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MeiraGTx(MGTX) - 2025 Q1 - Quarterly Results
MeiraGTxMeiraGTx(US:MGTX)2025-05-13 12:05

Executive Summary / Q1 2025 Highlights Strategic Collaboration with Hologen AI MeiraGTx formed a strategic collaboration with Hologen AI, receiving a $200 million upfront payment and establishing Hologen Neuro AI Ltd, with Hologen committing an additional $230 million for AAV-GAD Parkinson's disease development - MeiraGTx received a $200 million cash upfront payment from Hologen AI134 - A joint venture, Hologen Neuro AI Ltd, was established, with Hologen committing an additional $230 million to accelerate AAV-GAD development for Parkinson's disease134 - MeiraGTx will hold a 30% equity stake in the joint venture and lead all clinical development and manufacturing4 Regulatory and Clinical Milestones The company achieved significant Q1 2025 regulatory and clinical progress, including AAV-GAD RMAT designation, AAV2-hAQP1 Phase 2 AQUAx2 FDA alignment, and positive AAV-AIPL1 LCA4 data published in The Lancet with planned MAA submission - AAV-GAD received FDA Regenerative Medicine Advanced Therapy (RMAT) designation for Parkinson's disease treatment1210 - The Phase 2 AQUAx2 study for AAV2-hAQP1 in radiation-induced xerostomia (RIX) reached FDA alignment to support a potential BLA submission1211 - Efficacy data for rAAV8.hRKp.AIPL1 in AIPL1-related retinal dystrophy (LCA4) was published in The Lancet, showing meaningful responses in all 11 treated children115 - The company plans to submit a conditional Marketing Authorization Application (MAA) for AAV-AIPL1 to the UK MHRA and discuss similar US approval pathways with the FDA115 Recent Development Highlights AAV-GAD for Parkinson's Disease AAV-GAD received FDA RMAT designation based on three positive clinical studies, including two double-blind, sham-controlled Phase 2 trials showing significant motor benefits and AI-driven evidence of disease-modifying changes - AAV-GAD received FDA RMAT designation on May 8, 2025, for Parkinson's disease inadequately controlled by anti-Parkinsonian medications10 - RMAT designation was based on three positive clinical studies, including one Phase 1 and two double-blind, sham-controlled Phase 2 trials10 - Phase 2 study data demonstrated significant clinical benefit in the UPDRS Part 3 standard motor endpoint for AAV-GAD10 - Hologen's AI analysis confirmed AAV-GAD treatment leads to disease-modifying changes in Parkinson's brain circuits and potential protective changes in substantia nigra and cognitive/emotional brain regions310 - The company plans to initiate a Phase 3 study for AAV-GAD in H2 202510 AAV2-hAQP1 for the Treatment of Xerostomia AAV2-hAQP1 received FDA RMAT designation in December 2024 for Grade 2/3 radiation-induced xerostomia; the company aligned with FDA on Phase 2 AQUAx2 study requirements, targeting Q4 2025 enrollment completion and potential BLA submission by late 2026 - AAV2-hAQP1 received FDA RMAT designation in December 2024 for Grade 2/3 radiation-induced xerostomia (RIX)11 - The company reached FDA alignment on clinical and CMC requirements for the ongoing Phase 2 AQUAx2 study to support a potential BLA submission11 - The Phase 2 AQUAx2 study is actively enrolling and dosing across multiple centers in the US, Canada, and UK, targeting enrollment completion in Q4 20251112 - A potential BLA submission is anticipated by late 202612 AAV-AIPL1 for LCA4 Caused by Mutations in the AIPL1 Gene Positive AAV-AIPL1 efficacy data for LCA4, showing significant improvement in all 11 treated children, was published in The Lancet; the company is preparing a conditional MAA for MHRA and discussing accelerated US approval with FDA, having secured Orphan Drug and Rare Pediatric Disease designations - In February 2025, The Lancet published efficacy data for rAAV8.hRKp.AIPL1 in LCA4, showing meaningful responses in all 11 treated children15 - The company is preparing to submit a conditional Marketing Authorization Application (MAA) to the MHRA, requiring no further clinical data15 - Alignment has been reached with the FDA on clinical, non-clinical, and CMC requirements to advance potential accelerated approval in the US15 - AAV-AIPL1 has received US Orphan Drug designation, EU Orphan designation, and FDA Rare Pediatric Disease designation15 Botaretigene Sparoparvovec for the Treatment of X-linked Retinitis Pigmentosa (XLRP) Botaretigene sparoparvovec (bota-vec) Phase 3 LUMEOS trial missed its primary endpoint but showed directional support and benefits across most secondary endpoints; the therapy is safe, has FDA Fast Track and Orphan Drug, and EU PRIME and Orphan Drug designations, with MeiraGTx eligible for up to $285 million in milestone payments - The Phase 3 LUMEOS trial for botaretigene sparoparvovec (bota-vec) missed its primary endpoint (visual mobility assessment) but showed directional support15 - All secondary endpoints, except one, including functional vision, retinal function, and visual function, demonstrated benefit1518 - Bota-vec's safety profile was as expected and manageable, with no new safety signals and over 90% of treatment-related inflammatory adverse events being mild18 - Bota-vec has received FDA Fast Track and Orphan Drug designations, along with EU Priority Medicines (PRIME) and Orphan Drug designations18 - MeiraGTx is eligible for up to $285 million in milestone payments upon bota-vec's first commercial sale and manufacturing technology transfer18 Riboswitch Gene Regulation Technology Platform for in vivo Delivery MeiraGTx is advancing its riboswitch technology platform, initially targeting obesity, metabolic diseases, neuropathic pain, and CAR-T therapies, with compelling preclinical data and plans to initiate first-in-human studies in 2025 - The company is advancing its riboswitch technology platform, initially focusing on obesity and metabolic diseases, neuropathic pain, and CAR-T18 - Preclinical data for metabolic peptides and hormones (including incretins, amylin, and leptin) showed greater efficacy in weight loss and fat-to-lean muscle ratio18 - The company plans to initiate first-in-human studies using the riboswitch platform in 202518 Manufacturing Capabilities United Kingdom (MeiraGTx UK II Ltd.) MeiraGTx's UK manufacturing facility holds two MHRA authorizations (MIA(IMP) and Special Licence), successfully renewed in May 2024, confirming GMP compliance and supporting commercial MIA license applications - The UK manufacturing facility holds MIA(IMP) and Special Licence authorizations from the MHRA1923 - Following a May 2024 inspection, licenses were successfully renewed, confirming GMP compliance and supporting commercial MIA license applications19 Ireland (MeiraGTx Ireland DAC) MeiraGTx's Shannon facility holds two HPRA authorizations (MIA and MIA(IMP)), with its QC lab actively conducting PPQ batch release and stability testing; a successful February 2025 HPRA inspection renewed two QC licenses and added viral vector manufacturing to the MIA(IMP) license, a first for an Irish gene therapy facility - The Shannon facility in Ireland holds MIA and MIA(IMP) licenses from the HPRA2024 - The QC laboratory actively conducts release and stability testing for PPQ batches20 - A successful February 2025 HPRA inspection renewed two QC licenses and added viral vector manufacturing to the MIA(IMP) license, a first for an Irish gene therapy facility21 Financial Results Cash Position and Liquidity As of March 31, 2025, MeiraGTx's cash and cash equivalents were approximately $66.5 million, down from $120.3 million on March 31, 2024; the company expects existing funds, combined with Hologen collaboration proceeds, to support operations and capital expenditures through 2027 and repay $75 million debt due August 2026 Cash, Cash Equivalents, and Restricted Cash | Metric | March 31, 2025 (Millions of USD) | March 31, 2024 (Millions of USD) | | :--- | :--- | :--- | | Cash, Cash Equivalents, and Restricted Cash | 68.6 | 120.3 | - As of March 31, 2025, cash and cash equivalents were approximately $66.5 million22 - The company anticipates that existing capital, combined with proceeds from the Hologen collaboration, will be sufficient to fund operations and capital expenditures through 2027 and repay $75 million debt due August 202622 Statement of Operations (Income Statement) In Q1 2025, the company saw year-over-year service revenue growth but a decrease in total operating expenses; net loss increased from $20.4 million in Q1 2024 to $39.98 million, primarily due to the absence of non-financial asset sale gains from the prior year Key Financial Data for Q1 2025 (Compared to Q1 2024) | Metric | March 31, 2025 (Thousands of USD) | March 31, 2024 (Thousands of USD) | Change (Thousands of USD) | Change Rate | | :--- | :--- | :--- | :--- | :--- | | Service revenue - related party | 1,926 | 697 | 1,229 | +176.3% | | Cost - related party service revenue | 1,378 | — | 1,378 | N/A | | General and administrative expenses | 9,364 | 13,147 | (3,783) | -28.8% | | Research and development expenses | 32,780 | 34,322 | (1,542) | -4.5% | | Operating loss | (41,596) | (46,772) | 5,176 | -11.1% | | Foreign currency exchange gain (loss) | 3,687 | (535) | 4,222 | N/A | | Interest income | 971 | 1,097 | (126) | -11.5% | | Interest expense | (3,043) | (3,250) | 207 | -6.4% | | Gain on sale of non-financial assets | — | 29,018 | (29,018) | -100.0% | | Net loss | (39,981) | (20,442) | (19,539) | +95.6% | | Basic and diluted net loss per share | (0.51) | (0.32) | (0.19) | +59.4% | | Weighted average common shares outstanding | 79,032,341 | 64,065,895 | 14,966,446 | +23.4% | - Service revenue increased by $1.2 million, primarily due to accelerated progress on process performance qualification (PPQ) services under the asset purchase agreement with Johnson & Johnson Innovative Medicine27 - General and administrative expenses decreased by $3.8 million, mainly due to lower share-based compensation, legal and accounting fees, consulting fees, and other office-related costs, along with a gain from early termination of a lease agreement, partially offset by increased salaries and related costs29 - Research and development expenses decreased by $1.5 million, primarily due to reclassification of manufacturing costs and reduced R&D expenses, partially offset by lower reimbursements from Johnson & Johnson Innovative Medicine, increased clinical trial expenses for the AAV-hAQP1 program and other ophthalmology programs, and higher preclinical program expenses30 - Foreign currency exchange shifted from a loss to a gain, primarily due to the weakening of the US dollar against the British Pound and Euro31 - Gain on sale of non-financial assets decreased from $29 million to $0, leading to a significant increase in net loss3536 Balance Sheet Highlights As of March 31, 2025, total assets were $224 million, a decrease from $269.75 million on December 31, 2024; total liabilities were $190.9 million, and total stockholders' equity was $33.1 million Key Balance Sheet Data (As of March 31, 2025 vs. December 31, 2024) | Metric | March 31, 2025 (Thousands of USD) | December 31, 2024 (Thousands of USD) | | :--- | :--- | :--- | | Cash and cash equivalents | 66,523 | 103,659 | | Total current assets | 79,889 | 123,518 | | Total assets | 224,014 | 269,751 | | Total current liabilities | 52,372 | 60,783 | | Total liabilities | 190,915 | 201,924 | | Total stockholders' equity | 33,099 | 67,827 | About MeiraGTx MeiraGTx is a vertically integrated clinical-stage gene therapy company with a broad pipeline, including four late-stage clinical programs, focusing on localized, low-dose delivery for genetic and common diseases, supported by comprehensive in-house manufacturing across five global facilities, two with GMP viral vector licenses, and an innovative riboswitch gene regulation platform for precise, dose-responsive gene expression control via oral small molecules in metabolic peptides, cell therapies, and chronic pain - MeiraGTx is a vertically integrated clinical-stage gene therapy company with four late-stage clinical programs37 - The company possesses comprehensive in-house manufacturing capabilities with five global facilities, including two with GMP viral vector manufacturing licenses and one GMP QC facility licensed for clinical and commercial use37 - MeiraGTx has developed an innovative riboswitch gene regulation technology for precise, dose-responsive control of gene expression via oral small molecules37 - The riboswitch platform focuses on metabolic peptides (e.g., GLP-1, GIP, glucagon, amylin, PYY, and leptin), CAR-T cell therapies, and PNS targets for intractable pain37 Forward Looking Statement This press release contains forward-looking statements as defined by the Private Securities Litigation Reform Act of 1995, covering product candidate development, clinical data, regulatory matters, milestone payments, Hologen collaboration, AI technology efficacy, and manufacturing technology development, which are based on current management expectations and involve known and unknown risks, uncertainties, and other factors that may cause actual results to differ materially Contacts Provides contact information for investors and media