uniQure Announces First Quarter 2025 Financial Results and Highlights of Recent Company Progress
- FDA granted Breakthrough Therapy designation to AMT-130 for Huntington's disease
- Strong cash position of $409.0 million, funding operations into H2 2027
- AMT-130 shows good safety profile with no treatment-related serious adverse events
- Successful completion of $80.5 million public offering
- Net loss decreased to $43.6 million from $65.6 million year-over-year
- Revenue declined to $1.6 million from $8.5 million in Q1 2024
- Contract manufacturing revenue decreased by $4.0 million
- Collaboration revenue decreased by $3.3 million
Insights
AMT-130's breakthrough designation and BLA progress mark significant advancements for uniQure despite quarterly revenue decline and continued losses.
uniQure's Q1 financial results reveal some crucial developments for this gene therapy developer. The Breakthrough Therapy designation for AMT-130 from the FDA represents a significant regulatory milestone that potentially accelerates the pathway to approval for their Huntington's disease therapy. This designation, coupled with constructive FDA interactions regarding a potential Accelerated Approval pathway, substantially de-risks the regulatory process for their lead asset.
While quarterly revenue declined to just
The safety data from AMT-130's third cohort is particularly encouraging - showing the therapy continues to be generally well-tolerated with no treatment-related serious adverse events. The three serious adverse events reported were related to immunosuppression rather than the therapy itself, and all resolved with supportive care. This safety profile strengthens AMT-130's case ahead of the potential BLA submission.
uniQure's pipeline diversification continues with progress across multiple programs:
- AMT-260 for epilepsy - initial data from first patient expected May 29
- AMT-191 for Fabry disease - four patients treated with initial data expected H2 2025
- AMT-162 for ALS - favorable safety review and second dose cohort completed
Financially, uniQure strengthened its position with a successful
The company's planned BLA submission timeline update expected in Q2 2025 will be a crucial catalyst, as will the upcoming clinical data readouts from multiple programs. These potential value inflection points may significantly impact uniQure's trajectory over the coming year.
uniQure shows promising regulatory progress and strong cash position despite revenue decline, positioning well for potential commercialization.
From a financial perspective, uniQure's Q1 2025 results present a nuanced picture that requires looking beyond the headline numbers. Revenue decreased substantially to
More encouraging is the
The company has successfully bolstered its balance sheet through a
The
From an investment standpoint, uniQure is effectively managing its burn rate while advancing multiple clinical programs and preparing for potential commercialization of AMT-130. The Breakthrough Therapy designation significantly reduces regulatory risk for their lead asset and potentially accelerates the timeline to market. The company appears financially disciplined in resource allocation, focusing investments strategically across their pipeline while maintaining adequate liquidity.
With multiple catalysts ahead including a regulatory update on AMT-130's BLA timeline in Q2 and clinical data readouts across three programs in 2025, uniQure has effectively positioned itself financially to navigate these crucial near-term milestones that could drive significant value creation.
~ AMT-130 granted Breakthrough Therapy designation by FDA ~
~ Initial safety data from third cohort of Phase I/II study show AMT-130 continues to be generally well-tolerated, with no treatment-related serious adverse events ~
~ Held Type B FDA meetings in the first and second quarters of 2025 to advance BLA preparations for AMT-130; regulatory update expected in the second quarter of 2025 ~
~ AMT-260 clinical data from first patient to be presented at Epilepsy Therapies & Diagnostics Development Symposium on May 29, 2025 ~
~ Cash, cash equivalents and current investment securities of approximately
~ uniQure to host inaugural earnings call at 8:30 a.m. ET ~
LEXINGTON, Mass. and AMSTERDAM, the Netherlands, May 09, 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 first quarter of 2025 and highlighted recent progress across its business.
“We continued to have constructive interactions with the U.S. Food and Drug Administration (FDA) in the first and second quarters of 2025, enabling us to further advance AMT-130 towards a planned Biologics License Application (BLA) submission,” said Matt Kapusta, chief executive officer of uniQure. “The recently granted Breakthrough Therapy designation is a significant milestone and underscores the FDA’s continued engagement with AMT-130 as a potential disease-modifying treatment for Huntington’s disease. We expect to provide a regulatory update in the second quarter, including guidance on the potential timing of our BLA submission for AMT-130, and anticipate sharing new clinical data from our AMT-130 Phase I/II trials in the third quarter. As we prepare for potential commercialization, we remain committed to making financially disciplined investments.”
Mr. Kapusta continued, “In addition to AMT-130, it’s important to highlight the continued progress of our broader pipeline of investigational gene therapies. We look forward to sharing initial data from the first patient treated with AMT-260 for refractory mesial temporal lobe epilepsy later this quarter, and we remain on track to present initial data from our AMT-191 study in Fabry disease in the second half of the year. With our strong financial position, we believe that we are well-resourced to execute across our pipeline and deliver meaningful value to patients and shareholders alike.”
Recent Company Developments and Updates
- Advancing AMT-130 Toward Accelerated Approval for Huntington’s disease
- 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 designation followed a November 2024 multidisciplinary meeting with the FDA during which key elements of an Accelerated Approval pathway for AMT-130 were discussed and aligned.
- In March 2025, the Company held a Type B meeting with the FDA to discuss CMC requirements in support of a planned BLA submission. An additional Type B meeting focused on the pivotal statistical analysis plan was held in the second quarter of 2025. The Company expects to provide a regulatory update, including the potential timing of a BLA submission, after the receipt of official meeting minutes.
- In February 2025, the Company completed enrollment of all 12 patients in the third cohort of the Phase I/II study. Patients were blinded and randomized to receive either the high (n=6) or low dose (n=6) of AMT-130 in conjunction with an immunosuppression regimen.
- 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 designation followed a November 2024 multidisciplinary meeting with the FDA during which key elements of an Accelerated Approval pathway for AMT-130 were discussed and aligned.
Key observations include:
- AMT-130 was generally well-tolerated at both dose levels;
- No treatment-related serious adverse events were reported;
- Three serious adverse events related to immunosuppression (mania, infection, and fever) were observed; all resolved fully with supportive care and;
- Perioperative changes in CSF NfL were consistent with previously reported observations, reinforcing their association with the surgical administration.
The Company plans to review these data with outside advisors and does not anticipate any impact on the potential timing of a BLA submission.
- 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 data on 24 treated patients.
- Advancing additional clinical programs to proof-of-concept
- AMT-260 for the treatment of refractory mesial temporal lobe epilepsy (mTLE) – The Company continues to screen patients across 12 clinical sites, with plans to activate additional centers in the second half of the year. The Company expects to present initial safety and exploratory efficacy data from the first treated patient at the Epilepsy Therapies & Diagnostics Development Symposium on Thursday, May 29, 2025.
- AMT-191 for the treatment of Fabry disease – In February 2025, the Company announced a favorable recommendation from the independent data monitoring committee (IDMC) following a review of safety data from the first two patients treated in the Phase I/IIa study. The Company has treated a total of four patients in the first dose cohort. The Company plans to present initial data from the study in the second half of 2025.
- 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 initial safety data from the first study cohort in the Phase I/II EPISOD1 study. The Company has completed enrollment in a second dose cohort and expects to initiate enrollment in the third dose cohort in the third quarter of 2025. The Company expects to present initial data from the study in the first half of 2026.
- AMT-260 for the treatment of refractory mesial temporal lobe epilepsy (mTLE) – The Company continues to screen patients across 12 clinical sites, with plans to activate additional centers in the second half of the year. The Company expects to present initial safety and exploratory efficacy data from the first treated patient at the Epilepsy Therapies & Diagnostics Development Symposium on Thursday, May 29, 2025.
- 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 public offering price of
$17.00 per share for net proceeds of approximately$80.5 million after deducting underwriting discounts and commissions and offering expenses.
- 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 public offering price of
Financial Highlights
Cash position: As of March 31, 2025, the Company held cash, cash equivalents and current investment securities of
Revenues: Revenue for the three months ended March 31, 2025 was
Cost of contract manufacturing revenues: Cost of contract manufacturing revenues were nil for the three months ended March 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
Other non-operating items, net: Other non-operating items, net was an expense of
Net loss: The net loss for the three months ending March 31, 2025, was
Investor Conference Call and Webcast Information
uniQure management will host an investor conference call and webcast today, Friday, May 9 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 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 timing of its planned BLA submission; whether AMT-130 continues to be well tolerated when dosed with immunosuppression and the Company’s review of data from the third cohort of AMT-130; the Company’s commercialization plans; and the Company’s plans for further clinical updates and plans to announce additional interim data in its AMT-130, AMT-260, 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 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 filed with the SEC on 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 Direct: 617-306-9137 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 | ||||||
| March 31, | December 31, | |||||
| 2025 | 2024 | |||||
| (in thousands, except share and per share amounts) | ||||||
| Current assets | ||||||
| Cash and cash equivalents | $ | 217,229 | $ | 158,930 | ||
| Current investment securities | 191,782 | 208,591 | ||||
| Accounts receivable | 2,522 | 5,881 | ||||
| Prepaid expenses | 13,420 | 9,281 | ||||
| Other current assets and receivables | 10,468 | 7,606 | ||||
| Total current assets | 435,421 | 390,289 | ||||
| Non-current assets | ||||||
| Property, plant and equipment, net | 18,081 | 20,424 | ||||
| Other investments | 28,273 | 27,464 | ||||
| Operating lease right-of-use assets | 13,466 | 13,647 | ||||
| Intangible assets, net | 71,992 | 71,043 | ||||
| Goodwill | 23,351 | 22,414 | ||||
| Deferred tax assets, net | 9,390 | 9,856 | ||||
| Other non-current assets | 5,429 | 1,399 | ||||
| Total non-current assets | 169,982 | 166,247 | ||||
| Total assets | $ | 605,403 | $ | 556,536 | ||
| Current liabilities | ||||||
| Accounts payable | $ | 5,268 | $ | 7,227 | ||
| Accrued expenses and other current liabilities | 27,344 | 29,225 | ||||
| Current portion of operating lease liabilities | 3,698 | 3,601 | ||||
| Total current liabilities | 36,310 | 40,053 | ||||
| Non-current liabilities | ||||||
| Long-term debt | 51,592 | 51,324 | ||||
| Liability from royalty financing agreement | 446,660 | 434,930 | ||||
| Operating lease liabilities, net of current portion | 10,906 | 11,136 | ||||
| Contingent consideration, net of current portion | 12,560 | 10,860 | ||||
| Deferred tax liability, net | 7,338 | 7,043 | ||||
| Other non-current liabilities | 6,351 | 7,942 | ||||
| Total non-current liabilities | 535,407 | 523,235 | ||||
| Total liabilities | 571,717 | 563,288 | ||||
| Shareholders' equity / (deficit) | ||||||
| Total shareholders' equity / (deficit) | 33,686 | (6,752 | ) | |||
| Total liabilities and shareholders' equity / (deficit) | $ | 605,403 | $ | 556,536 | ||
| uniQure N.V. UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS | |||||||
| Three months ended March 31, | |||||||
| 2025 | 2024 | ||||||
| (in thousands, except share and per share amounts) | |||||||
| Total revenues | $ | 1,567 | $ | 8,485 | |||
| Operating expenses: | |||||||
| Cost of license revenues | (197 | ) | (150 | ) | |||
| Cost of contract manufacturing revenues | - | (9,076 | ) | ||||
| Research and development expenses | (36,140 | ) | (40,692 | ) | |||
| Selling, general and administrative expenses | (10,908 | ) | (13,937 | ) | |||
| Total operating expenses | (47,245 | ) | (63,855 | ) | |||
| Other income | 8,306 | 1,376 | |||||
| Other expense | (1,959 | ) | (234 | ) | |||
| Loss from operations | (39,331 | ) | (54,228 | ) | |||
| Non-operating items, net | (3,810 | ) | (10,734 | ) | |||
| Loss before income tax expense | $ | (43,141 | ) | $ | (64,962 | ) | |
| Income tax expense | (496 | ) | (656 | ) | |||
| Net loss | $ | (43,637 | ) | $ | (65,618 | ) | |
| Basic and diluted net loss per ordinary share | $ | (0.82 | ) | $ | (1.36 | ) | |
| Weighted average shares used in computing basic and diluted net loss per ordinary share | 53,110,580 | 48,384,510 | |||||
FAQ
What is the significance of AMT-130's Breakthrough Therapy designation from the FDA for QURE stock?
What is uniQure's (QURE) cash runway as of Q1 2025?
How did uniQure's (QURE) revenue perform in Q1 2025 compared to Q1 2024?
What are the key pipeline developments for uniQure (QURE) in 2025?
What was uniQure's (QURE) net loss per share in Q1 2025?
