uniQure Announces Second Quarter 2025 Financial Results and Highlights of Recent Company Progress
Rhea-AI Summary
uniQure (NASDAQ: QURE) reported Q2 2025 financial results and significant progress across its gene therapy pipeline. The company achieved FDA alignment for AMT-130 in Huntington's disease, supporting a planned BLA submission in Q1 2026. Key highlights include a 92% seizure reduction in the first AMT-260 patient for epilepsy treatment and the appointment of Kylie O'Keefe as Chief Customer and Strategy Officer.
Financial results show $377.0 million in cash and investments as of June 30, 2025, expected to fund operations into H2 2027. Q2 revenues were $5.3 million, down from $11.1 million in Q2 2024. Net loss improved to $37.7 million ($0.69 per share) compared to $56.3 million ($1.16 per share) in the same period last year.
Positive
- FDA granted Breakthrough Therapy designation to AMT-130 for Huntington's disease
- Strong cash position of $377.0 million expected to fund operations into second half of 2027
- 92% seizure reduction observed in first AMT-260 patient with no serious adverse events
- Net loss improved to $37.7 million from $56.3 million year-over-year
- Alignment with FDA on BLA pathway for AMT-130, positioning it as potential first disease-modifying therapy for Huntington's
Negative
- Revenue declined to $5.3 million from $11.1 million year-over-year
- R&D expenses increased to $35.4 million from $33.7 million year-over-year
- Other expense increased to $2.2 million from $0.2 million in prior year
News Market Reaction
On the day this news was published, QURE declined 8.68%, reflecting a notable negative market reaction. Argus tracked a peak move of +13.8% during that session. Argus tracked a trough of -13.6% from its starting point during tracking. Our momentum scanner triggered 62 alerts that day, indicating high trading interest and price volatility. This price movement removed approximately $85M from the company's valuation, bringing the market cap to $893M at that time. Trading volume was elevated at 2.0x the daily average, suggesting increased selling activity.
Data tracked by StockTitan Argus on the day of publication.
~ Achieved alignment with the FDA on the AMT-130 statistical analysis plan and CMC requirements to support a planned BLA submission in the first quarter of 2026;
~ Company expects to present AMT-130 topline three-year data in September 2025 ~
~ Presented case study from first participant treated with AMT-260 for refractory mesial temporal lobe epilepsy showing
~ Appointment of Kylie O’Keefe as Chief Customer and Strategy Officer ~
~ Cash, cash equivalents and current investment securities of approximately
~ uniQure to host earnings call at 8:30 a.m. ET ~
LEXINGTON, Mass. and AMSTERDAM, July 29, 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 second quarter of 2025 and highlighted recent progress across its business.
“We delivered tremendous progress across our business in the first half of 2025, setting the stage for a transformative period ahead for uniQure,” said Matt Kapusta, chief executive officer of uniQure. “With alignment from the FDA on a biological license application (BLA) pathway and pivotal topline data expected in September, AMT-130 is well-positioned to potentially become the first disease-modifying therapy for people living with Huntington’s disease.”
“At the same time, we’re encouraged by early clinical signals from the first patient treated with AMT-260 in refractory mesial temporal lobe epilepsy and the continued advancement of our Fabry and ALS programs,” continued Mr. Kapusta. “Backed by a strong balance sheet and an exceptional team, we believe that we are well prepared to execute on the opportunities ahead, including the planned commercialization of AMT-130, and delivering lasting value to patients and shareholders.”
Recent Company Developments and Updates
- Alignment with FDA continues to support Accelerated Approval pathway for AMT-130
- In June 2025, the Company announced its alignment with the FDA on the statistical analysis plan (SAP) and Chemistry, Manufacturing and Controls (CMC) requirements to support a planned Biologics License Application (BLA) submission for AMT-130 in the first quarter of 2026. The primary efficacy analysis will evaluate the three-year change in the composite Unified Huntington’s Disease Rating Scale (cUHDRS) in patients receiving the high dose of AMT-130 compared to patients in a propensity score-matched external control arm derived from the ENROLL-HD data set. Certain sensitivity analyses, including one using a propensity score-weighted methodology, will also be provided. The FDA also agreed to uniQure’s approach of leveraging prior knowledge and manufacturing experience from the HEMGENIX® process, supplemented with additional full-scale Good Manufacturing Practices (GMP) batches and a single Process Performance Qualification (PPQ) run.
- In July 2025, the Company submitted the final SAP to the FDA. Additionally, the Company initiated its PPQ campaign to support the planned BLA submission.
- In May 2025, the Company announced key safety observations from the third cohort of the Phase I/II study evaluating the impact of prophylactic immunosuppression in 12 patients receiving AMT-130. AMT-130 was generally well-tolerated at both doses with no treatment-related serious adverse events. Three serious adverse events related to immunosuppression were observed and resolved with supportive care. The Company expects a short-term steroid regimen will be incorporated as a prophylactic immunosuppression strategy.
- In April 2025, the FDA granted Breakthrough Therapy designation to AMT-130 based on clinical evidence from Phase I/II trials showing the potential for slowing progression of Huntington’s disease.
- The Company continues to prepare for a BLA submission of AMT-130 in the treatment of Huntington’s disease. Certain next steps and expected timing include:
- September 2025: Present topline, three-year data from the ongoing Phase I/II trials.
- Q3 2025: Initiate a fourth cohort evaluating high-dose AMT-130 in six patients with lower striatal volumes compared to those of patients enrolled in previous cohorts.
- Q4 2025: Hold pre-BLA meeting with FDA and complete PPQ campaign.
- Q1 2026: Submit BLA with a request for priority review designation.
- September 2025: Present topline, three-year data from the ongoing Phase I/II trials.
- In June 2025, the Company announced its alignment with the FDA on the statistical analysis plan (SAP) and Chemistry, Manufacturing and Controls (CMC) requirements to support a planned Biologics License Application (BLA) submission for AMT-130 in the first quarter of 2026. The primary efficacy analysis will evaluate the three-year change in the composite Unified Huntington’s Disease Rating Scale (cUHDRS) in patients receiving the high dose of AMT-130 compared to patients in a propensity score-matched external control arm derived from the ENROLL-HD data set. Certain sensitivity analyses, including one using a propensity score-weighted methodology, will also be provided. The FDA also agreed to uniQure’s approach of leveraging prior knowledge and manufacturing experience from the HEMGENIX® process, supplemented with additional full-scale Good Manufacturing Practices (GMP) batches and a single Process Performance Qualification (PPQ) run.
- Advancing additional clinical programs to proof-of-concept
- 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. The 14 clinical sites in the U.S. continue to screen patients for this study. - AMT-191 for the treatment of Fabry disease – The Company continues to enroll patients in the Phase I/IIa study and expects to present initial safety and exploratory efficacy data at the 15th ICIEM 2025 in Kyoto, Japan on Friday, September 5, 2025.
- AMT-162 for the treatment of SOD1 amyotrophic lateral sclerosis (ALS) – The Company continues to enroll patients in the Phase I/II EPISOD1 study and expects to present initial data in the first half of 2026.
- 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
- Strengthening the leadership team
- In June 2025, Kylie O’Keefe was appointed as Chief Customer and Strategy Officer, responsible for leading all commercial and medical affairs at uniQure. Ms. O’Keefe brings decades of commercial experience to the company having most recently served as Chief Commercial Officer at PTC Therapeutics, where she led global commercial strategy, operations and portfolio management for multiple rare neurology and metabolic commercial products across more than 50 countries.
- In June 2025, Kylie O’Keefe was appointed as Chief Customer and Strategy Officer, responsible for leading all commercial and medical affairs at uniQure. Ms. O’Keefe brings decades of commercial experience to the company having most recently served as Chief Commercial Officer at PTC Therapeutics, where she led global commercial strategy, operations and portfolio management for multiple rare neurology and metabolic commercial products across more than 50 countries.
- Strong financial position
- As of June 30, 2025, the Company had cash, cash equivalents and investment securities of
$377.0 million . The Company expects that cash, cash equivalents and investment securities will be sufficient to fund operations into the second half of 2027, including the launch of AMT-130.
- As of June 30, 2025, the Company had cash, cash equivalents and investment securities of
Financial Highlights
Cash position: As of June 30, 2025, the Company held cash, cash equivalents and current investment securities of
Revenues: Revenue for the three months ended June 30, 2025 was
Cost of contract manufacturing revenues: Cost of contract manufacturing revenues were nil for the three months ended June 30, 2025, compared to
R&D expenses: Research and development expenses were
SG&A expenses: Selling, general and administrative expenses were
Other expense: Other expense was
Other non-operating items, net: Other non-operating items, net was an income of
Net loss: The net loss for the three months ending June 30, 2025, was
Investor Conference Call and Webcast Information
uniQure management will host an investor conference call and webcast today, Tuesday, July 29 at 8:30 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. Interested parties participating by phone will need to register using this online form. After registering for dial-in details, all phone participants will receive an auto-generated e-mail containing a link to the dial-in number along with a personal PIN number to use to access the event by phone. If you are joining the conference call, please dial in 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 2027; the timing and outcome of regulatory interactions with respect to the AMT-130 program, FDA’s support for the program and the Company’s plans to provide further regulatory updates, including the timing of its planned BLA submission and anticipated request for priority review designation; the Company’s plans to incorporate a course of steroids as an immunosuppression strategy for patients receiving AMT-130; the Company’s plans to announce AMT-130 topline three-year data in September 2025; the initiation of a fourth cohort evaluating high-dose AMT-130; the Company’s commercialization plans, including with respect to AMT-130; and the Company’s plans for further clinical updates and plans to announce initial data in its AMT-191 and AMT-162 programs. 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 and timing for 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 filed with the SEC on February 27, 2025, its Quarterly Report on Form 10-Q filed with the SEC on May 9, 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: 781-491-4371 | 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 | ||||||||
| June 30, | December 31, | |||||||
| 2025 | 2024 | |||||||
| (in thousands, except share and per share amounts) | ||||||||
| Current assets | ||||||||
| Cash and cash equivalents | $ | 253,778 | $ | 158,930 | ||||
| Current investment securities | 123,196 | 208,591 | ||||||
| Accounts receivable | 5,606 | 5,881 | ||||||
| Prepaid expenses | 18,538 | 9,281 | ||||||
| Other current assets and receivables | 7,455 | 7,606 | ||||||
| Total current assets | 408,573 | 390,289 | ||||||
| Non-current assets | ||||||||
| Property, plant and equipment, net | 17,492 | 20,424 | ||||||
| Other investments | 29,735 | 27,464 | ||||||
| Operating lease right-of-use assets | 13,635 | 13,647 | ||||||
| Intangible assets, net | 75,604 | 71,043 | ||||||
| Goodwill | 25,340 | 22,414 | ||||||
| Deferred tax assets, net | 9,017 | 9,856 | ||||||
| Other non-current assets | 5,494 | 1,399 | ||||||
| Total non-current assets | 176,317 | 166,247 | ||||||
| Total assets | $ | 584,890 | $ | 556,536 | ||||
| Current liabilities | ||||||||
| Accounts payable | $ | 5,541 | $ | 7,227 | ||||
| Accrued expenses and other current liabilities | 31,479 | 29,225 | ||||||
| Current portion of operating lease liabilities | 3,909 | 3,601 | ||||||
| Total current liabilities | 40,929 | 40,053 | ||||||
| Non-current liabilities | ||||||||
| Long-term debt | 51,842 | 51,324 | ||||||
| Liability from royalty financing agreement | 455,259 | 434,930 | ||||||
| Operating lease liabilities, net of current portion | 10,946 | 11,136 | ||||||
| Contingent consideration, net of current portion | 15,931 | 10,860 | ||||||
| Deferred tax liability, net | 7,963 | 7,043 | ||||||
| Other non-current liabilities | 6,023 | 7,942 | ||||||
| Total non-current liabilities | 547,964 | 523,235 | ||||||
| Total liabilities | 588,893 | 563,288 | ||||||
| Shareholders' (deficit) / equity | ||||||||
| Total shareholders' (deficit) / equity | (4,003 | ) | (6,752 | ) | ||||
| Total liabilities and shareholders' (deficit) / equity | $ | 584,890 | $ | 556,536 | ||||
| uniQure N.V. | ||||||||
| UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS | ||||||||
| Three months ended June 30, | ||||||||
| 2025 | 2024 | |||||||
| (in thousands, except share and per share amounts) | ||||||||
| Total revenues | $ | 5,262 | $ | 11,126 | ||||
| Operating expenses: | ||||||||
| Cost of license revenues | (656 | ) | (234 | ) | ||||
| Cost of contract manufacturing revenues | - | (7,227 | ) | |||||
| Research and development expenses | (35,383 | ) | (33,655 | ) | ||||
| Selling, general and administrative expenses | (13,500 | ) | (15,767 | ) | ||||
| Total operating expenses | (49,539 | ) | (56,883 | ) | ||||
| Other income | 2,597 | 1,983 | ||||||
| Other expense | (2,185 | ) | (236 | ) | ||||
| Loss from operations | (43,865 | ) | (44,010 | ) | ||||
| Non-operating items, net | 6,571 | (11,341 | ) | |||||
| Loss before income tax (expense) / benefit | $ | (37,294 | ) | $ | (55,351 | ) | ||
| Income tax (expense) / benefit | (425 | ) | (948 | ) | ||||
| Net loss | $ | (37,719 | ) | $ | (56,299 | ) | ||
| Basic and diluted net loss per ordinary share | $ | (0.69 | ) | $ | (1.16 | ) | ||
| Weighted average shares used in computing basic and diluted net loss per ordinary share | 54,807,967 | 48,622,440 | ||||||
FAQ
What were uniQure's (QURE) key financial results for Q2 2025?
What progress did uniQure make with AMT-130 for Huntington's disease?
What were the initial results for uniQure's AMT-260 epilepsy treatment?
How long can uniQure fund its operations with current cash position?
Who is uniQure's new Chief Customer and Strategy Officer?
