MeiraGTx(MGTX) - 2025 Q2 - Quarterly Results

Executive Summary & Corporate Update MeiraGTx achieved significant regulatory milestones in Q2 2025, including FDA alignment for AQUAx2, RMAT for AAV-GAD, and planned MAA/BLA filings for LCA4, while the CEO highlighted productive regulatory interactions and advancements in late-stage programs Q2 2025 Highlights MeiraGTx reported significant regulatory and operational progress in Q2 2025, including FDA alignment for the pivotal Phase 2 AQUAx2 study for radiation-induced xerostomia (RIX), RMAT designation for AAV-GAD in Parkinson's disease, and plans to file for Marketing Authorization Approval for LCA4 in the UK and US - Gained alignment with U.S. Food and Drug Administration (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 Biologics License Application (BLA) filing; on track for potential data readout late 2026[1] - FDA Granted Regenerative Medicine Advanced Therapy (RMAT) designation for AAV-GAD for the treatment of Parkinson's disease[1] - On track to file 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 BLA in the US with the FDA via a similar pathway to approval in the fourth quarter of 2025[1] CEO Statement Dr. Alexandria Forbes highlighted productive regulatory interactions, successful FDA audit for AAV-GAD, and the advancement of late-stage clinical programs as planned - CEO emphasized productive regulatory interactions with MHRA and FDA around multiple later stage clinical programs as well as manufacturing[2] - Expressed excitement about the potential of AAV-GAD to show disease modifying changes to pathological circuitry and the substantia nigra, and the opportunity to discuss the use of AI analysis of imaging data to support labeling claims for the proposed Phase 3 study in Parkinson's disease[2][3] - Engaged with regulators to be in a position to initiate first-in-human studies using the transformative riboswitch platform by the end of 2025[3] Pipeline & Clinical Development Updates MeiraGTx advanced multiple late-stage clinical programs, including AAV2-hAQP1 for RIX, AAV-GAD for Parkinson's, AAV-AIPL1 for LCA4, and bota-vec for XLRP, achieving key regulatory designations and publishing compelling data AAV2-hAQP1 for Radiation-Induced Xerostomia (RIX) The AAV2-hAQP1 program for RIX received RMAT designation and achieved FDA alignment on clinical and CMC requirements for its pivotal Phase 2 AQUAx2 study, on track for data readout in late 2026 - Granted RMAT designation by the FDA for AAV2-hAQP1 for the treatment of Grade 2/3 RIX in December 2024[5] - Aligned with the FDA on both the CMC and clinical requirements for the ongoing Phase 2 AQUAx2 study, including the use of a single Patient Reported Outcome (PRO) as primary endpoint and statistical analyses[5] - The Phase 2 AQUAx2 study is currently enrolling the final high dose cohorts, targeting completion of enrollment in the fourth quarter of 2025, with potential for pivotal data readout late 2026[5] AAV-GAD for Parkinson's Disease AAV-GAD received RMAT designation for Parkinson's disease, with a joint venture formed with Hologen AI to use AI-driven imaging analysis for a proposed Phase 3 study aiming for a disease modification claim - FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to AAV-GAD for the treatment of Parkinson's disease on May 8th, 2025, following positive data from 3 clinical studies[5][6] - MeiraGTx and partner Hologen are currently in discussion with the FDA around use of AI driven analysis of imaging data from the proposed double-blind, sham-surgery controlled Phase 3 study with the potential to support a disease modification claim on the label for AAV-GAD[8] - MeiraGTx and Hologen are forming a joint venture, Hologen Neuro AI Ltd, with additional committed funding from Hologen of up to $230 million to finance the development of the AAV-GAD program to commercialization. MeiraGTx will hold a 30% ownership and lead clinical development and manufacturing[8] AAV-AIPL1 for LCA4 Published data for AAV-AIPL1 for LCA4 showed meaningful responses in all treated children, leading to preparations for MAA and BLA submissions supported by Orphan Drug and Rare Pediatric Disease Designations - Data demonstrating the efficacy of rAAV8.hRKp.AIPL1 for the treatment of LCA4 were published in The Lancet in February 2025, showing meaningful responses in 11 out of 11 treated children, with all children blind at birth now able to see[13] - Preparing the submission of an MAA in the UK and a BLA in the US for AAV-AIPL1, a treatment for LCA4, based on clinical data and feedback from MHRA and FDA on a potentially expedited CMC PPQ package[13] - AAV-AIPL1 for the treatment of LCA4 has orphan drug designation in the US and EU, and Rare Pediatric Disease Designation (RPDD) from the FDA[13] Botaretigene Sparoparvovec (bota-vec) for X-linked Retinitis Pigmentosa (XLRP) Compelling Phase 3 data for bota-vec for XLRP received strong support, with the program holding Fast Track and orphan drug designations and MeiraGTx eligible for significant milestone payments - Compelling Phase 3 data for botaretigene sparoparvovec (bota-vec) for the treatment of X-linked retinitis pigmentosa was presented, leading to strong public support for its filing and ultimate approval from the Foundation Fighting Blindness[13] - The FDA has granted Fast Track and orphan drug designations to bota-vec, and EU regulatory authorities have granted Priority Medicines (PRIME), advanced therapy medicinal product (ATMP), and orphan drug designations[13] - MeiraGTx is eligible to receive up to $285 million upon the first commercial sales of bota-vec in the US and EU and manufacturing tech transfer, and anticipates additional revenue from a commercial supply agreement with Johnson & Johnson Innovative Medicine[13][14] Technology Platforms MeiraGTx is advancing its riboswitch gene regulation technology for in vivo delivery, focusing on metabolic diseases and CAR-T, and acquired ProTcell technology to enhance allogeneic RiboCAR-T therapies Riboswitch Gene Regulation Technology The riboswitch platform is progressing for in vivo delivery, initially focusing on obesity, metabolic disease, neuropathic pain, and CAR-T, with first-in-human studies anticipated in 2025 - MeiraGTx continues to progress its riboswitch technology platform in multiple potential indications, with an initial focus on obesity and metabolic disease, neuropathic pain and CAR-T[18] - Generated compelling preclinical data with metabolic peptides and hormones, including incretins, myokines and leptin, indicating greater efficacy on weight loss and positive impact on fat to muscle ratio and post prandial glucose control[18] - Pre-clinical data from Riboswitch delivered leptin is particularly compelling and likely to be the first IND using the Company's riboswitch small molecule platform, with first-in-human studies intended to initiate in 2025[18] ProTcell Technology & RiboCAR-T MeiraGTx acquired ProTcell technology to complement its RiboCAR platform, enabling the generation of T-cell progenitors outside the body for allogeneic high-performance RiboCAR-T therapies - Acquired ProTcell technology via the acquisition of certain assets and operations of Smart Immune, which allows T-cell progenitors to be generated outside the body, complementing the Company's RiboCAR platform[18] - This technology provides a unique potential for allogeneic high performance RiboCAR-T, with ProTcell technology having shown proof of concept in 20 patients treated in 3 clinical studies[18] Manufacturing Capabilities MeiraGTx maintains robust manufacturing capabilities with two licensed facilities in the UK and Ireland, ensuring GMP compliance and readiness for commercial production of gene therapies United Kingdom Facility MeiraGTx's UK manufacturing facility holds two MHRA authorizations and successfully renewed its licenses following a May 2024 inspection, confirming GMP compliance for IMPs and readiness for a commercial MIA license application - MeiraGTx's UK manufacturing facility holds two authorizations issued by the MHRA[16] - The UK facility was inspected in May 2024, and the licences were successfully renewed, confirming GMP compliance for Investigational Medicinal Products (IMPs) and readiness to support an application for a commercial MIA licence[16] Ireland Facility MeiraGTx's Shannon facility holds two HPRA authorizations, including an MIA Licence for QC testing and an MIA(IMP) Licence for manufacturing, with a recent inspection adding viral vector manufacturing capability - MeiraGTx's Shannon facility holds two authorizations issued by Ireland's Health Products Regulatory Authority (HPRA): MIA Licence (M1316) for QC testing of commercial products and MIA(IMP) Licence (MIA(IMP) 45522) for manufacturing, fill-finish, and QC testing of Investigational Medicinal Products (IMPs)[17][18] - The latest HPRA inspection in February 2025 was highly successful, renewing both QC licenses and adding viral vector manufacturing to the MIA(IMP) license, a first-of-its-kind license for a gene therapy facility in Ireland[20] Financial Review MeiraGTx reported $32.2 million in cash as of Q2 2025, with a positive financial outlook into 2027 supported by the Hologen collaboration and potential bota-vec milestones, while Q2 2025 saw increased service revenue and an improved net loss Financial Outlook & Liquidity As of June 30, 2025, MeiraGTx had $32.2 million in cash and cash equivalents, expecting to fund operations into 2027 and repay its $75.0 million debt with anticipated proceeds from the Hologen collaboration and bota-vec milestones Cash and Receivables (as of June 30, 2025) | Item | Amount (Millions) | | :-------------------------- | :---------------- | | Cash and cash equivalents | $32.2 | | Receivables (Johnson & Johnson) | $2.3 | | Tax incentive receivables | $4.5 | - The Company believes that with current funds, $17.0 million received in Q3 2025, and remaining proceeds from the Hologen collaboration, it will have sufficient capital to fund operating expenses and capital expenditure requirements into 2027 and to repay its debt obligation of $75.0 million (due August 2026)[21] - This estimate does not include the $285.0 million in milestones the Company is eligible to receive under the asset purchase agreement upon first commercial sale of bota-vec in the US and EU, manufacturing tech transfer, and regulatory approval of J&J's manufacturing facility[21] Q2 2025 Financial Results For Q2 2025, MeiraGTx reported a significant increase in service revenue to $3.7 million, with a net loss attributable to ordinary shareholders improving to $38.8 million, resulting in a lower basic and diluted net loss per share of $0.48 Q2 2025 vs Q2 2024 Key Financials (in thousands) | Metric | Q2 2025 | Q2 2024 | Change (YoY) | | :-------------------------------- | :------ | :------ | :----------- | | Service revenue | $3,691 | $282 | +$3,409 | | Cost of service revenue | $2,676 | $0 | +$2,676 | | General and administrative expenses | $12,313 | $11,257 | +$1,056 | | Research and development expenses | $33,495 | $34,934 | -$1,439 | | Foreign currency gain (loss) | $8,624 | $(284) | +$8,908 | | Interest income | $408 | $827 | -$419 | | Interest expense | $3,034 | $3,254 | -$220 | | Net loss | $(38,795) | $(48,620) | +$9,825 | | Basic and diluted net loss per share | $(0.48) | $(0.76) | +$0.28 | - Service revenue increased by $3.4 million for the three months ended June 30, 2025, compared to the prior year, due to increased progress of PPQ services under the asset purchase agreement with Johnson & Johnson Innovative Medicine[22] - Research and development expenses decreased by $1.4 million, primarily due to reclassification of batch costs and cost of service revenue, and reduced expenses for certain programs, partially offset by increased clinical trial expenses for the AAV-GAD program[25] - Foreign currency gain was $8.6 million for Q2 2025, a significant change from a $0.3 million loss in Q2 2024, primarily due to the weakening of the U.S. dollar against the pound sterling and euro[26] Company Overview MeiraGTx is a vertically integrated, clinical-stage genetic medicines company with a broad pipeline of four late-stage clinical programs, leveraging innovative technology for capsid, promoter, and translational control optimization, supported by extensive in-house manufacturing capabilities and a transformative riboswitch gene regulation technology - MeiraGTx is a vertically integrated, clinical-stage genetic medicines company with a broad pipeline of four late-stage clinical programs, using local delivery of small doses for disease modifying effects in inherited and common diseases[30] - Utilizes innovative technology in optimization of capsids, promoters and novel translational control elements to develop best in class, potent, safe viral vectors[30] - Possesses comprehensive end-to-end in-house manufacturing capabilities with 5 facilities globally, including two licensed for GMP viral vector production and a GMP QC facility with clinical and commercial licensure[30] - Developed a novel riboswitch gene regulation technology for in vivo delivery of any biologic therapeutic using oral small molecules, focusing on regulated in vivo delivery of metabolic peptides (e.g., GLP-1, Leptin) and cell therapy (CAR-T)[31] Forward-Looking Statements This section contains forward-looking statements regarding product development, clinical trial milestones, regulatory matters, potential milestone payments, and the Hologen collaboration, which are subject to risks and uncertainties - This press release contains forward-looking statements regarding product candidate development, anticipated milestones, clinical data, regulatory matters, potential milestone payments, and the collaboration with Hologen[32] - These statements are based on management's current expectations and involve known and unknown risks, uncertainties, and other important factors that may cause actual results, performance or achievements to be materially different[32] - The company disclaims any obligation to update such forward-looking statements at some point in the future, unless required by law[33] Contacts This section provides contact information for investor relations and media inquiries Financial Statements The financial statements present the unaudited condensed consolidated statements of operations and balance sheets, detailing the company's financial performance and position for Q2 2025 and year-to-date Condensed Consolidated Statements of Operations and Comprehensive Loss The unaudited condensed consolidated statements of operations show a net loss of $38.8 million for Q2 2025, an improvement from $48.6 million in Q2 2024, driven by increased service revenue and slightly decreased R&D expenses Condensed Consolidated Statements of Operations and Comprehensive Loss (Q2 2025 vs Q2 2024, in thousands) | Metric | Q2 2025 | Q2 2024 | 6M 2025 | 6M 2024 | | :-------------------------------- | :------ | :------ | :------ | :------ | | Service revenue | $3,691 | $282 | $5,617 | $979 | | Cost of service revenue | $2,676 | — | $4,054 | — | | General and administrative | $12,313 | $11,257 | $21,677 | $24,404 | | Research and development | $33,495 | $34,934 | $66,275 | $69,256 | | Loss from operations | $(44,793) | $(45,909) | $(86,389) | $(92,681) | | Foreign currency gain (loss) | $8,624 | $(284) | $12,311 | $(819) | | Interest income | $408 | $827 | $1,379 | $1,924 | | Interest expense | $(3,034) | $(3,254) | $(6,077) | $(6,504) | | Net loss | $(38,795) | $(48,620) | $(78,776) | $(69,062) | | Basic and diluted net loss per share | $(0.48) | $(0.76) | $(0.99) | $(1.08) | | Weighted-average shares outstanding | 80,585,625 | 64,376,396 | 79,813,273 | 64,221,145 | Condensed Consolidated Balance Sheets The unaudited condensed consolidated balance sheets show total assets decreased to $198.7 million as of June 30, 2025, from $269.8 million at December 31, 2024, with a significant decrease in cash and cash equivalents Condensed Consolidated Balance Sheets (as of June 30, 2025 vs Dec 31, 2024, in thousands) | Asset/Liability/Equity | June 30, 2025 | December 31, 2024 | | :-------------------------------- | :------------ | :---------------- | | ASSETS: | | | | Cash and cash equivalents | $32,166 | $103,659 | | Total Current Assets | $47,084 | $123,518 | | Property, plant and equipment, net | $110,816 | $102,878 | | TOTAL ASSETS | $198,716 | $269,751 | | LIABILITIES: | | | | Total Current Liabilities | $53,436 | $60,783 | | Deferred revenue - related party | $63,044 | $57,576 | | Note payable, net | $73,773 | $73,221 | | TOTAL LIABILITIES | $195,756 | $201,924 | | SHAREHOLDERS' EQUITY: | | | | Total Shareholders' Equity | $2,960 | $67,827 |