Reports Q1 revenue $1.9M vs. $697,000 last year. “The first quarter of 2025 was a pivotal quarter for MeiraGTx (MGTX), as we engaged in positive interactions with the FDA around each of our late-stage clinical programs and move forward with potentially BLA-supporting Phase 2 and Phase 3 clinical studies and BLA filings in each of our late-stage clinical programs,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. “We are working with global regulators to file for expedited approval under exceptional circumstances of AAV-AIPL1 for children with LCA4 based on the unprecedented data from the treatment of the 11 young children under our Specials License in the U.K. We have now engaged in positive discussions with the FDA aligning on the requirements to support a similar pathway in the US. We have also gained alignment with the FDA on the requirements for the ongoing Phase 2 AQUAx2 study for AAV-hAQP1 for the treatment of RIX to support a potential BLA. In both cases, our alignment with the FDA for CMC requirements is a critical aspect of the discussions. We are very happy to have just received RMAT designation for our Parkinson’s disease program based on the data from our three positive clinical studies, including two double-blind, sham-controlled studies. In addition to the rapid pace of regulatory discussions and clinical progress, we meaningfully strengthened our balance sheet with non-dilutive funding through the announcement of our strategic collaboration with Hologen AI. Our agreement with Hologen includes a $200 million upfront payment to MeiraGTx, as well as the formation of a new joint venture, Hologen Neuro AI Ltd, which will be funded by an additional $230 million in committed capital from Hologen.The Hologen Neuro AI joint venture is the first neuro-AI clinical-stage drug development company to transform the discovery and development of therapies targeting CNS circuitry in neurodegenerative and neuropsychiatric disorders. The initial focus of the joint venture is to accelerate the AAV-GAD program through the use of Hologen’s technology applied to the data from our double-blind, sham-controlled Phase 2 studies. Our studies have shown clinically significant and statistically significant benefit using the standard clinical endpoint of Unified Parkinson’s Disease Rating Scale Part 3, and now we have demonstrated a disease modifying change in the circuitry of the brain of the patients treated with AAV-GAD as well as potentially protective changes in the substantia nigra and regions of the brain involved in cognition and mood. This is the first time sham-controlled gene or cell therapy Phase 2 studies have shown significant benefit in UPDRS and now, to our knowledge, the only demonstration of disease modification in a sham or placebo-controlled study in Parkinson’s disease.” “We are very encouraged by the breadth of positive, clear, timely, science-based discussions and interactions we have experienced with the FDA and MHRA, and we plan to work closely with these regulatory agencies to move each of our late-stage clinical programs forward to approval and ultimately deliver these therapies to those patients currently without effective treatments,” stated Dr. Forbes.
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