uniQure Announces First Quarter 2026 Financial Results and Provides Recent Company Updates
~ Advancing FDA interactions on AMT-130 for Huntington’s disease; Type B meeting scheduled for the second quarter of 2026 ~
~ Progressing AMT-130 toward expected UK regulatory submission; MAA on track for third quarter of 2026 following successful pre-submission meeting with UK MHRA ~
~ Enrollment in AMT-260 temporal lobe epilepsy program on track; clinical update from first cohort in Phase I/IIa study to be presented at the Epilepsy Foundation Pipeline Conference ~
~ Presented updated data from AMT-191 Phase I/IIa in Fabry disease study showed sustained increases in α-Gal A Enzyme Activity and stable Lyso-Gb3 levels; subsequently all 11 dosed patients have discontinued enzyme replacement therapy ~
~ Strong balance sheet with $586.6 million in cash, cash equivalents and current investment securities as of March 31, 2026 and runway into the second half of 2029 ~
~ uniQure to host earnings call at 8:30 a.m. ET ~
LEXINGTON, Mass. and AMSTERDAM, May 05, 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 first quarter of 2026 and highlighted recent progress across its business.
“During the first quarter of 2026, we remained focused on advancing AMT-130 to patients globally as rapidly as possible while executing across our broader pipeline,” said Matthew Kapusta, chief executive officer at uniQure. “We believe our data continue to support the potential for AMT-130 to fundamentally change the treatment landscape for Huntington’s disease, and we look forward to continued engagement with the FDA. In parallel, following a constructive interaction with the MHRA, we are preparing to submit an MAA in the third quarter and evaluating additional international opportunities.”
“We expect to deliver key clinical updates throughout 2026, including data from our AMT-260 program in refractory mesial temporal lobe epilepsy later in the second quarter and four-year AMT-130 data analysis in the third quarter,” Mr. Kapusta continued. “With these important milestones ahead, we remain committed to advancing our programs with urgency while maintaining disciplined capital allocation to drive long-term shareholder value.”
Recent Company Developments and Updates
Advancing AMT-130 for the treatment of Huntington’s disease
Continued clinical progress in pipeline programs
AMT-260 for the treatment of refractory mesial temporal lobe epilepsy (MTLE)
AMT-191 for the treatment of Fabry disease
AMT-162 for the treatment of SOD1 amyotrophic lateral sclerosis (ALS)
Focused execution and strong financial position
Financial Highlights
Cash Position: As of March 31, 2026, the Company held $586.6 million in cash, cash equivalents and current investment securities, compared to $622.5 million as of December 31, 2025. The Company expects that cash, cash equivalents and investment securities will be sufficient to fund operations into the second half of 2029.
Revenues: Revenue for the three months ended March 31, 2026 was $3.6 million, compared to $1.6 million in the same period in 2025. The increase of $2.0 million is due to an increase in license revenue, compared to the prior period.
R&D Expenses: Research and development expenses were $29.2 million for the three months ended March 31, 2026, compared to $36.1 million during the same period in 2025. The $6.9 million decrease was driven by a $2.6 million decrease in the fair value of contingent consideration, a $1.2 million decrease in costs related to external program spend, a $1.6 million decrease in employee and contractor-related expenses, including share-based compensation, and a $1.6 million decrease in facilities and other expenses, compared to the prior period.
SG&A Expenses: Selling, general and administrative expenses were $20.1 million for the three months ended March 31, 2026, compared to $10.9 million during the same period in 2025. The $9.2 million increase was primarily related to a $5.5 million increase in employee and contractor-related expenses, including share-based compensation, mainly as a result of employees recruited in 2025 to support commercial planning of AMT-130, a $1.8 million increase in professional fees, a $0.6 million increase in intellectual property fees, and a $1.3 million increase in information technology costs and other expenses, compared to the prior period.
Other Income: Other income was $1.6 million for the three months ended March 31, 2026, compared to $8.3 million during the comparative period in 2025. The $6.7 million decrease was primarily related to a prior period one-time gain of $6.0 million related to the sale of critical reagents and a $0.8 million decrease in research and development grants, compared to the prior period.
Other Expense: Other expense was $1.5 million for the three months ended March 31, 2026, compared to $2.0 million during the same period in 2025. The decrease was primarily due to a $0.3 million decrease in costs associated with the supply of Hemgenix® to CSL Behring and a $0.2 million decrease in sublease expenses in the three months ended March 31, 2026, compared to the prior period.
Other non-Operating Items, net: Other non-operating items, net was an expense of $7.3 million for the three months ended March 31, 2026, compared to an expense of $3.8 million for the same period in 2025. The $3.5 million increase was primarily related to an increase in net foreign currency losses of $9.5 million, offset by a $1.1 million increase in interest income, a $1.1 million decrease in interest expense, and a $3.8 million gain resulting from changes in the fair value of the liability related to pre-funded warrants, compared to the prior period.
Net loss: The net loss for the three months ending March 31, 2026, was $53.5 million, or $0.85 basic and diluted loss per ordinary share, compared to a $43.6 million net loss for the comparative period in 2025, or $0.82 basic and diluted loss per ordinary share.
Upcoming investor events:
Investor Conference Call and Webcast Information
uniQure management will host an investor conference call and webcast today, Tuesday, May 5th 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. 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, 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; the Company’s ability and plans to strategically advance its programs; 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 and have a Type B meeting regarding a potential new clinical trial design and statistical analysis plan for the four-year analysis of AMT-130, and the Company’s plans to submit a MAA to the MHRA in the third quarter of 2026; the potential for AMT-130 to change the treatment landscape for Huntington’s disease; the Company’s plans to enroll an additional six patients in a second cohort in the Phase I/IIa study for AMT-260 by mid-2026; the Company’s plans to provide further clinical updates, including plans to announce additional data from the Company’s AMT-260 program in June 2026 and four-year data from the Company’s AMT-130 program in the third quarter of 2026; the Company’s plans to continue to collect safety data from patients in the EPISOD1 trial of AMT-162; the timing of when the Company’s HEMGENIX® supply and minimum purchase commitments are expected to terminate pursuant to agreements entered into with CSL Behring and Genezen; 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 and MHRA, 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 new 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.
1 Normal range (1.38 – 8.66 nmol); mean normal of 3.57 nmol