uniQure (QURE -8.68%), a gene therapy developer focused on rare diseases, released results for the second quarter of fiscal 2025 on July 29, 2025. The headline news: the company's net loss narrowed to $0.69 per share (GAAP) in Q2 2025, ahead of analyst expectations for a GAAP loss of $0.89. Revenue for the quarter came in at $5.3 million, just below the $5.4 million expected and well down from Q2 2024 GAAP revenue of $11.1 million. However, the company ended the period with a robust cash balance, providing enough runway to fund operations into the second half of 2027. Overall, the quarter illustrated careful cost control and regulatory progress, but highlighted the ongoing need for successful clinical milestones in its core gene therapy pipeline.
| Metric | Q2 2025 | Q2 2025 Estimate | Q2 2024 | Y/Y Change |
|---|---|---|---|---|
| EPS (GAAP) | $(0.69) | $(0.89) | $(1.16) | 40.5 % |
| Revenue (GAAP) | $5.3 million | $5.4 million | $11.1 million | (52.6 %) |
| Research & Development Expenses | $35.4 million | $33.7 million | 5.0 % | |
| Selling, General & Administrative Expenses | $13.5 million | $15.8 million | (14.6 %) | |
| Cash, Cash Equivalents & Current Investment Securities | $377.0 million | $367.5 million† | 2.6 % |
Source: Analyst estimates for the quarter provided by FactSet.
Business Overview and Focus Areas
uniQure (QURE -8.68%) develops genetic therapies for rare and serious diseases, using viral vectors to deliver corrected genetic material into patient cells. Its current lead asset is AMT-130, a gene therapy for Huntington’s disease. Huntington’s is a progressive genetic disorder that causes degeneration in nerve cells, with no cures currently available.
The company’s key efforts center on winning regulatory approval for AMT-130, which could become the first disease-modifying therapy for Huntington’s. Its focus extends to advancing other clinical-stage programs, covering mesial temporal lobe epilepsy (with gene therapy AMT-260), SOD1-amyotrophic lateral sclerosis or ALS (with AMT-162), and Fabry disease (with AMT-191, another gene therapy candidate). Success depends on demonstrating robust clinical benefit, navigating complex regulatory requirements, and securing sufficient resources to reach its milestones. Ongoing engagement with regulatory agencies is also essential to its strategy.
Quarter in Detail: Financials, Pipeline, and Regulatory Progress
The most notable accomplishment in Q2 2025 was the narrowing of net loss (GAAP), due in part to lower operating expenses and significant non-operating income. Reported net 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 result beat analyst expectations by about $1.58 per share, a 176.8% outperformance (GAAP). The improvement was driven by higher non-operating income, mainly from foreign currency gains, and reduced spending on selling, general, and administrative costs (down 14.6% to $13.5 million, GAAP). Research and development expenses increased by $1.7 million year-over-year, primarily from increased external program spending and contingent consideration. Revenue (GAAP) dropped 52.3% year-over-year to $5.3 million, falling just shy of the $5.4 million GAAP consensus estimate. The revenue contraction followed the July 2024 divestiture of its Lexington manufacturing site, reducing contract manufacturing and collaboration income, as reflected in GAAP revenue.
On the regulatory front, AMT-130, the lead gene therapy for Huntington’s disease, achieved two major milestones. In April, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to AMT-130, recognizing clinical evidence from Phase I/II studies. This designation follows the earlier Regenerative Medicine Advanced Therapy (RMAT) status, both aimed at speeding development for drugs targeting serious or life-threatening conditions. The company also aligned with the FDA on the key statistical analysis plan and Chemistry, Manufacturing, and Controls (CMC) requirements, clearing a pathway to a planned Biologics License Application (BLA) submission in early 2026. During the period, all 12 patients in the third trial cohort received either a high or low dose of AMT-130 plus immunosuppression. The treatment’s safety remained consistent, with no drug-related serious adverse events, and three immunosuppression-related serious events that resolved with standard care. Updated, three-year data from the first two AMT-130 cohorts is slated for September 2025.
Further pipeline developments included first efficacy findings for AMT-260 (epilepsy gene therapy), with the first treated patient experiencing a 92% reduction in seizure frequency over five months. Four patients have now been treated with AMT-191 (Fabry disease gene therapy), with initial safety and exploratory efficacy data expected in the second half of the year. Enrollment continued in the AMT-162 program for SOD1-ALS, with new data expected in the first half of 2026. While these programs are still in the early clinical phases, any successful clinical proof will be an important milestone for future value and risk reduction. Diversification beyond Huntington’s disease offers future optionality.
Strategically, the company completed the divestiture of its Lexington, Massachusetts manufacturing facility during the period. As a result, contract manufacturing revenue is now recognized net within other expenses. This move is part of a broader effort to streamline operations, focus on core research and development, and prepare for commercial launch of products. Leadership additions included the appointment of a new Chief Customer and Strategy Officer, reflecting increased focus on commercial readiness as the business approaches potential product approval milestones.
On the financial front, uniQure ended the quarter with $377.0 million in cash, cash equivalents, and investment securities, a small increase from $367.5 million at year-end (December 2024). This was primarily supported by proceeds from a public offering that raised approximately $80.5 million after expenses. Management reiterated that available cash should fund operations into the second half of 2027. Balance sheet totals showed significant liabilities exceeding assets, leading to negative shareholders’ equity. This is mainly due to a royalty financing liability and outstanding long-term debt—risks that may require future capital raising if product launches do not materialize. Non-operating income turned positive—helped by foreign currency gains—which contributed to the narrower net loss. Operating cash needs remain focused on research and development, consistent with its status as a late-stage pipeline company. There was no dividend paid or announced.
Looking Ahead: Milestones and Investor Watchpoints
Company leadership did not offer formal financial guidance for revenue or profits for future quarters. However, it directed attention to several near-term catalysts. The most critical is the three-year data readout for AMT-130, scheduled for September 2025. Should these results hold up to expectations and regulatory requirements, a BLA submission for AMT-130 is planned for the first quarter of 2026, with the company requesting a “priority review” from the FDA. Further milestones include the first clinical data from the Fabry disease (AMT-191) and ALS (AMT-162) programs, and initiation of a fourth AMT-130 cohort targeting patients with lower striatal volumes.
Investors will be focused on several risks in the coming quarters. The ability to convert clinical progress on AMT-130 into regulatory approval and eventual product revenue is crucial, given the continued decline in contract manufacturing and collaboration income.
Revenue and net income presented using U.S. generally accepted accounting principles (GAAP) unless otherwise noted.
