MeiraGTx Announces Third Quarter 2024 Financial and Operational Results and Recent Business Updates
MeiraGTx announced significant Q3 2024 developments, including three FDA Rare Pediatric Disease Designations for inherited retinal therapies. The company reported positive data from its AAV-GAD Parkinson's disease study, showing significant improvements in key efficacy endpoints. The MHRA agreed to review a Marketing Authorization Application for AAV-AIPL1 without further clinical studies. Financial highlights include $122.9 million in cash and equivalents, with funding projected into Q2 2026. Q3 service revenue was $10.9 million, with a net loss of $39.3 million ($0.55 per share), compared to $44.3 million ($0.74 per share) in Q3 2023.
MeiraGTx ha annunciato sviluppi significativi per il terzo trimestre del 2024, inclusi tre riconoscimenti da parte della FDA per malattie rare pediatriche per terapie retiniche ereditarie. L'azienda ha riportato dati positivi dal suo studio sull'AAV-GAD per il morbo di Parkinson, mostrando miglioramenti significativi nei principali endpoint di efficacia. L'MHRA ha accettato di esaminare una domanda di autorizzazione al commercio per l'AAV-AIPL1 senza ulteriori studi clinici. Tra i punti salienti finanziari, ci sono 122,9 milioni di dollari in contante e equivalenti, con finanziamenti previsti fino al secondo trimestre del 2026. Le entrate dai servizi per il terzo trimestre sono state di 10,9 milioni di dollari, con una perdita netta di 39,3 milioni di dollari (0,55 dollari per azione), rispetto ai 44,3 milioni di dollari (0,74 dollari per azione) nel terzo trimestre del 2023.
MeiraGTx anunció desarrollos significativos para el tercer trimestre de 2024, incluidos tres designaciones de enfermedad pediátrica rara de la FDA para terapias retinianas hereditarias. La compañía reportó datos positivos de su estudio sobre AAV-GAD para la enfermedad de Parkinson, mostrando mejoras significativas en los principales puntos finales de eficacia. La MHRA aceptó revisar una Solicitud de Autorización de Comercialización para AAV-AIPL1 sin más estudios clínicos. Los aspectos financieros destacados incluyen 122.9 millones de dólares en efectivo y equivalentes, con financiamiento proyectado hasta el segundo trimestre de 2026. Los ingresos por servicios del tercer trimestre fueron de 10.9 millones de dólares, con una pérdida neta de 39.3 millones de dólares (0.55 dólares por acción), en comparación con 44.3 millones de dólares (0.74 dólares por acción) en el tercer trimestre de 2023.
MeiraGTx는 2024년 3분기에 중요한 발전을 발표했으며, 유전성 망막 요법에 대해 FDA의 희귀 소아 질병 지정을 3건 받았습니다. 이 회사는 AAV-GAD 파킨슨병 연구에서 긍정적인 데이터를 보고했으며, 주요 효능 끝점에서 상당한 개선을 보여주었습니다. MHRA는 추가 임상 연구 없이 AAV-AIPL1에 대한 마케팅 허가 신청서를 검토하기로 합의했습니다. 재무 하이라이트에는 1억 2,290만 달러의 현금 및 동등 자산이 있으며, 자금 지원은 2026년 2분기까지 예상됩니다. 3분기 서비스 수익은 1,090만 달러였으며, 순손실은 3,930만 달러(주당 0.55달러)로, 2023년 3분기의 4,430만 달러(주당 0.74달러)와 비교됩니다.
MeiraGTx a annoncé des développements significatifs pour le troisième trimestre 2024, y compris trois désignations de maladies pédiatriques rares par la FDA pour des thérapies rétiniennes héréditaires. La société a rapporté des données positives de son étude AAV-GAD sur la maladie de Parkinson, montrant des améliorations significatives dans les principaux critères d'efficacité. La MHRA a accepté d'examiner une demande d'autorisation de mise sur le marché pour l'AAV-AIPL1 sans études cliniques supplémentaires. Les points saillants financiers comprennent 122,9 millions de dollars en espèces et équivalents, avec des financements projetés jusqu'au deuxième trimestre 2026. Les revenus des services pour le troisième trimestre s'élevaient à 10,9 millions de dollars, avec une perte nette de 39,3 millions de dollars (0,55 dollar par action), contre 44,3 millions de dollars (0,74 dollar par action) au troisième trimestre 2023.
MeiraGTx hat bedeutende Entwicklungen für das 3. Quartal 2024 angekündigt, darunter drei FDA-Kennzeichnungen für seltene pädiatrische Erkrankungen für erbliche Netzhautherapien. Das Unternehmen berichtete von positiven Daten aus seiner AAV-GAD-Studie zur Parkinson-Krankheit, die signifikante Verbesserungen bei den wichtigsten Wirksamkeitskriterien zeigten. Die MHRA stimmte dem Prüfungsverfahren für einen Antrag auf Marktzulassung für AAV-AIPL1 ohne weitere klinische Studien zu. Zu den finanziellen Highlights gehören 122,9 Millionen Dollar in Barmitteln und -äquivalenten, mit einer Finanzierung bis ins 2. Quartal 2026. Die Serviceumsätze im 3. Quartal betrugen 10,9 Millionen Dollar, mit einem Nettoverlust von 39,3 Millionen Dollar (0,55 Dollar pro Aktie), gegenüber 44,3 Millionen Dollar (0,74 Dollar pro Aktie) im 3. Quartal 2023.
- Received three Rare Pediatric Disease Designations from FDA, potentially worth $150-158M each upon approval
- Positive clinical data from AAV-GAD Parkinson's study showing significant efficacy improvements
- MHRA agreed to review AAV-AIPL1 marketing application without additional clinical studies
- Strong cash position of $122.9M with runway into Q2 2026
- Eligible for $285M milestone payments upon first commercial sales of bota-vec
- Net loss of $39.3M in Q3 2024
- Increased general and administrative expenses by $2.7M year-over-year
- No license revenue in Q3 2024 compared to $5.1M in Q3 2023
Insights
The Q3 2024 results reveal significant regulatory and clinical progress for MeiraGTx. Three key developments stand out: Rare Pediatric Disease Designations for three retinal therapies, a potential expedited Marketing Authorization pathway for AAV-AIPL1 in the UK and positive clinical data for AAV-GAD in Parkinson's disease.
The financial position shows
The RPDD designations are particularly valuable, as recent Priority Review Vouchers have sold for
The company's financial structure is evolving with the J&J partnership generating service revenue but showing increased operational costs. The
The cash runway extension into Q2 2026 provides adequate operational flexibility. The foreign currency position improved significantly, with a
- Received 3 Rare Pediatric Disease Designations (RPDD) from FDA for each of 3 potential therapies for 3 different rare inherited retinopathies including AAV-AIPL1
- Agreed on pathway with MHRA for Marketing Authorization Application (MAA) under exceptional circumstances for AAV-AIPL1 for the treatment of Leber congenital amaurosis (LCA4) retinal dystrophy without further clinical studies
- Announced positive data from randomized, sham-controlled clinical bridging study of AAV-GAD for the treatment of Parkinson’s disease
LONDON and NEW YORK, Nov. 13, 2024 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage genetic medicines company, today announced financial and operational results for the third quarter ended September 30, 2024, and provided a corporate update. The Company also announced that following meetings with the UK Medicines and Healthcare products Regulatory Agency (MHRA), the Company intends to submit a Marketing Authorization Application under exceptional circumstances for AAV-AIPL1 in the United Kingdom without the need for further clinical studies. The Company is also initiating discussions with the FDA around the potential for a similar pathway to approval in the U.S. In addition, MeiraGTx announced that the Offices of Orphan Products Development and Pediatric Therapeutics of the U.S. Food and Drug Administration (FDA) have granted the Company three Rare Pediatric Disease Designations to its AAV8-RK-AIPL1 program, AAV8-RK-BBS10 program, and AAV5-RDH12 program, each for the treatment of inherited retinal diseases.
“The past few months at MeiraGTx have been highlighted by exceptional clinical, regulatory, and research and development advancements,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. “In October, we announced positive data from our AAV-GAD bridging study using material manufactured in house at MeiraGTx. This study was a randomized, sham-controlled clinical study of AAV-GAD for the treatment of Parkinson’s disease, demonstrating that the treatment is safe and leads to significant and clinically meaningful improvements in key efficacy endpoints including UPDRS Part 3 ’off’ score and PDQ-39 quality of life measure. Based on these extremely promising results, we are engaging with global regulatory agencies to initiate a Phase 3 registrational study.”
Dr. Forbes added, “We also just received RPDD for three additional programs in our pipeline, a remarkable regulatory achievement for the Company. This underscores the groundbreaking therapeutic potential of our technology to uniquely address these severe childhood blinding conditions and offer hope to the families impacted. Upon FDA approval of a product with RPDD, we are eligible to receive a priority review voucher which could provide meaningful non-dilutive capital, as such vouchers have sold for
Dr. Forbes continued, “The AIPL1 program exemplifies how MeiraGTx has leveraged our internal manufacturing infrastructure and clinical expertise and worked with regulators to expedite by many years the delivery of these potentially life changing treatments to affected children. By releasing AAV-AIPL1 under our MHRA manufacturing specials license, MeiraGTx was uniquely placed to provide expert clinicians with a potential therapy for these children prior to formal clinical studies. LCA4 caused by mutations in the AIPL1 gene results in blindness from birth, with complete degeneration of the retina by the age of four. Eleven children were treated between 1 and 4 years old with MeiraGTx’s AAV-AIPL1 therapy. All 11 children, each of whom was blind from birth gained vision within 6 weeks of treatment. These extraordinary results supported a successful application to the MHRA Innovative Licensing and Access Pathway (ILAP), and with the award of the Innovation Passport, allowed an expedited Scientific Advice Meeting with the MHRA. During the meeting, agreement was reached that we are in a position to file a Marketing Authorization Application under exceptional circumstance based on the data from these 11 children with no further clinical studies required. In addition, because of our end-to-end internal manufacturing infrastructure, we have also agreed on the CMC requirement for approval. We have already engaged with the FDA to discuss a path to potential approval in the U.S., and we will continue to explore this type of expedited approval pathway for AIPL1 with other global agencies while investigating a similar strategy with the other RPDD awarded indications, including BBS10.”
“The potential approval of transformative products for rare and devastating pediatric disorders in an expedited fashion is extremely exciting, allowing us to more rapidly advance potential treatments to severely impacted children many years faster than possible via the standard approval pathway. This is an illustration of the practical importance of optimizing our approach to viral vector development, as well as internalizing full commercial ready CMC capabilities, in developing effective treatments for rare, severe, rapidly degenerative diseases.”
Recent Development Highlights and Anticipated Milestones
AAV-GAD for the Treatment of Parkinson’s Disease:
The primary study objective of safety and tolerability was met and significant and clinically meaningful improvements from baseline were demonstrated for key efficacy endpoints at 26 weeks.
Top-line data summary:
- AAV-GAD was safe and well tolerated, with no serious adverse events (SAEs) related to AAV-GAD treatment.
- At Week 26, a statistically significant 18-point average improvement from baseline in Unified Parkinson’s Disease Rating Scale (UPDRS) Part 3 “off” medication score was demonstrated in the high dose group (p=0.03), with no significant change in the sham or low dose groups. For the UPDRS Part 3 in the “off” state, a change of 5 to 10 points is considered clinically meaningful.
- Significant improvements from baseline in the disease-specific, patient-reported quality of life Parkinson’s Disease Questionnaire (PDQ-39) score were demonstrated in both the high and low dose groups with no significant change in the sham group at Week 26:
- In the high dose AAV-GAD group, the PDQ-39 score improved by 8 points from baseline (p=0.02), the low dose group improved by 6 points from baseline (p=0.04), while the 0.2 point worsening in the sham surgery group was not statistically significant. For the PDQ-39, a 2 to 4-point change is considered clinically meaningful.
- A dose response in PDQ-39 score was observed, with
100% of participants in the high dose group,60% of participants in the low dose group, and25% of participants in the sham surgery group reporting an improvement. - For the PDQ-39 score, there was a trend to significance between the high dose and sham surgery groups at 6 months (n=4 evaluable per group).
AAV-AIPL1 for the Treatment of Leber Congenital Amaurosis (LCA4) Retinal Dystrophy:
- Following recent meetings with the MHRA, the Company intends to submit a Marketing Authorization Application (MAA) under exceptional circumstances for AAV-AIPL1 in the United Kingdom.
- The Company is currently engaging with the FDA to discuss a path forward for regulatory approval in the United States.
- MeiraGTx was awarded an Innovation Passport designation by the U.K. Innovative Licensing and Access Pathway Steering Group for AAV8-RK-AIPL1.
- Meaningful responses have been observed in 11 out of 11 LCA4 children treated to date with AAV-AIPL1. All children were treated between 1 and 4 years old, all were blind on treatment, and all gained visual acuity 4 or more weeks following treatment.
- The Company’s AAV-AIPL1 for the treatment of inherited retinal dystrophy due to defects in the AIPL1 gene has been granted orphan drug and now RPDD by the FDA and orphan designation by the European Commission.
Rare Pediatric Disease Designation Awards from the FDA:
- The Offices of Orphan Products Development and Pediatric Therapeutics of the FDA has granted RPDD to three of MeiraGTx’s inherited retinal disease programs:
- AAV8-RK-AIPL1 for the treatment of LCA4 retinal dystrophy
- AAV8-RK-BBS10 for the treatment of Bardet-Biedl syndrome (BBS) due to BBS10 mutations
- AAV5-RDH12 for the treatment of RDH12 associated retinal dystrophy
An RPDD may be granted by the FDA to drugs and biologics intended to treat certain orphan diseases affecting fewer than 200,000 patients in the U.S., the serious or life-threatening manifestations of which primarily affect individuals aged 18 years or younger. Under the FDA’s Rare Pediatric Disease Priority Review Voucher (PRV) program, a sponsor that receives approval for a biologics license application for a rare pediatric disease may be eligible to receive a voucher for a priority review of a subsequent marketing application for a different product. PRVs may be used by the sponsor or sold to another sponsor for their use and have recently been sold for between
AAV2-hAQP1 for the Treatment of Xerostomia:
- Data from the Company’s Phase 1 AQUAx clinical trial were presented in an oral session at the American Academy of Oral Medicine (AAOM) 2024 annual meeting in April 2024, demonstrating that treatment with AAV2-hAQP1 resulted in significant improvements across three different patient-reported outcomes and in saliva production, with no treatment-related serious adverse events or dose-limiting toxicities reported.
- The Company continues to enroll and dose participants at multiple sites in the U.S., Canada and the U.K. in the Phase 2 AQUAx2 (NCT05926765) randomized, double-blind, placebo-controlled study.
- The Company recently gained alignment with the FDA on requirements for the ongoing Phase 2 AQUAx2 clinical trial for grade 2/3 radiation-induced xerostomia to be considered a pivotal trial in support of a potential BLA filing.
Botaretigene Sparoparvovec for the Treatment of XLRP:
- Data from the Phase 3 LUMEOS trial of botaretigene sparoparvovec (bota-vec) for the treatment of X-linked retinitis pigmentosa in collaboration with Johnson & Johnson Innovative Medicine is expected towards the end of this year. The Company is eligible to receive up to
$285 million upon the first commercial sales of bota-vec in the U.S. and EU and manufacturing tech transfer. - MeiraGTx also entered into a commercial supply agreement with Johnson & Johnson Innovative Medicine for bota-vec manufacturing, which the Company anticipates will generate additional revenue during the product launch.
Riboswitch Gene Regulation Technology Platform for in vivo Delivery:
- MeiraGTx continues to progress its riboswitch technology platform in multiple potential indications, with an initial focus on obesity and metabolic disease and CAR-T for oncology and autoimmune disease.
- The Company continues to generate compelling preclinical data with metabolic peptides and hormones including incretins, myokines and leptin which suggests greater efficacy on weight loss as well as positive impact on fat to muscle ratio with certain novel combinations of peptides.
- The Company is in dialogue with regulatory agencies and intends to initiate first in human studies using the riboswitch platform for an undisclosed metabolic disease indication in 2025.
As of September 30, 2024, MeiraGTx had cash and cash equivalents of approximately
Financial Results
Cash, cash equivalents and restricted cash were
Service revenue was
There was no license revenue for the three months ended September 30, 2024, compared to
Cost of service revenue was
General and administrative expenses were
Research and development expenses for the three months ended September 30, 2024 were
Foreign currency gain was
Interest income was
Interest expense was
Loss on sale of nonfinancial assets was
Net loss attributable to ordinary shareholders for the quarter ended September 30, 2024, was
About AAV8-RK-AIPL1
AAV8-RK-AIPL1 is an investigational genetic medicine for the treatment of one of the most severe forms of Leber congenital amaurosis (LCA) owing to genetic deficiency of Aryl-hydrocarbon-interacting protein-like 1 (AIPL1). It is delivered via subretinal injection to children, and through a one-time administration, AAV8-RK-AIPL1 is designed to deliver functional copies of the AIPL1 gene to cone and rod photoreceptors in the central retina to slow further degeneration and restore vision.
About AAV8-RK-BBS10
The investigational genetic medicine AAV8-RK-BBS10 is an adeno-associated virus with a serotype 8 capsid with a complementary DNA (cDNA) encoding the human BBS10 gene for treatment of Bardet-Biedl syndrome (BBS) due to BBS10 mutations. BBS is a rare genetic disease affecting approximately 1 in 250,000 people around the world. One of the primary symptoms of BBS is visual impairment secondary to retinal degeneration. More than 20 different genes are associated with the development of BBS, with BBS10 accounting for approximately
About AAV5-RDH12
The investigational genetic medicine AAV5-RDH12 is an adeno-associated virus serotype 5 containing the human RDH12 gene for treatment of RDH12 associated retinal dystrophy. Defects in retinol dehydrogenase 12 (RDH12) account for 3–
About MeiraGTx
MeiraGTx (Nasdaq: MGTX) is a vertically integrated, clinical-stage genetic medicines company with a broad pipeline of late-stage clinical programs supported by end-to-end manufacturing capabilities. MeiraGTx has internal plasmid production for GMP, two GMP viral vector production facilities as well as an in-house Quality Control hub for stability and release, all fit for IND through commercial supply. In addition, MeiraGTx has developed a proprietary manufacturing platform with leading yield and quality aspects and commercial readiness, core capabilities in viral vector design and optimization and a transformative riboswitch gene regulation platform technology that allows for the precise, dose-responsive control of gene expression by oral small molecules. MeiraGTx is focusing the riboswitch platform on the delivery of metabolic peptides, including GLP-1, GIP, Glucagon, and PYY, using oral small molecules, as well as cell therapy for oncology and autoimmune diseases. MeiraGTx has developed the technology to apply genetic medicine to more common diseases, increasing efficacy, addressing novel targets, and expanding access in some of the largest disease areas where the unmet need remains high.
For more information, please visit www.meiragtx.com.
Forward Looking Statement
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding our product candidate development, and anticipated milestones regarding our pre-clinical and clinical data, reporting of such data and the timing of results of data and regulatory matters, as well as statements that include the words “expect,” “will,” “intend,” “plan,” “believe,” “project,” “forecast,” “estimate,” “may,” “could,” “should,” “would,” “continue,” “anticipate” and similar statements of a future or forward-looking nature. These forward-looking statements are based on management’s current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, our incurrence of significant losses; any inability to achieve or maintain profitability, raise additional capital, repay our debt obligations, identify additional and develop existing product candidates, successfully execute strategic transactions or priorities, bring product candidates to market, expansion of our manufacturing facilities and processes, successfully enroll patients in and complete clinical trials, accurately predict growth assumptions, recognize benefits of any orphan drug or rare pediatric disease designations, retain key personnel or attract qualified employees, or incur expected levels of operating expenses; the impact of pandemics, epidemics or outbreaks of infectious diseases on the status, enrollment, timing and results of our clinical trials and on our business, results of operations and financial condition; failure of early data to predict eventual outcomes; failure to obtain FDA or other regulatory approval for product candidates within expected time frames or at all; the novel nature and impact of negative public opinion of gene therapy; failure to comply with ongoing regulatory obligations; contamination or shortage of raw materials or other manufacturing issues; changes in healthcare laws; risks associated with our international operations; significant competition in the pharmaceutical and biotechnology industries; dependence on third parties; risks related to intellectual property; changes in tax policy or treatment; our ability to utilize our loss and tax credit carryforwards; litigation risks; and the other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, as such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SEC’s website at www.sec.gov. These and other important factors could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management’s estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, unless required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Thus, one should not assume that our silence over time means that actual events are bearing out as expressed or implied in such forward-looking statements. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release.
Contacts
Investors:
MeiraGTx
Investors@meiragtx.com
or
Media:
Jason Braco, Ph.D.
LifeSci Communications
jbraco@lifescicomms.com
| MEIRAGTX HOLDINGS PLC AND SUBSIDIARIES | ||||||||||||||||
| CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS | ||||||||||||||||
| (unaudited) | ||||||||||||||||
| (in thousands, except share and per share amounts) | ||||||||||||||||
| FortheThree-Month Periods Ended September 30, | FortheNine-Month Periods Ended September 30, | |||||||||||||||
| 2024 | 2023 | 2024 | 2023 | |||||||||||||
| Revenues: | ||||||||||||||||
| Service revenue – related party | $ | 10,910 | $ | — | $ | 11,889 | $ | — | ||||||||
| License revenue – related party | — | 5,103 | — | 11,977 | ||||||||||||
| Total revenue | 10,910 | 5,103 | 11,889 | 11,977 | ||||||||||||
| Operating expenses: | ||||||||||||||||
| Cost of service revenue – related party | 11,985 | — | 11,985 | — | ||||||||||||
| General and administrative | 12,723 | 10,009 | 37,127 | 35,169 | ||||||||||||
| Research and development | 26,243 | 27,856 | 95,499 | 70,115 | ||||||||||||
| Total operating expenses | 50,951 | 37,865 | 144,611 | 105,284 | ||||||||||||
| Loss from operations | (40,041 | ) | (32,762 | ) | (132,722 | ) | (93,307 | ) | ||||||||
| Other non-operating income (expense): | ||||||||||||||||
| Foreign currency gain (loss) | 3,463 | (8,677 | ) | 2,644 | (2,915 | ) | ||||||||||
| Interest income | 1,189 | 523 | 3,113 | 1,723 | ||||||||||||
| Interest expense | (3,357 | ) | (3,381 | ) | (9,861 | ) | (9,796 | ) | ||||||||
| (Loss) gain on sale of nonfinancial assets | (584 | ) | — | 28,434 | — | |||||||||||
| Fair value adjustment | — | — | — | 53 | ||||||||||||
| Net loss | (39,330 | ) | (44,297 | ) | (108,392 | ) | (104,242 | ) | ||||||||
| Other comprehensive loss: | ||||||||||||||||
| Foreign currency translation (loss) gain | (1,234 | ) | 6,007 | (3,413 | ) | 1,113 | ||||||||||
| Comprehensive loss | $ | (40,564 | ) | $ | (38,290 | ) | $ | (111,805 | ) | $ | (103,129 | ) | ||||
| Net loss | $ | (39,330 | ) | $ | (44,297 | ) | $ | (108,392 | ) | $ | (104,242 | ) | ||||
| Basic and diluted net loss per ordinary share | $ | (0.55 | ) | $ | (0.74 | ) | $ | (1.62 | ) | $ | (1.91 | ) | ||||
| Weighted-average number of ordinary shares outstanding | 71,633,150 | 59,526,642 | 66,709,847 | 54,544,660 | ||||||||||||
| MEIRAGTX HOLDINGS PLC AND SUBSIDIARIES | ||||||||
| CONDENSED CONSOLIDATED BALANCE SHEETS | ||||||||
| (unaudited) | ||||||||
| (in thousands, except share and per share amounts) | ||||||||
| September30, | December31, | |||||||
| 2024 | 2023 | |||||||
| ASSETS | ||||||||
| CURRENT ASSETS: | ||||||||
| Cash and cash equivalents | $ | 122,873 | $ | 129,566 | ||||
| Accounts receivable – related party | 3,279 | 10,138 | ||||||
| Prepaid expenses | 7,029 | 5,625 | ||||||
| Tax incentive receivable | 5,152 | 13,277 | ||||||
| Other current assets | 713 | 1,016 | ||||||
| Total Current Assets | 139,046 | 159,622 | ||||||
| Property, plant and equipment, net | 112,541 | 115,896 | ||||||
| Intangible assets, net | 951 | 1,118 | ||||||
| Restricted cash | 2,156 | 1,083 | ||||||
| Other assets | 1,139 | 1,917 | ||||||
| Equity method and other investments | 6,766 | 6,766 | ||||||
| Right-of-use assets – operating leases, net | 12,782 | 15,910 | ||||||
| Right-of-use assets – finance leases, net | 24,107 | 24,432 | ||||||
| TOTAL ASSETS | $ | 299,488 | $ | 326,744 | ||||
| LIABILITIES AND SHAREHOLDERS' EQUITY | ||||||||
| CURRENT LIABILITIES: | ||||||||
| Accounts payable | $ | 29,504 | $ | 16,042 | ||||
| Accrued expenses | 19,341 | 42,639 | ||||||
| Lease obligations, current | 4,183 | 4,193 | ||||||
| Deferred revenue – related party, current | 5,107 | 2,926 | ||||||
| Other current liabilities | 1,283 | 1,278 | ||||||
| Total Current Liabilities | 59,418 | 67,078 | ||||||
| Deferred revenue – related party | 58,902 | 34,017 | ||||||
| Lease obligations | 9,610 | 12,952 | ||||||
| Asset retirement obligations | 2,880 | 2,401 | ||||||
| Note payable, net | 72,942 | 72,119 | ||||||
| TOTAL LIABILITIES | 203,752 | 188,567 | ||||||
| COMMITMENTS AND CONTINGENCIES (Note 11) | ||||||||
| SHAREHOLDERS' EQUITY: | ||||||||
| Ordinary Shares, | 3 | 2 | ||||||
| Capital in excess of par value | 763,204 | 693,841 | ||||||
| Accumulated other comprehensive loss | (4,848 | ) | (1,435 | ) | ||||
| Accumulated deficit | (662,623 | ) | (554,231 | ) | ||||
| Total Shareholders' Equity | 95,736 | 138,177 | ||||||
| TOTAL LIABILITIES AND SHAREHOLDERS' EQUITY | $ | 299,488 | $ | 326,744 | ||||
FAQ
What were MeiraGTx (MGTX) Q3 2024 financial results?
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What were the results of MeiraGTx (MGTX) AAV-GAD Parkinson's disease study?
