uniQure (QURE -5.21%), a gene remedy developer targeted on uncommon illnesses, launched outcomes for the second quarter of fiscal 2025 on July 29, 2025. The headline information: the corporate’s internet loss narrowed to $0.69 per share (GAAP) in Q2 2025, forward of analyst expectations for a GAAP lack of $0.89. Income for the quarter got here in at $5.3 million, just under the $5.4 million anticipated and properly down from Q2 2024 GAAP income of $11.1 million. Nevertheless, the corporate ended the interval with a strong money stability, offering sufficient runway to fund operations into the second half of 2027. Total, the quarter illustrated cautious value management and regulatory progress, however highlighted the continuing want for profitable scientific milestones in its core gene remedy pipeline.
MetricQ2 2025Q2 2025 EstimateQ2 2024Y/Y ChangeEPS (GAAP)$(0.69)$(0.89)$(1.16)40.5 %Income (GAAP)$5.3 million$5.4 million$11.1 million(52.6 %)Analysis & Improvement Bills$35.4 million$33.7 million5.0 %Promoting, Normal & Administrative Bills$13.5 million$15.8 million(14.6 %)Money, Money Equivalents & Present Funding Securities$377.0 million$367.5 million†2.6 %
Supply: Analyst estimates for the quarter supplied by FactSet.
Enterprise Overview and Focus Areas
uniQure (QURE -5.21%) develops genetic therapies for uncommon and critical illnesses, utilizing viral vectors to ship corrected genetic materials into affected person cells. Its present lead asset is AMT-130, a gene remedy for Huntington’s illness. Huntington’s is a progressive genetic dysfunction that causes degeneration in nerve cells, with no cures presently out there.
The corporate’s key efforts middle on profitable regulatory approval for AMT-130, which might change into the primary disease-modifying remedy for Huntington’s. Its focus extends to advancing different clinical-stage applications, masking mesial temporal lobe epilepsy (with gene remedy AMT-260), SOD1-amyotrophic lateral sclerosis or ALS (with AMT-162), and Fabry illness (with AMT-191, one other gene remedy candidate). Success is determined by demonstrating strong scientific profit, navigating complicated regulatory necessities, and securing adequate sources to succeed in its milestones. Ongoing engagement with regulatory companies can be important to its technique.
Quarter in Element: Financials, Pipeline, and Regulatory Progress
Essentially the most notable accomplishment in Q2 2025 was the narrowing of internet loss (GAAP), due partially to decrease working bills and important non-operating earnings. Reported internet loss was $37.7 million (or $0.69 per share) (GAAP), down from $56.3 million (or $1.16 per share) in Q2 2024 (GAAP). This outcome beat analyst expectations by about $1.58 per share, a 176.8% outperformance (GAAP). The development was pushed by increased non-operating earnings, primarily from international forex beneficial properties, and decreased spending on promoting, basic, and administrative prices (down 14.6% to $13.5 million, GAAP). Analysis and growth bills elevated by $1.7 million year-over-year, primarily from elevated exterior program spending and contingent consideration. Income (GAAP) dropped 52.3% year-over-year to $5.3 million, falling simply shy of the $5.4 million GAAP consensus estimate. The income contraction adopted the July 2024 divestiture of its Lexington manufacturing web site, decreasing contract manufacturing and collaboration earnings, as mirrored in GAAP income.
On the regulatory entrance, AMT-130, the lead gene remedy for Huntington’s illness, achieved two main milestones. In April, the U.S. Meals and Drug Administration (FDA) granted Breakthrough Remedy designation to AMT-130, recognizing scientific proof from Part I/II research. This designation follows the sooner Regenerative Drugs Superior Remedy (RMAT) standing, each geared toward dashing growth for medication concentrating on critical or life-threatening situations. The corporate additionally aligned with the FDA on the important thing statistical evaluation plan and Chemistry, Manufacturing, and Controls (CMC) necessities, clearing a pathway to a deliberate Biologics License Utility (BLA) submission in early 2026. Through the interval, all 12 sufferers within the third trial cohort acquired both a excessive or low dose of AMT-130 plus immunosuppression. The therapy’s security remained constant, with no drug-related critical adversarial occasions, and three immunosuppression-related critical occasions that resolved with customary care. Up to date, three-year information from the primary two AMT-130 cohorts is slated for September 2025.
Additional pipeline developments included first efficacy findings for AMT-260 (epilepsy gene remedy), with the primary handled affected person experiencing a 92% discount in seizure frequency over 5 months. 4 sufferers have now been handled with AMT-191 (Fabry illness gene remedy), with preliminary security and exploratory efficacy information anticipated within the second half of the 12 months. Enrollment continued within the AMT-162 program for SOD1-ALS, with new information anticipated within the first half of 2026. Whereas these applications are nonetheless within the early scientific phases, any profitable scientific proof will likely be an necessary milestone for future worth and danger discount. Diversification past Huntington’s illness gives future optionality.
Strategically, the corporate accomplished the divestiture of its Lexington, Massachusetts manufacturing facility through the interval. Consequently, contract manufacturing income is now acknowledged internet inside different bills. This transfer is a part of a broader effort to streamline operations, deal with core analysis and growth, and put together for business launch of merchandise. Management additions included the appointment of a brand new Chief Buyer and Technique Officer, reflecting elevated deal with business readiness because the enterprise approaches potential product approval milestones.
On the monetary entrance, uniQure ended the quarter with $377.0 million in money, money equivalents, and funding securities, a small improve from $367.5 million at year-end (December 2024). This was primarily supported by proceeds from a public providing that raised roughly $80.5 million after bills. Administration reiterated that out there money ought to fund operations into the second half of 2027. Stability sheet totals confirmed important liabilities exceeding belongings, resulting in damaging shareholders’ fairness. That is primarily attributable to a royalty financing legal responsibility and excellent long-term debt—dangers which will require future capital elevating if product launches don’t materialize. Non-operating earnings turned constructive—helped by international forex beneficial properties—which contributed to the narrower internet loss. Working money wants stay targeted on analysis and growth, per its standing as a late-stage pipeline firm. There was no dividend paid or introduced.
Wanting Forward: Milestones and Investor Watchpoints
Firm management didn’t supply formal monetary steering for income or earnings for future quarters. Nevertheless, it directed consideration to a number of near-term catalysts. Essentially the most important is the three-year information readout for AMT-130, scheduled for September 2025. Ought to these outcomes maintain as much as expectations and regulatory necessities, a BLA submission for AMT-130 is deliberate for the primary quarter of 2026, with the corporate requesting a “precedence evaluation” from the FDA. Additional milestones embrace the primary scientific information from the Fabry illness (AMT-191) and ALS (AMT-162) applications, and initiation of a fourth AMT-130 cohort concentrating on sufferers with decrease striatal volumes.
Traders will likely be targeted on a number of dangers within the coming quarters. The flexibility to transform scientific progress on AMT-130 into regulatory approval and eventual product income is essential, given the continued decline in contract manufacturing and collaboration earnings.
Income and internet earnings offered utilizing U.S. usually accepted accounting rules (GAAP) except in any other case famous.
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