Hologen.

The company reported $32.2 million in cash as of June 30, 2025, with expected proceeds to fund operations into 2027. Dr. Alexandria Forbes, president and chief executive officer of MeiraGTx, stated, “Our late-stage clinical programs are all advancing as planned, with our pivotal Phase 2 study of AAV-AQP1 in RIX targeting enrollment by the end of the year with the potential for BLA enabling data read out at the end of 2026” [1].

MeiraGTx also announced that it is preparing submissions for Marketing Authorization Approval (MAA) under exceptional circumstances with the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of LCA4, and a Biologics License Application (BLA) in the US with the FDA via a similar pathway to approval in the fourth quarter of 2025. The company is also engaged with regulators to initiate first-in-human studies using its transformative riboswitch platform by the end of 2025.

The strategic collaboration with Hologen includes the formation of a joint venture, Hologen Neuro AI Ltd, with committed funding of up to $230 million. MeiraGTx will hold a 30% ownership in the joint venture and lead all clinical development and manufacturing. The joint venture will use Hologen’s proprietary multi-modal generative foundation models (LMMs) to further accelerate the optimization of MeiraGTx’s proprietary manufacturing capabilities.

In addition to the above achievements, MeiraGTx has received orphan drug designation for AAV-AIPL1 in the US and the European Union, and Rare Pediatric Disease Designation (RPDD) for the treatment of LCA4 retinal dystrophy. The company is preparing submissions for MAA in the UK and BLA in the US for AAV-AIPL1.

MeiraGTx’s significant regulatory milestones and strategic funding position the company well for future growth and potential market entry in the fourth quarter of 2025.

References:
[1] https://www.globenewswire.com/news-release/2025/08/14/3133409/0/en/MeiraGTx-Reports-Second-Quarter-2025-Financial-and-Operational-Results.html