QURE Stock: Insider Activity, Filings & Research
uniQure N.V. (QURE) — Drillr’s hub for QURE insider activity, SEC filings, earnings signals and AI research. Over the trailing 3 months, QURE insiders filed 0 open-market buys and 2 sales (SEC Form 4).
QURE insider trading activity (SEC Form 4)
| Date | Insider | Type | Shares | Price |
|---|---|---|---|---|
| May 12, 2026 | Abi-Saab Walidofficer: Chief Medical Officer | Option | 25,000 | $5.59 |
| May 12, 2026 | Abi-Saab Walidofficer: Chief Medical Officer | Sell | 20,000 | $25.00 |
| May 12, 2026 | Abi-Saab Walidofficer: Chief Medical Officer | Sell | 25,000 | $25.10 |
| Mar 9, 2026 | O'Keefe Kylieofficer: Chief Customer & Strat Officer | Grant | 31,900 | $9.04 |
| Mar 9, 2026 | O'Keefe Kylieofficer: Chief Customer & Strat Officer | Grant | 19,800 | — |
| Mar 6, 2026 | Kapusta Matthew Cdirector, officer: CEO, Managing Director | Sell | 14,581 | $9.06 |
| Mar 6, 2026 | Potts Jeannetteofficer: Chief Legal Officer | Sell | 3,412 | $9.06 |
| Mar 6, 2026 | Kapusta Matthew Cdirector, officer: CEO, Managing Director | Grant | 70,600 | — |
| Mar 6, 2026 | Potts Jeannetteofficer: Chief Legal Officer | Grant | 57,200 | $9.04 |
| Mar 6, 2026 | Kapusta Matthew Cdirector, officer: CEO, Managing Director | Grant | 113,800 | $9.04 |
| Mar 6, 2026 | Abi-Saab Walidofficer: Chief Medical Officer | Sell | 808 | $9.06 |
| Mar 6, 2026 | KLEMT CHRISTIANofficer: Chief Financial Officer | Grant | 39,600 | — |
| Mar 6, 2026 | KLEMT CHRISTIANofficer: Chief Financial Officer | Grant | 63,900 | $9.04 |
| Mar 6, 2026 | Abi-Saab Walidofficer: Chief Medical Officer | Grant | 67,200 | $9.04 |
| Mar 6, 2026 | Abi-Saab Walidofficer: Chief Medical Officer | Grant | 41,700 | — |
Source: QURE SEC Form 4 filings, latest May 12, 2026. For informational purposes only — not investment advice.
uniQure N.V. company profile
Overview
uniQure N.V. (NASDAQ:QURE) is a Dutch gene therapy company founded in 1998 and headquartered in Amsterdam, the Netherlands. The company went public on NASDAQ in February 2014. uniQure focuses on developing gene therapies for patients suffering from genetic and other devastating diseases, with a particular emphasis on rare neurological conditions and inherited disorders. The company has evolved from its early research phases to become a clinical-stage biotechnology firm with multiple programs advancing through various stages of human trials.
Business
uniQure operates in the gene therapy sector of biotechnology, which involves introducing genetic material into patients' cells to treat or prevent disease. Gene therapy represents a cutting-edge approach to medicine that aims to correct defective genes or provide new cellular functions by delivering therapeutic DNA or RNA directly into patient cells. The company's pipeline consists of several distinct therapeutic programs targeting different diseases: 1. AMT-130 for Huntington's Disease - This is uniQure's most advanced program, currently in Phase I/II clinical trials. Huntington's disease is a fatal inherited disorder that causes progressive breakdown of nerve cells in the brain, affecting movement, cognitive ability, and emotional control. AMT-130 has received FDA Breakthrough Therapy Designation, which expedites the development and review process for treatments addressing unmet medical needs. 2. Etranacogene dezaparvovec (AMT-061) for Hemophilia B - Previously the company's lead program, this gene therapy was designed to treat hemophilia B, a rare bleeding disorder caused by insufficient levels of clotting factor IX. The program completed Phase III trials but faced regulatory challenges. 3. AMT-210 for Parkinson's Disease - A preclinical program targeting this neurodegenerative disorder that affects movement and can cause tremors, stiffness, and difficulty with balance and coordination. 4. AMT-260 for Temporal Lobe Epilepsy - Recently initiated clinical studies for refractory temporal lobe epilepsy, a form of seizure disorder that doesn't respond well to conventional treatments. 5. Additional Programs - The company has several other programs in development including treatments for Fabry disease (a rare genetic disorder), SOD1-ALS (a form of amyotrophic lateral sclerosis), and Alzheimer's disease. Revenue is currently minimal and primarily comes from research collaborations and licensing agreements rather than product sales, as the company has no approved commercial products yet.
Revenue model
uniQure's business model is typical of a clinical-stage biotechnology company, with revenue currently generated through research collaborations, licensing agreements, and milestone payments from pharmaceutical partners. The company reported only $1.6 million in revenue for Q1 2025, down from $8.5 million in the prior year period, reflecting the lumpy nature of partnership income. The primary path to profitability lies in successfully advancing gene therapies through clinical trials to regulatory approval and eventual commercialization. Once approved, gene therapies typically command premium pricing due to their innovative nature and treatment of rare diseases with limited alternatives. The company's focus on rare genetic diseases positions it in markets where high prices can be justified by the severity of conditions and lack of treatment options. Key factors that could positively impact margins include successful clinical trial outcomes, regulatory approvals (particularly for AMT-130 in Huntington's disease), strategic partnerships with larger pharmaceutical companies for commercialization, and the ability to leverage manufacturing platforms across multiple programs. The company recently divested its manufacturing facility to reduce cash burn, indicating a shift toward outsourced production models. Factors that could negatively impact the business include clinical trial failures, regulatory setbacks, increased competition in gene therapy, manufacturing challenges and costs, and the inherently high cost of developing treatments for rare diseases. The gene therapy field faces ongoing scrutiny regarding safety profiles and long-term efficacy, which could affect regulatory pathways and market acceptance. Additionally, the company's current high cash burn rate of approximately $44 million per quarter creates pressure to achieve meaningful clinical milestones before funding runs out.
Competitive moat
uniQure's competitive position is relatively weak to moderate in the rapidly evolving gene therapy landscape. The company's primary potential moat lies in its clinical data and regulatory positioning, particularly with AMT-130's FDA Breakthrough Therapy Designation for Huntington's disease. This designation provides regulatory advantages and reflects the FDA's recognition of the program's potential to address an unmet medical need. However, the company faces significant competitive threats. The gene therapy field is highly competitive with numerous well-funded biotechnology companies and pharmaceutical giants investing heavily in similar approaches. Larger competitors often have superior financial resources, manufacturing capabilities, and regulatory expertise. The company's decision to divest its manufacturing facility reduces its control over a critical component of gene therapy production. The intellectual property position, while important, may not provide durable protection given the rapid pace of innovation in gene therapy and the potential for competitors to develop alternative approaches to the same targets. Additionally, uniQure's focus on rare diseases, while potentially lucrative, also means smaller addressable markets that may not support multiple competitors. The company's main competitive advantage currently rests on execution speed and clinical data quality rather than structural barriers to entry. Success will largely depend on achieving superior clinical outcomes and navigating regulatory processes more effectively than competitors. The Huntington's disease program represents the best opportunity to establish a meaningful market position, as there are currently no disease-modifying treatments available for this condition.
Risks & safety
uniQure presents moderate financial risk with reasonable near-term liquidity but ongoing cash burn concerns. • Liquidity Position: Strong current ratio of 12.0x with $217 million in cash and short-term investments as of Q1 2025, providing runway into H2 2027 • Cash Burn: High quarterly burn rate of approximately $44 million in free cash flow, requiring continued clinical progress to maintain investor confidence • Debt Level: Manageable debt-to-equity ratio of 1.97x, though total liabilities of $572 million exceed total assets of $605 million • Solvency Risk: Moderate risk given cash runway extends 2+ years, but dependent on successful clinical outcomes and potential additional financing • Valuation Metrics: Trading at negative P/E due to losses; Price-to-book ratio of 16.7x reflects significant premium to tangible assets • Revenue Multiple: Extremely high given minimal current revenue of $1.6 million quarterly • Other Considerations: Recent $80 million follow-on offering demonstrates continued access to capital markets; clinical-stage biotech inherently carries binary risk based on trial outcomes
Recent development
Over the past few years, uniQure has undergone significant strategic shifts, moving away from its previous focus on hemophilia B toward neurological disorders. The company's AMT-061 hemophilia B program, once its lead candidate, faced regulatory challenges that prompted a strategic pivot toward its Huntington's disease program AMT-130. The AMT-130 program for Huntington's disease has become the company's flagship, receiving FDA Breakthrough Therapy Designation and completing enrollment of Cohort 3 with prophylactic immunosuppression. The company is planning a Biologics License Application (BLA) submission with three-year data and expects regulatory updates in Q2 2025. This represents a major strategic focus shift toward addressing neurodegenerative diseases where there are currently no disease-modifying treatments available. uniQure has also expanded its pipeline breadth by initiating clinical studies in multiple new indications including refractory temporal lobe epilepsy, Fabry disease, and SOD1-ALS. The company completed enrollment of the first two dose cohorts in its SOD1-ALS trial and expects to present initial Fabry disease data in the second half of 2025. From an operational standpoint, the company has taken steps to reduce cash burn and streamline operations, including the divestiture of its manufacturing facility. This move reflects a strategic decision to focus resources on clinical development rather than manufacturing infrastructure, potentially improving capital efficiency while relying on contract manufacturing organizations for production needs. The company completed an $80 million follow-on offering to strengthen its financial position and extend its operational runway into the second half of 2027, providing sufficient time to advance its key programs through critical clinical milestones.
QURE company profile · for informational purposes only — not investment advice.
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