uniQure Announces 2024 Financial Results and Highlights Recent Company Progress
uniQure (NASDAQ: QURE) reported its 2024 financial results and company progress. Key highlights include FDA alignment on Accelerated Approval pathway for AMT-130 in Huntington's disease and completion of patient enrollment in its third cohort Phase I/II study. The company initiated dosing in Phase I/II studies for multiple programs including AMT-260 for epilepsy, AMT-191 for Fabry disease, and AMT-162 for SOD1-ALS.
Financial position shows cash and equivalents of $367.5 million as of December 31, 2024, plus $80.7 million from recent financing, expected to fund operations into second half of 2027. Revenue increased to $27.1 million in 2024 from $15.8 million in 2023. The company reported a net loss of $239.6 million ($4.92 per share) compared to $308.5 million ($6.47 per share) in 2023.
uniQure (NASDAQ: QURE) ha riportato i risultati finanziari e i progressi aziendali per il 2024. I punti salienti includono l'allineamento con la FDA sul percorso di approvazione accelerata per AMT-130 nella malattia di Huntington e il completamento dell'arruolamento dei pazienti nel suo terzo studio di fase I/II. L'azienda ha avviato la somministrazione negli studi di fase I/II per diversi programmi, tra cui AMT-260 per l'epilessia, AMT-191 per la malattia di Fabry e AMT-162 per SOD1-ALS.
La posizione finanziaria mostra cassa e equivalenti di 367,5 milioni di dollari al 31 dicembre 2024, oltre a 80,7 milioni di dollari da recenti finanziamenti, previsti per finanziare le operazioni fino alla seconda metà del 2027. I ricavi sono aumentati a 27,1 milioni di dollari nel 2024 rispetto ai 15,8 milioni di dollari nel 2023. L'azienda ha riportato una perdita netta di 239,6 milioni di dollari (4,92 dollari per azione) rispetto ai 308,5 milioni di dollari (6,47 dollari per azione) del 2023.
uniQure (NASDAQ: QURE) informó sobre sus resultados financieros y avances de la empresa para 2024. Los puntos clave incluyen la alineación con la FDA sobre la vía de Aprobación Acelerada para AMT-130 en la enfermedad de Huntington y la finalización de la inscripción de pacientes en su tercer estudio de fase I/II. La empresa inició la dosificación en estudios de fase I/II para múltiples programas, incluidos AMT-260 para la epilepsia, AMT-191 para la enfermedad de Fabry y AMT-162 para SOD1-ELA.
La posición financiera muestra efectivo y equivalentes de 367,5 millones de dólares al 31 de diciembre de 2024, más 80,7 millones de dólares de financiamiento reciente, que se espera financien las operaciones hasta la segunda mitad de 2027. Los ingresos aumentaron a 27,1 millones de dólares en 2024 desde 15,8 millones de dólares en 2023. La empresa reportó una pérdida neta de 239,6 millones de dólares (4,92 dólares por acción) en comparación con 308,5 millones de dólares (6,47 dólares por acción) en 2023.
uniQure (NASDAQ: QURE)는 2024년 재무 결과와 회사 진행 상황을 보고했습니다. 주요 내용으로는 헌팅턴병 치료를 위한 AMT-130의 가속 승인 경로에 대한 FDA의 일치와 제3코호트 1상/2상 연구에서 환자 등록 완료가 포함됩니다. 회사는 AMT-260(간질 치료), AMT-191(파브리병 치료), AMT-162(SOD1-ALS 치료) 등 여러 프로그램에 대한 1상/2상 연구에서 투약을 시작했습니다.
재무 상태는 2024년 12월 31일 기준으로 3억 6천7백50만 달러의 현금 및 현금성 자산을 보여주며, 최근 자금 조달로 8천7백만 달러가 추가되어 2027년 하반기까지 운영 자금을 지원할 것으로 예상됩니다. 수익은 2023년 1천5백80만 달러에서 2024년 2천7백10만 달러로 증가했습니다. 회사는 2023년의 3억 8천5백만 달러(주당 6.47달러)와 비교하여 2억 3천9백60만 달러의 순손실을 보고했습니다(주당 4.92달러).
uniQure (NASDAQ: QURE) a rapporté ses résultats financiers et les progrès de l'entreprise pour 2024. Les points clés incluent l'alignement avec la FDA sur la voie d'approbation accélérée pour AMT-130 dans la maladie de Huntington et l'achèvement de l'inscription des patients dans sa troisième cohorte d'étude de phase I/II. L'entreprise a commencé à administrer des traitements dans les études de phase I/II pour plusieurs programmes, y compris AMT-260 pour l'épilepsie, AMT-191 pour la maladie de Fabry et AMT-162 pour SOD1-ALS.
La situation financière montre une trésorerie et des équivalents de 367,5 millions de dollars au 31 décembre 2024, plus 80,7 millions de dollars provenant d'un financement récent, qui devrait financer les opérations jusqu'à la seconde moitié de 2027. Les revenus ont augmenté à 27,1 millions de dollars en 2024 contre 15,8 millions de dollars en 2023. L'entreprise a enregistré une perte nette de 239,6 millions de dollars (4,92 dollars par action) par rapport à 308,5 millions de dollars (6,47 dollars par action) en 2023.
uniQure (NASDAQ: QURE) hat seine finanziellen Ergebnisse und den Fortschritt des Unternehmens für 2024 berichtet. Zu den wichtigsten Punkten gehören die Abstimmung mit der FDA über den beschleunigten Genehmigungsweg für AMT-130 bei der Huntington-Krankheit und der Abschluss der Patientenrekrutierung in seiner dritten Kohorte der Phase I/II-Studie. Das Unternehmen hat die Dosierung in Phase I/II-Studien für mehrere Programme, einschließlich AMT-260 für Epilepsie, AMT-191 für die Fabry-Krankheit und AMT-162 für SOD1-ALS, eingeleitet.
Die finanzielle Lage zeigt Liquiden Mittel und Äquivalente in Höhe von 367,5 Millionen US-Dollar zum 31. Dezember 2024, zusätzlich zu 80,7 Millionen US-Dollar aus einer kürzlichen Finanzierung, die voraussichtlich die Betriebskosten bis zur zweiten Hälfte des Jahres 2027 decken wird. Der Umsatz stieg auf 27,1 Millionen US-Dollar im Jahr 2024, verglichen mit 15,8 Millionen US-Dollar im Jahr 2023. Das Unternehmen berichtete von einem Nettoverlust von 239,6 Millionen US-Dollar (4,92 US-Dollar pro Aktie) im Vergleich zu 308,5 Millionen US-Dollar (6,47 US-Dollar pro Aktie) im Jahr 2023.
- FDA alignment on Accelerated Approval pathway for AMT-130, eliminating need for additional pre-submission study
- Revenue increased 71% to $27.1M in 2024
- Reduced annual cash burn by $70M through restructuring
- Strong cash position of $448M to fund operations through H2 2027
- Multiple clinical trial progressions across pipeline
- Net loss of $239.6M in 2024
- Workforce reduction of 65%
- Sale of Lexington manufacturing facility
- Increased non-operating expenses by $29.1M
Insights
uniQure's 2024 results reveal a strategic transformation toward becoming a commercial-stage company, with its lead candidate AMT-130 for Huntington's disease advancing through a potentially accelerated regulatory pathway. The FDA agreement that data from ongoing Phase I/II studies could serve as the primary basis for a BLA submission represents a critical milestone that could significantly reduce time-to-market for what would be the first disease-modifying treatment for this devastating neurological condition.
The company has executed a dramatic operational restructuring, divesting its Lexington manufacturing facility and reducing its workforce by
Financial results show progress toward efficiency with net losses improving to
The pipeline continues to advance with multiple clinical milestones anticipated in 2025-2026. Beyond AMT-130, the company is progressing AMT-191 for Fabry disease (data expected H2 2025), AMT-162 for SOD1-ALS, and AMT-260 for refractory epilepsy (both with data expected H1 2026). The FDA's protocol amendment expanding inclusion criteria for the AMT-260 trial should accelerate enrollment.
A concerning element in the financials is the
uniQure has successfully transformed from a broad R&D organization to a focused late-stage company prioritizing its lead asset while maintaining a balanced pipeline. The next 12-18 months will be pivotal as regulatory interactions continue and potential commercialization approaches.
uniQure's progress with AMT-130 represents a potential breakthrough in Huntington's disease treatment, where no disease-modifying therapies currently exist. The FDA's agreement on an Accelerated Approval pathway using ongoing Phase I/II studies versus natural history controls is particularly significant for this devastating neurodegenerative condition. AMT-130 employs a one-time administered AAV5 vector delivering an engineered microRNA that silences the mutant huntingtin protein expression – addressing the fundamental cause of the disease rather than just managing symptoms.
The FDA's acceptance of the composite Unified Huntington's Disease Rating Scale (cUHDRS) as an intermediate endpoint, supported by neurofilament light chain (NfL) biomarker data, is scientifically meaningful. NfL is a well-established biomarker of neuronal damage, and its reduction would suggest neuroprotective effects. This regulatory flexibility acknowledges the challenges of demonstrating clinical efficacy in slowly progressive neurodegenerative diseases within traditional trial timeframes.
The optimization of immunosuppression in the third cohort addresses a critical challenge for AAV-based gene therapies – managing immune responses that can limit transgene expression and potentially compromise safety. This refinement could significantly improve the therapeutic window of AMT-130.
Beyond Huntington's, uniQure's pipeline demonstrates strategic diversification:
- AMT-260 for mesial temporal lobe epilepsy targets the hippocampus with a microRNA approach to reduce focal excitability. The protocol amendment to include non-lesional patients expands the addressable population while maintaining focus on the neuroanatomically defined seizure origin.
- AMT-191 for Fabry disease delivers functional GLA enzyme via an AAV vector to liver cells, potentially offering a one-time treatment alternative to biweekly enzyme replacement therapy.
- AMT-162 for SOD1-ALS employs a miQURE™ silencing technology targeting the mutant SOD1 gene, a well-validated genetic driver in approximately
2% of ALS cases.
The company's strategic restructuring, while extending the cash runway, raises questions about manufacturing capability following the Lexington facility divestiture. However, the partnership with Genezen includes favorable terms for HEMGENIX® manufacturing, suggesting a thoughtful transition strategy for future commercial production needs.
With multiple data readouts expected through 2026 and potential AMT-130 BLA submission approaching, uniQure has transformed from a broad research organization to a focused late-stage company with a clear path to potential commercialization for its lead asset in an area of significant unmet need.
~ Announced alignment with the U.S. Food and Drug Administration (FDA) on key elements of the Accelerated Approval pathway for AMT-130 in Huntington’s disease; Initiated preparations for a potential Biologics License Application (BLA) submission ~
~ Completed patient enrollment in the third cohort of the Phase I/II study of AMT-130 ~
~ Initiated dosing of the Phase I/II study of AMT-260 in mesial temporal lobe epilepsy (mTLE);
Implementing protocol changes to expand study inclusion criteria ~
~ Completed enrollment of the first cohorts in the Phase I/II studies of AMT-191 in Fabry disease and AMT-162 in SOD1-ALS; Received favorable recommendations from the respective Independent Data Monitoring Committees (IDMC) to proceed with dosing the second cohorts ~
~ Cash and cash equivalents of approximately
LEXINGTON, Mass. and AMSTERDAM, Feb. 27, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the fourth quarter and full year of 2024 and highlighted recent progress across its business.
“This past year was transformative for uniQure, marked by significant clinical and operational progress,” stated Matt Kapusta, chief executive officer of uniQure. “On the clinical front, we made great strides advancing AMT-130 for Huntington’s disease, including securing alignment with the FDA on key elements of the Accelerated Approval pathway, a major milestone that brings us closer to delivering the first potentially disease-modifying treatment for this devastating condition. With this regulatory clarity, we have initiated BLA-readiness activities and look forward to further engagement with the FDA throughout the first half of 2025. Our RMAT designation has enabled a productive and expedited dialogue with the FDA, which we will continue leveraging to advance AMT-130 through the regulatory process as rapidly as possible. This is welcome news for patients awaiting further development of our groundbreaking therapies.”
Mr. Kapusta continued, “Beyond AMT-130, we continue to advance our broader pipeline of investigational gene therapies, with patient enrollment progressing in the Phase I/II studies of AMT-191 for Fabry disease and AMT-162 for SOD1-ALS. Additionally, we are implementing FDA-approved protocol changes to the Phase I/II study of AMT-260 in mTLE, including broadening the inclusion criteria for certain patients in the first cohort – a step we anticipate will help accelerate trial enrollment.”
“Operationally, we took decisive steps in 2024 to streamline our organization, including the sale of our Lexington manufacturing facility and a company-wide restructuring that significantly reduced our cash burn and strengthened our financial position,” Mr. Kapusta continued. “These strategic actions enable us to prioritize investments and achieve multiple value-creating milestones, including the potential approval and commercial launch of AMT-130.”
Recent Company Developments and Updates
- Pursuing Accelerated Approval of AMT-130 for the treatment of Huntington’s disease
- In May 2024, the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation for AMT-130, stating that preliminary clinical evidence indicates that AMT-130 has the potential to address unmet medical needs for treatment of Huntington’s disease.
- In December 2024, uniQure reached agreement with the FDA on key elements of an Accelerated Approval pathway for AMT-130 in Huntington’s disease. As part of the RMAT Type B meeting, the FDA agreed that data from the ongoing Phase I/II studies, compared to a natural history external control, may serve as the primary basis for a BLA submission, eliminating the need for an additional pre-submission study. The FDA also agreed that the composite Unified Huntington’s Disease Rating Scale (cUHDRS) may be used as an intermediate clinical endpoint and reductions in neurofilament light chain (NfL) in the cerebrospinal fluid (CSF) may serve as supportive evidence of therapeutic benefit.
- The Company has scheduled a Type B meeting with the FDA in the first quarter of 2025 to discuss chemistry, manufacturing and control (CMC) requirements to support its planned BLA submission. A separate Type B meeting to discuss the pivotal statistical analysis plan for the BLA is anticipated to take place in the second quarter of 2025. After the completion of these interactions, the Company expects to provide a regulatory update in the second quarter of 2025, including the expected timing of a potential BLA submission.
- In February 2025, the Company completed enrollment of all 12 patients in the third cohort investigating an optimized immunosuppression regimen. The Company expects to provide an initial safety update on the third cohort in the second quarter of 2025.
- In the third quarter of 2025, the Company expects to present data from its ongoing Phase I/II studies of AMT-130 in support of a potential BLA submission. The update will include follow-up data on all patients treated with AMT-130 in the first two cohorts, including three years of follow-up on 24 treated patients.
- In May 2024, the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation for AMT-130, stating that preliminary clinical evidence indicates that AMT-130 has the potential to address unmet medical needs for treatment of Huntington’s disease.
- Advancing additional clinical programs to proof-of-concept
- AMT-260 for the treatment of refractory mesial temporal lobe epilepsy (mTLE) – In November 2024, the Company announced the first patient dosed in the Phase I/II clinical trial of AMT-260 for the treatment of mTLE. The FDA recently approved a protocol amendment expanding the inclusion criteria for certain patients in the first cohort to include patients with non-lesional mesial temporal lobe epilepsy in the non-dominant hemisphere. This broader inclusion criteria may assist in accelerated enrollment. The Company expects to present initial data from the study in the first half of 2026.
- AMT-162 for the treatment of SOD1 amyotrophic lateral sclerosis (ALS) – In January 2025, the Company announced a favorable recommendation from the IDMC based on the review of 28-day safety data from the first study cohort in the Phase I/II EPISODE1 study. The Company initiated enrollment in the second dose cohort in the first quarter of 2025 and expects to present initial data from the study in the first half of 2026.
- AMT-191 for the treatment of Fabry disease – In February 2025, the Company announced completion of enrollment in the first cohort in the Phase I/IIa clinical trial of AMT-191 and a favorable recommendation from the IDMC having reviewed safety data from the initial two patients. The Company expects to initiate enrollment in the second dose cohort in the second quarter of 2025 and to present initial data from the study in the second half of 2025.
- AMT-260 for the treatment of refractory mesial temporal lobe epilepsy (mTLE) – In November 2024, the Company announced the first patient dosed in the Phase I/II clinical trial of AMT-260 for the treatment of mTLE. The FDA recently approved a protocol amendment expanding the inclusion criteria for certain patients in the first cohort to include patients with non-lesional mesial temporal lobe epilepsy in the non-dominant hemisphere. This broader inclusion criteria may assist in accelerated enrollment. The Company expects to present initial data from the study in the first half of 2026.
- Strong financial position
- In the first quarter of 2025, the Company completed a public offering of 5.1 million ordinary shares, including the full exercise of the underwriters’ overallotment option, at a price of
$17.00 per share. Net proceeds from the offering are expected to fund operations into the second half of 2027, including the potential BLA submission and U.S. commercial launch of AMT-130. - In the second quarter of 2024, the Company completed the sale of its Lexington, MA manufacturing facility to Genezen and retired
$50 million of its outstanding debt with Hercules Capital. - In the third quarter of 2024, the Company announced an organization restructuring which, combined with the Lexington facility sale, eliminated approximately
65% of the global workforce and reduced recurring cash burn by approximately$70 million per year.
- In the first quarter of 2025, the Company completed a public offering of 5.1 million ordinary shares, including the full exercise of the underwriters’ overallotment option, at a price of
Upcoming Investor Events
- TD Cowen 45th Annual Healthcare Conference, March 3rd – Boston, MA
- Leerink’s Global Healthcare Conference 2025, March 10th – Miami, FL
- Kempen Life Sciences Conference, April 3rd – Amsterdam, NL
Financial Highlights
Cash position: As of December 31, 2024, the Company held cash, cash equivalents and investment securities of
Revenues: Revenue for the year ended December 31, 2024 was
Cost of contract manufacturing revenues: Cost of contract manufacturing revenues were
R&D expenses: Research and development expenses were
SG&A expenses: Selling, general and administrative expenses were
Other income: Other income was
Other expense: Other expense was
Other non-operating items, net: Other non-operating items, net was an expense of
Net loss: The net loss for the year ended December 31, 2024, was
About uniQure
uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. The approvals of uniQure’s gene therapy for hemophilia B – an historic achievement based on more than a decade of research and clinical development – represent a major milestone in the field of genomic medicine and ushers in a new treatment approach for patients living with hemophilia. uniQure is now advancing a pipeline of proprietary gene therapies for the treatment of patients with Huntington's disease, refractory temporal lobe epilepsy, ALS, Fabry disease, and other severe diseases. www.uniQure.com
uniQure Forward-Looking Statements
This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," “establish,” "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," “seek,” "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Examples of these forward-looking statements include, but are not limited to, statements concerning the Company’s cash runway and its ability to fund its operations into the second half of 2027 and the planned use of proceeds from its first quarter 2025 public offering; the Company’s plans for further interactions with the FDA to discuss the requirements for its planned BLA submission for AMT-130; the Company’s ability to utilize an accelerated pathway to progress AMT-130 through regulatory approval; the Company’s plans to announce additional interim data and regulatory updates from its ongoing Phase I/II clinical studies of AMT-130, along with an initial safety update on the third cohort of the AMT-130 study and other program updates; the effectiveness of planned protocol changes in accelerating enrollment in the AMT-260 study; and the Company’s organizational restructuring and other actions designed to increase shareholder value and fund its pipeline of gene therapy candidates. The Company’s actual results could differ materially from those anticipated in these forward-looking statements for many reasons. These risks and uncertainties include, among others: risks associated with the clinical results and the development and timing of the Company’s programs; the Company’s interactions with regulatory authorities, which may affect the initiation, timing and progress of clinical trials and pathways to regulatory approval; the Company’s ability to continue to build and maintain the company infrastructure and personnel needed to achieve its goals; the Company’s effectiveness in managing current and future clinical trials and regulatory processes; the continued development and acceptance of gene therapies; the Company’s ability to demonstrate the therapeutic benefits of its gene therapy candidates in clinical trials; the Company’s ability to obtain, maintain and protect intellectual property; and the Company’s ability to fund its operations and to raise additional capital as needed. These risks and uncertainties are more fully described under the heading "Risk Factors" in the Company’s periodic filings with the U.S. Securities & Exchange Commission (“SEC”), including its Annual Report on Form 10-K to be filed February 27, 2025 and in other filings that the Company makes with the SEC from time to time. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and the Company assumes no obligation to update these forward-looking statements, even if new information becomes available in the future.
uniQure Contacts:
| FOR INVESTORS: | FOR MEDIA: |
| Chiara Russo | Tom Malone |
| Direct: 617-306-9137 | Direct: 339-970-7558 |
| Mobile: 617-306-9137 | Mobile:339-223-8541 |
| c.russo@uniQure.com | t.malone@uniQure.com |
| uniQure N.V. | |||||||
| UNAUDITED CONSOLIDATED BALANCE SHEETS | |||||||
| December 31, | December 31, | ||||||
| 2024 | 2023 | ||||||
| (in thousands, except share and per share amounts) | |||||||
| Current assets | |||||||
| Cash and cash equivalents | $ | 158.930 | $ | 241.360 | |||
| Current investment securities | 208.591 | 376.532 | |||||
| Accounts receivable | 5.881 | 4.193 | |||||
| Inventories, net | — | 12.024 | |||||
| Prepaid expenses | 9.281 | 15.089 | |||||
| Other current assets and receivables | 7.606 | 2.655 | |||||
| Total current assets | 390.289 | 651.853 | |||||
| Non-current assets | |||||||
| Property, plant and equipment, net | $ | 20.424 | $ | 46.548 | |||
| Other investments | 27.464 | $ | 2.179 | ||||
| Operating lease right-of-use assets | 13.647 | 28.789 | |||||
| Intangible assets, net | 71.043 | 60.481 | |||||
| Goodwill | 22.414 | 26.379 | |||||
| Deferred tax assets, net | 9.856 | 12.276 | |||||
| Other non-current assets | 1.399 | 3.184 | |||||
| Total non-current assets | 166.247 | 179.836 | |||||
| Total assets | $ | 556.536 | $ | 831.689 | |||
| Current liabilities | |||||||
| Accounts payable | $ | 7.227 | $ | 6.586 | |||
| Accrued expenses and other current liabilities | 29.225 | 30.534 | |||||
| Current portion of contingent consideration | — | 28.211 | |||||
| Current portion of operating lease liabilities | 3.601 | 8.344 | |||||
| Total current liabilities | 40.053 | 73.675 | |||||
| Non-current liabilities | |||||||
| Long-term debt | 51.324 | 101.749 | |||||
| Liability from royalty financing agreement | 434.930 | 394.241 | |||||
| Operating lease liabilities, net of current portion | 11.136 | 28.316 | |||||
| Contingent consideration, net of current portion | 10.860 | 14.795 | |||||
| Deferred tax liability, net | 7.043 | 7.543 | |||||
| Other non-current liabilities | 7.942 | 3.700 | |||||
| Total non-current liabilities | 523.235 | 550.344 | |||||
| Total liabilities | 563.288 | 624.019 | |||||
| Shareholders' equity | |||||||
| Total shareholders' equity | (6.752 | ) | 207.670 | ||||
| Total liabilities and shareholders' equity | $ | 556.536 | $ | 831.689 | |||
| uniQure N.V. | ||||||||||||
| UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS | ||||||||||||
| Year ended December 31, | ||||||||||||
| 2024 | 2023 | 2022 | ||||||||||
| (in thousands, except share and per share amounts) | ||||||||||||
| License revenues | 10.133 | $ | 2.758 | $ | 100.000 | |||||||
| Contract manufacturing revenues | 6.114 | 10.835 | 1.717 | |||||||||
| Collaboration revenues | 10.872 | 2.250 | 4.766 | |||||||||
| Total revenues | 27.119 | 15.843 | 106.483 | |||||||||
| Operating expenses: | ||||||||||||
| Cost of license revenues | (1.267 | ) | (65 | ) | (1.254 | ) | ||||||
| Cost of contract manufacturing revenues | (17.060 | ) | (13.563 | ) | (2.089 | ) | ||||||
| Research and development expenses | (143.782 | ) | (214.864 | ) | (197.591 | ) | ||||||
| Selling, general and administrative expenses | (52.657 | ) | (74.591 | ) | (55.059 | ) | ||||||
| Total operating expenses | (214.766 | ) | (303.083 | ) | (255.993 | ) | ||||||
| Other income | 7.926 | 6.059 | 7.171 | |||||||||
| Other expense | (4.573 | ) | (1.690 | ) | (820 | ) | ||||||
| Loss from operations | (184.294 | ) | (282.871 | ) | (143.159 | ) | ||||||
| Non-operating items, net | (52.833 | ) | (23.686 | ) | 14.900 | |||||||
| Loss before income tax (expense) / benefit | $ | (237.127 | ) | $ | (306.557 | ) | $ | (128.259 | ) | |||
| Income tax (expense) / benefit | (2.429 | ) | (1.921 | ) | 1.470 | |||||||
| Net loss | $ | (239.556 | ) | $ | (308.478 | ) | $ | (126.789 | ) | |||
| Earnings per ordinary share - basic and diluted | ||||||||||||
| Basic and diluted net loss per ordinary share | $ | (4,92 | ) | $ | (6,47 | ) | $ | (2,71 | ) | |||
| Weighted average shares - basic and diluted | 48.649.129 | 47.670.986 | 46.735.045 | |||||||||
FAQ
What is the status of uniQure's AMT-130 Huntington's disease treatment approval process?
How much cash does QURE have and how long will it last?
What were uniQure's key financial metrics for 2024?
What clinical trial milestones did QURE achieve in 2024-2025?
