MeiraGTx Reports First Quarter 2025 Financial and Operational Results

About this update from Meiragtx Holdings Plc

- Announced strategic collaboration with Hologen AI, including a $200 million cash upfront payment to MeiraGTx and the formation of a joint venture, Hologen Neuro AI Ltd , with a further $230 million in capital committed to initially focus on expediting Phase 3 clinical development of AAV-GAD for Parkinson’s disease - U.S. Food and Drug Administration (FDA) Granted Regenerative Medicine Advanced Therapy (RMAT) designation for AAV-GAD for the treatment of Parkinson’s disease - Gained alignment with FDA on the ongoing Phase 2 AQUAx2 randomized double-blind, placebo-controlled pivotal study in Grade 2/3 radiation-induced xerostomia (RIX) to support a potential BLA filing - Efficacy data of rAAV8.hRKp.AIPL1 for the treatment of AIPL1-related retinal dystrophy, or LCA4, published in The Lancet , demonstrating meaningful responses in 11 out of 11 LCA4 children treated. Filing for Marketing Authorization Approval (MAA) under exceptional circumstances with the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) and in discussions with the FDA around a potentially similar pathway to approval in the US LONDON and NEW YORK, May 13, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical-stage genetic medicines company, today announced financial and operational results for the first quarter ended March 31, 2025 , and provided a corporate update. “The first quarter of 2025 was a pivotal quarter for MeiraGTx , 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.” Dr. Forbes continued, “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.” Dr. Forbes added, “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 (UPDRS) 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 . Recent Development Highlights Strategic Collaboration with Hologen AI: MeiraGTx to receive $200 million in upfront cash consideration at closing. MeiraGTx and Hologen will form a joint venture, Hologen Neuro AI Ltd , with additional committed funding from Hologen of up to $230 million into the joint venture to finance the development of the AAV-GAD program in Parkinson’s disease to commercialization, as well as other locally-delivered therapies to the CNS. The joint venture, Hologen Neuro AI Ltd , will use Hologen’s proprietary multi-modal generative foundation models (LMMs). MeiraGTx will hold a 30% ownership in the joint venture and lead all clinical development and manufacturing. Hologen Neuro AI Ltd will enter into both clinical and commercial manufacturing supply agreements with MeiraGTx for exclusive manufacturing of AAV-GAD and other locally-delivered genetic medicines targeting the CNS. Hologen will own a minority stake in MeiraGTx’s manufacturing subsidiary and will contribute a portion of the annual funding and deploy Hologen’s world leading generative AI capabilities to further accelerate the optimization of MeiraGTx’s proprietary manufacturing capabilities. AAV-GAD for the Treatment of Parkinson’s Disease : On May 8 th, 2025, the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to AAV-GAD for the treatment of Parkinson’s disease not adequately controlled with anti-Parkinsonian medications. This RMAT was awarded following the presentation to the FDA of positive data from 3 clinical studies demonstrating the benefit of AAV-GAD when administered in a one-time stereotactic infusion to the subthalamic nucleus in the brain. A Phase 1 dose escalating clinical study (n=12) was conducted, followed by a double-blind, sham-controlled Phase 2 study (n=45) and a second randomized, double-blind, sham-controlled dose ranging clinical bridging study (n=14). Data from the double-blind, sham-controlled Phase 2 studies show significant clinically meaningful benefit on the standard motor endpoint in Parkinson’s disease, UPDRS Part 3, as well as other validated measures of Parkinson’s symptoms. Through the use of Hologen’s AI technology to analyze the data from double-blind sham controlled studies, disease modifying changes in the circuitry of the brain of the Parkinson’s patients receiving AAV-GAD therapy have been demonstrated. In addition, potentially protective changes in the substantia nigra and regions of the brain involving cognition and mood have been shown following AAV-GAD treatment. The Company plans to initiate a Phase 3 study of AAV-GAD in the second half of 2025 and will continue to work with the FDA to expedite the development of the program supported by MeiraGTx’s end-to-end in-house manufacturing capabilities. RMAT Designation: The requirements for receiving an RMAT designation include that the drug candidate is an advanced regenerative medicine, in this case a gene therapy; that the therapy is targeting a serious condition, in this case, Parkinson’s disease; and that the applicant has presented clinical evidence demonstrating that the drug candidate has the potential to address an unmet need in the serious condition. The RMAT requirement for clinical data supporting a benefit in an unmet need is a high hurdle, with less than half of all RMAT designation applications granted. RMAT designation includes the benefits of Fast Track and Breakthrough Therapy designations with rolling review and potential Priority Review of a product’s BLA. RMAT designation also allows for increased interaction with the FDA and immediate multidisciplinary comprehensive discussions of the ongoing product development program, clinical trials and plans for expediting the manufacturing development strategy, both clinical and CMC. AAV2-hAQP1 for the Treatment of Xerostomia: In December 2024 , MeiraGTx was granted RMAT designation by the FDA for AAV2-hAQP1 for the treatment of Grade 2/3 RIX. Following FDA interactions over the past three months, the Company has aligned with the agency on both the clinical and CMC requirements for the ongoing Phase 2 AQUAx2 randomized, double-blind, placebo-controlled study to support a potential BLA. The Phase 2 AQUAx2 ( NCT05926765 ) randomized, double-blind, placebo-controlled study continues to enroll and dose participants at multiple sites in the US, Canada and the U.K. The low dose cohorts have completed enrollment. Screening and enrollment of the remaining high dose cohorts is ongoing with the target for completion of enrollment in the fourth quarter of 2025, and the potential for a BLA filing supported by this study at the end of 2026. AAV-AIPL1 for LCA4 Caused by Mutations in the AIPL1 Gene: In February 2025 , the Company announced that data demonstrating the efficacy of rAAV8. hRKp.AIPL1 for the treatment of LCA4 were published in The Lancet in a paper titled, “ Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study ”; the Company held a webcast to review the data, and a replay of the webcast can be accessed here . As disclosed in the paper, 4 out of 4 young children with the AIPL1-associated retinal dystrophy, LCA4, benefited substantially from unilateral subretinal administration of rAAV8 .hRKp.AIPL1 with improved visual acuity, functional vision, and protection against progressive retinal degeneration. Following the strong safety and substantial efficacy demonstrated in this first cohort of 4 children treated unilaterally, a further 7 children were treated bilaterally, and all showed substantial benefit from treatment with rAAV8. hRKp.AIPL1. Meaningful responses have been observed in 11 out of 11 LCA4 children treated to date with rAAV8. hRKp.AIPL1. Following recent meetings with the MHRA, the Company is in the process of preparing the submission of Marketing Authorization Application (MAA) under exceptional circumstances for rAAV8. hRKp.AIPL1 based on the results from the 11 treated children, with no further clinical data required. The Company has had positive engagement with the FDA over the past few weeks and aligned with the agency on the requirements in the areas of clinical, non-clinical and CMC to move forward towards a potentially expedited approval in the US. AAV-AIPL1 for the treatment of LCA4 has orphan drug designation in the US and orphan designation in the European Union , and the Offices of Orphan Products Development and Pediatric Therapeutics of the FDA have granted Rare Pediatric Disease Designation (RPDD) to AAV8-RK-AIPL1 for the treatment of LCA4 retinal dystrophy. Botaretigene Sparoparvovec for the Treatment of X-linked Retinitis Pigmentosa (XLRP): Data from the Phase 3 LUMEOS trial of botaretigene sparoparvovec (bota-vec) for the treatment of X-linked retinitis pigmentosa was presented by Dr. Michael Clark , the primary clinical lead on the study from Johnson & Johnson, at the Foundation Fighting Blindness 2025 Retinal Therapeutics Innovation Summit on May 2 nd, 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, was not met but was directionally supportive. However, all but one of the secondary endpoints, including measures from all three visual domains – Functional Vision, Retinal Function and Visual Function – showed benefit with 95% CI intervals not including 0. These included: Functional Vision: PRO: LLQ Extreme lighting domain score, a patient reported measure; Retinal Function: three measures of retinal sensitivity by static perimetry, 5-point 7-decibel responders in both the Central 30 degrees and also in the full field, as well as mean retinal sensitivity in the central 10 degrees; Visual Function: Low luminance visual acuity (LLVA, ETDRS). In addition, 40% (22/55) of treated patients showed improvement in ≥2 endpoints each in different domains of vision compared to 0% in the control group. Safety profile of bota-vec was as expected and manageable with no new safety signals. >90% of treatment related inflammation adverse events were mild,

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