uniQure Announces 2025 Financial Results and Provides Recent Company Updates
Rhea-AI Summary
uniQure (NASDAQ: QURE) reported 2025 results and program updates on March 2, 2026. Key highlights include $622.5 million in cash and investments as of Dec 31, 2025, expected to fund operations into H2 2029, and $16.1 million revenue for 2025 with a $199.0 million net loss.
Clinical updates: AMT-130 showed a 75% slowing on cUHDRS at 36 months but FDA recommended a prospective randomized, sham-controlled study; AMT-260 completed first cohort enrollment; AMT-191 showed durable, dose-dependent α-Gal A increases with dosing pauses for asymptomatic Grade 3 liver enzyme elevations; AMT-162 remains on voluntary pause.
Positive
- Cash position of $622.5 million as of Dec 31, 2025
- Proceeds of $404.2 million raised via 2025 public offerings
- AMT-130: 75% slowing in cUHDRS at 36 months (high-dose, p=0.003)
- AMT-260 completed enrollment in first Phase I/IIa dose cohort
Negative
- FDA said Phase I/II external-control data are insufficient to support marketing application
- Net loss of $199.0 million for 2025 (EPS loss $3.46)
- Revenue declined to $16.1 million in 2025 from $27.1 million in 2024
- AMT-191 dosing paused in some cohorts due to Grade 3 liver enzyme elevations; AMT-162 enrollment remains on pause
Key Figures
Market Reality Check
Peers on Argus
QURE fell 8.68% on heavy volume while closely scored peers showed smaller, mixed moves: EYPT -0.9%, TSHA +1.12%, ABUS -0.21%, OCS -1.21%, UPB -2.35%. This points to a stock-specific reaction to the earnings and regulatory update rather than a broad gene therapy selloff.
Previous Earnings Reports
| Date | Event | Sentiment | Move | Catalyst |
|---|---|---|---|---|
| Nov 10 | Q3 2025 earnings | Neutral | -5.9% | Q3 2025 results plus AMT-130 data and emerging FDA uncertainty. |
| Jul 29 | Q2 2025 earnings | Positive | -8.7% | Q2 2025 results, FDA alignment for AMT-130 and pipeline progress. |
| May 09 | Q1 2025 earnings | Positive | +4.4% | Q1 2025 results with AMT-130 BTD and strengthened cash balance. |
| Feb 27 | 2024 annual results | Positive | +3.4% | 2024 results, accelerated approval alignment and expanded early-stage pipeline. |
| Nov 05 | Q3 2024 earnings | Positive | +9.0% | Q3 2024 results, strong cash and positive AMT-130 interim data. |
Earnings updates have typically produced modest moves, with mostly positive clinical/financial narratives but occasional sharp downside when regulatory uncertainty around AMT-130 increases.
Over the past five earnings and annual results releases since Nov 2024, uniQure has consistently highlighted AMT-130 progress alongside a strengthening cash position. Cash rose from $367.5M at Dec 31, 2024 to higher levels through 2025 financings, while net losses gradually improved. Earlier earnings featured FDA alignment on an accelerated approval pathway, but later updates introduced regulatory uncertainty around using Phase I/II external-control data as primary evidence. Today’s 2025 results and FDA feedback extend that narrative, pairing strong clinical signals with a more complex approval path and a longer cash runway.
Historical Comparison
In the past five earnings-related releases, average stock move was about 0.45%. Today’s -8.68% reaction to 2025 results and the AMT-130 FDA feedback represents a materially larger downside move than prior earnings updates.
Across recent earnings cycles, uniQure has moved from FDA alignment on an accelerated pathway for AMT-130 to later communications highlighting regulatory uncertainty, while simultaneously bolstering its cash balance and advancing AMT-260, AMT-191 and AMT-162 through early clinical milestones.
Market Pulse Summary
This announcement combines robust AMT-130 efficacy data, continued progress in AMT-260 and AMT-191, and a strengthened balance sheet with $622.5M in cash and investments, alongside a $199.0M annual net loss. The key risk is the FDA’s request for a randomized, sham-controlled study rather than accepting Phase I/II external-control data for approval. Investors may focus on how the company manages trial design, spending on R&D and SG&A, and whether the cash runway into the second half of 2029 holds without additional dilution.
Key Terms
type a meeting regulatory
type b meeting regulatory
biologics license application regulatory
enzyme replacement therapy medical
dose-limiting toxicities medical
independent data monitoring committee medical
AI-generated analysis. Not financial advice.
~ Held Type A meeting with FDA to discuss AMT-130 for Huntington’s disease; Company evaluating Phase III development considerations and plans to request follow-up Type B meeting in the second quarter of 2026 ~
~ Completed enrollment of the first cohort in the Phase I/IIa study of AMT-260 in refractory mesial temporal lobe epilepsy, with additional clinical data expected in the first half of 2026 ~
~ Presented updated Phase I/II data from AMT-191 in Fabry disease showing durable, dose-dependent increases in α-Gal A enzyme activity ~
~ Cash, cash equivalents and current investment securities of approximately
~ uniQure to host earnings call at 8:00 a.m. ET ~
LEXINGTON, Mass. and AMSTERDAM, March 02, 2026 (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 2025 and highlighted recent progress across its business.
“In 2025, we presented compelling 36-month clinical data from AMT-130 that we believe meaningfully demonstrate its potential to become a first disease-modifying therapy for people living with Huntington’s disease,” said Matt Kapusta, chief executive officer at uniQure. “While we have not reached alignment with the FDA on an approval pathway, we remain confident in the strength and durability reflected in our dataset. We are committed to engaging with the FDA to define a clear and efficient path to bring this potentially transformative therapy to Huntington’s disease patients in urgent need for treatments.”
“Beyond Huntington’s disease, we have also made meaningful progress across our broader clinical portfolio and look forward to additional data readouts later this year,” continued Mr. Kapusta. “Entering 2026 with a strong balance sheet, we remain financially disciplined and well-positioned to continue advancing our programs strategically and responsibly.”
Recent Company Developments and Updates
Advancing AMT-130 for the treatment of Huntington’s disease
- In September 2025, the Company announced positive topline data from the pivotal Phase I/II study for AMT-130 for the treatment of Huntington’s disease. Topline 36-month efficacy results for 12 patients receiving high-dose AMT-130 included the following (data cutoff as of June 30, 2025):
-
- A statistically significant
75% slowing in disease progression measured by the composite Unified Huntington’s Disease Rating Scale (cUHDRS) was observed which met the primary endpoint compared to a propensity score-matched external control (p=0.003). - A key secondary endpoint of Total Functional Capacity (TFC) demonstrated a statistically significant
60% slowing of disease progression compared to a propensity score-matched external control (p=0.033).
- A statistically significant
- A mean reduction from baseline in cerebrospinal neurofilament light protein (NfL) of -
8.2% was observed at 36 months in the high-dose of AMT-130 of the Phase I/II studies. - AMT-130 was generally well-tolerated across both doses, with a manageable safety profile and no new drug-related serious adverse events observed since December 2022. The most common adverse events in the treatment groups were related to the administration procedure.
-
- In the fourth quarter of 2025, dosing was completed in a fourth cohort of six patients receiving high-dose AMT-130 to evaluate safety and efficacy in patients with lower baseline striatal volumes compared to previous cohorts in the U.S. Phase I/II study.
- On February 23, 2026, the Company presented new analyses at the 21st Annual Huntington’s Disease Therapeutics Conference in Palm Springs, California showing that propensity score methodology using clinical covariates with TRACK-HD/TRACK-ON and PREDICT-HD datasets effectively substitutes for baseline striatal volume in predicting Huntington’s disease progression. These covariates were the same as those used in the 3-year data analysis to match AMT-130-treated patients to the external comparator cohort derived from Enroll-HD.
- The Company held a pre-BLA meeting with the U.S. Food and Drug Administration (FDA) in October 2025 and a Type A meeting in January 2026 to discuss the regulatory path forward. Following receipt of final meeting minutes from the Type A meeting, the Company announced that the FDA stated it cannot agree that data from the Phase I/II studies, compared to an external control, are sufficient to provide the primary evidence of effectiveness required to support a marketing application for AMT-130. The FDA strongly recommended the Company conduct a prospective, randomized, double-blind, sham surgery-controlled study. The Company intends to continue engaging with the FDA regarding Phase III development considerations and plans to request a Type B meeting in the second quarter of 2026 to further discuss potential study design approaches.
Continued clinical progress in pipeline programs
AMT-260 for the treatment of refractory mesial temporal lobe epilepsy (MTLE)
- In May 2025, the Company presented initial safety and exploratory efficacy data from the first treated patient showing a
92% reduction in seizure frequency observed through the first five months of follow up, with no serious safety events. - In 2025, the Company completed enrollment in the first dose cohort of six patients in the Phase I/IIa study. Enrollment has been initiated in a second dose cohort expected to include an additional six patients.
- The Company expects to provide updated data from the Phase I/IIa study in the second quarter of 2026.
AMT-191 for the treatment of Fabry disease
- In February 2026, the Company presented updated safety and exploratory efficacy data from the Phase I/II study of AMT-191 in Fabry disease (data cutoff as of January 8, 2026):
-
- Dose-dependent elevations were observed across 11 patients in three dose levels with α-Gal A activity ranging from 0.34- to 82.2-fold above mean normal range1 at the lowest dose, 1.6- to 312.52-fold at the mid dose, and 27.7- to 223.7-fold at the highest dose.
- These increases were durable across follow-up periods ranging from four months to more than one year.
- Six of 11 dosed patients were withdrawn from enzyme replacement therapy (ERT).
- Plasma lyso-Gb3 levels were stable post-dose across all cohorts, regardless of ERT status
-
- Based on data observed to date, AMT-191 showed a manageable safety profile at all dose levels. No SAEs related to AMT-191 were observed at the 4x1013 gc/kg and 2x1013 gc/kg doses. No additional SAEs were observed at the 6x1013 gc/kg dose beyond the five previously reported in September 2025 in two patients.
- Per protocol, additional dosing in the mid- and high-dose cohorts has been paused pending further evaluation of asymptomatic Grade 3 liver enzyme elevations reported in two patients from the mid-dose cohort, which were confirmed as dose-limiting toxicities.
AMT-162 for the treatment of SOD1 amyotrophic lateral sclerosis (ALS)
- Enrollment remains on voluntary pause following the Independent Data Monitoring Committee recommendation after review of preliminary safety and efficacy data in September 2025, which included one serious adverse event of dose-limiting toxicity determined to be related to AMT-162. The Company continues to collect and evaluate data from the five patients treated in the Phase I/II EPISOD1 study.
Strengthened financial position
- In 2025, the Company received aggregate net proceeds of
$404.2 million , after deducting underwriting discounts and commissions and other offering expenses, through the completion of follow-on public offerings in which a total of 11.8 million ordinary shares were issued, and in lieu of ordinary shares to certain investors, pre-funded warrants to purchase 0.5 million of the Company’s ordinary shares. - In the third quarter of 2025, the Company further announced the refinancing of its existing
$50 million debt outstanding to extend the term to October 2030 and reduce its cost of capital. An additional term loan tranche of$100 million could be drawn down at the Company’s option subject to the achievement of a pre-defined regulatory milestone for AMT-130. A third tranche of$25 million is available subject to the lender’s approval. - As of December 31, 2025, the Company had cash, cash equivalents and current investment securities of
$622.5 million . The Company expects that cash, cash equivalents and investment securities will be sufficient to fund operations into the second half of 2029.
_____________________
1 Normal range (1.38 – 8.66 nmol); mean normal of 3.57 nmol
Financial Highlights
Cash Position: As of December 31, 2025, the Company held
Revenues: Revenue for the year ended December 31, 2025 was
Cost of Contract Manufacturing Revenues: Cost of contract manufacturing revenues was nil for the year ended December 31, 2025, compared to
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
Non-Operating Items, Net: Total non-operating items, net were an expense of
Income Tax Expense: Income tax expense was
Net loss: The net loss for the year ending December 31, 2025, was
Upcoming investor events:
- TD Cowen 46th Annual Health Care Conference, March 2nd – Boston, MA
- Leerink Global Partners Global Healthcare Conference, March 11th – Miami, FL
- Barclays 28th Annual Global Healthcare Conference, March 12th – Miami, FL
- Kempen Life Science Conference, April 15th – Amsterdam, Netherlands
Investor Conference Call and Webcast Information
uniQure management will host an investor conference call and webcast today, Monday, March 2nd at 8:00 a.m. ET. The event will be webcast under the Events & Presentations section of uniQure’s website at https://www.uniqure.com/investors-media/events-presentations, and following the event a replay will be archived for 90 days. Analysts wishing to participate in the question and answer session should access the live call by dialing (646) 307-1963 or toll-free (800) 715-9871 and entering conference ID 4607289. If you are joining the conference call, please join 15 minutes before the start time.
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 2029; AMT-130 as a potentially first disease-modifying therapy for people living with Huntington’s disease; the Company’s plans and timing with respect to future interactions with regulatory authorities and regulatory updates related to AMT-130, including the Company’s plans to continue engaging with the FDA regarding Phase III development considerations and request a Type B meeting with the FDA in the second quarter of 2026; the Company’s ability and plans to strategically advance its programs; the Company’s plans to enroll an additional six patients in a second cohort in the Phase I/IIa study for AMT-260; the Company’s plans for further clinical updates, including plans to announce additional data from the Company’s AMT-260 program in the first half of 2026; the Company’s plans to continue to collect data from patients in the EPISOD1 study; and the Company’s plans to attend upcoming investor events. 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, including the risk that clinical results will be unable to demonstrate data sufficient to support further clinical development or regulatory approval in any country where approval is pursued; the risk that more patient data become available that results in a different interpretation than the one derived from preliminary, interim or topline data; the Company’s interactions with regulatory authorities, including the FDA, which may affect the initiation, timing and progress of clinical trials and pathways and timing for regulatory approval; whether the measurements that the Company is evaluating are viewed as robust and sensitive measurements of disease progression suitable for regulatory approval; the Company’s ability to conduct and fund a Phase III or confirmatory study for AMT-130; the Company’s ability to continue to build and maintain the 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. 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 the Company’s Annual Reports on Form 10-K and Quarterly Reports on Form 10-Q, 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 Direct: 781-491-4371 Mobile: 617-306-9137 c.russo@uniQure.com | Tom Malone Direct: 339-970-7558 Mobile: 339-223-8541 t.malone@uniQure.com |
| uniQure N.V. | ||||||||
| UNAUDITED CONSOLIDATED BALANCE SHEETS | ||||||||
| December 31, | December 31, | |||||||
| 2025 | 2024 | |||||||
| (in thousands) | ||||||||
| Current assets | ||||||||
| Cash and cash equivalents | $ | 80,240 | $ | 158,930 | ||||
| Current investment securities | 542,301 | 208,591 | ||||||
| Accounts receivable | 5,863 | 5,881 | ||||||
| Prepaid expenses | 20,506 | 9,281 | ||||||
| Other current assets and receivables | 7,076 | 7,606 | ||||||
| Total current assets | 655,986 | 390,289 | ||||||
| Non-current assets | ||||||||
| Property, plant and equipment, net | 13,800 | 20,424 | ||||||
| Other investments | 30,237 | 27,464 | ||||||
| Operating lease right-of-use assets | 12,525 | 13,647 | ||||||
| Intangible assets, net | 72,790 | 71,043 | ||||||
| Goodwill | 25,355 | 22,414 | ||||||
| Deferred tax assets, net | 8,654 | 9,856 | ||||||
| Other non-current assets | 5,561 | 1,399 | ||||||
| Total non-current assets | 168,922 | 166,247 | ||||||
| Total assets | $ | 824,908 | $ | 556,536 | ||||
| Current liabilities | ||||||||
| Accounts payable | $ | 5,170 | $ | 7,227 | ||||
| Accrued expenses and other current liabilities | 41,292 | 29,225 | ||||||
| Liability related to pre-funded warrants | 12,595 | — | ||||||
| Current portion of operating lease liabilities | 3,862 | 3,601 | ||||||
| Total current liabilities | 62,919 | 40,053 | ||||||
| Non-current liabilities | ||||||||
| Long-term debt | 49,699 | 51,324 | ||||||
| Liability from royalty financing agreement | 473,199 | 434,930 | ||||||
| Operating lease liabilities, net of current portion | 9,832 | 11,136 | ||||||
| Contingent consideration | 18,736 | 10,860 | ||||||
| Deferred tax liability, net | 7,967 | 7,043 | ||||||
| Other non-current liabilities, net of current portion | 3,655 | 7,942 | ||||||
| Total non-current liabilities | 563,088 | 523,235 | ||||||
| Total liabilities | 626,007 | 563,288 | ||||||
| Shareholders' equity / (deficit) | ||||||||
| Total shareholders' equity / (deficit) | 198,901 | (6,752 | ) | |||||
| Total liabilities and shareholders' equity / (deficit) | $ | 824,908 | $ | 556,536 | ||||
| uniQure N.V. | ||||||||||||
| UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS | ||||||||||||
| Years ended December 31, | ||||||||||||
| 2025 | 2024 | 2023 | ||||||||||
| (in thousands, except share and per share amounts) | ||||||||||||
| License revenues | $ | 15,934 | $ | 10,133 | $ | 2,758 | ||||||
| Contract manufacturing revenues | — | 6,114 | 10,835 | |||||||||
| Collaboration revenues | 164 | 10,872 | 2,250 | |||||||||
| Total revenues | 16,098 | 27,119 | 15,843 | |||||||||
| Operating expenses: | ||||||||||||
| Cost of license revenues | (1,686 | ) | (1,267 | ) | (65 | ) | ||||||
| Cost of contract manufacturing revenues | — | (17,060 | ) | (13,563 | ) | |||||||
| Research and development expenses | (140,673 | ) | (143,782 | ) | (214,864 | ) | ||||||
| Selling, general and administrative expenses | (65,456 | ) | (52,657 | ) | (74,591 | ) | ||||||
| Total operating expenses | (207,815 | ) | (214,766 | ) | (303,083 | ) | ||||||
| Other income | 14,410 | 7,926 | 6,059 | |||||||||
| Other expense | (8,042 | ) | (4,573 | ) | (1,690 | ) | ||||||
| Loss from operations | (185,349 | ) | (184,294 | ) | (282,871 | ) | ||||||
| Non-Operating items, net | (7,991 | ) | (52,833 | ) | (23,686 | ) | ||||||
| Loss before income tax expense | $ | (193,340 | ) | $ | (237,127 | ) | $ | (306,557 | ) | |||
| Income tax expense | (5,631 | ) | (2,429 | ) | (1,921 | ) | ||||||
| Net loss | $ | (198,971 | ) | $ | (239,556 | ) | $ | (308,478 | ) | |||
| Earnings per ordinary share - diluted | ||||||||||||
| Diluted net loss per ordinary share | $ | (3.46 | ) | $ | (4.92 | ) | $ | (6.47 | ) | |||
| Weighted average shares - basic and diluted | 57,502,068 | 48,649,129 | 47,670,986 | |||||||||
FAQ
What did uniQure (QURE) report for cash and runway on March 2, 2026?
What did uniQure announce about AMT-130 regulatory feedback and next steps (QURE)?
What were the key 36-month efficacy results for AMT-130 reported by uniQure (QURE)?
What is the status of AMT-260 clinical development for epilepsy at uniQure (QURE)?
What safety and efficacy signals did uniQure report for AMT-191 in Fabry disease (QURE)?
How did uniQure perform financially in 2025 and what were the main drivers (QURE)?
