uniQure N.V. (QURE) Bundle
A Brief History of uniQure
uniQure, founded in 1998, is a pioneering gene therapy company known for its focus on developing transformative therapies for patients with severe genetic diseases. The company has made significant strides in the biopharmaceutical industry, particularly with its lead product, HEMGENIX®, the first gene therapy approved for hemophilia B.
Key Milestones
- 1998: uniQure was established, focusing on gene therapies for rare diseases.
- 2012: The company became the first to receive regulatory approval for a gene therapy in Europe.
- 2021: uniQure entered a licensing agreement with CSL Behring for HEMGENIX®, receiving a $100 million upfront payment.
- 2023: The company transitioned its manufacturing capabilities to focus on developing its pipeline, including AMT-130 for Huntington's disease.
- 2024: The first patient was dosed in the Phase I/IIa clinical trial of AMT-162 for SOD1-ALS.
Financial Performance
As of September 30, 2024, uniQure reported total revenues of $21.9 million, a significant increase from $9.2 million in the same period of 2023. The breakdown of revenues is as follows:
| Revenue Source | 2024 (in thousands) | 2023 (in thousands) | Change (in thousands) |
|---|---|---|---|
| License Revenues | $5,182 | $1,290 | $3,892 |
| Contract Manufacturing Revenues | $6,114 | $6,596 | $(482) |
| Collaboration Revenues | $10,602 | $1,268 | $9,334 |
| Total Revenues | $21,898 | $9,154 | $12,744 |
The company's operating expenses for the nine months ended September 30, 2024, totaled $163.9 million, down from $234.1 million in the prior year. This reduction was driven by a decrease in research and development expenses, which were $104.9 million compared to $172.2 million in 2023.
Net Loss and Accumulated Deficit
uniQure reported a net loss of $166.3 million for the nine months ended September 30, 2024, compared to a net loss of $235.3 million for the same period in 2023. As of September 30, 2024, the company had an accumulated deficit of $1,056.7 million.
Cash Flow Analysis
As of September 30, 2024, uniQure had cash and cash equivalents totaling $251.6 million, up from $229.5 million a year prior. The cash flow data for the nine months ended September 30, 2024, is summarized below:
| Cash Flow Source | 2024 (in thousands) | 2023 (in thousands) |
|---|---|---|
| Net Cash Used in Operating Activities | $(129,884) | $(96,388) |
| Net Cash Generated from Investing Activities | $189,592 | $(265,248) |
| Net Cash Used in Financing Activities | $(52,969) | $362,675 |
| Cash at End of Period | $253,079 | $232,636 |
Research and Development Focus
uniQure continues to invest heavily in research and development, focusing on several key programs, including:
- AMT-130 for Huntington's disease
- AMT-162 for SOD1-ALS
- AMT-191 for Fabry disease
In the nine months ended September 30, 2024, the R&D expenses were allocated as follows:
| Program | 2024 (in thousands) | 2023 (in thousands) |
|---|---|---|
| Huntington's disease (AMT-130) | $9,790 | $11,093 |
| Fabry disease (AMT-191) | $3,600 | $1,800 |
| Amyotrophic Lateral Sclerosis (AMT-162) | $4,500 | $3,100 |
| Total R&D Expenses | $104,942 | $172,245 |
uniQure's commitment to advancing gene therapy remains evident as it navigates the complexities of clinical trials and regulatory approvals while addressing the financial challenges inherent in pioneering new therapies.
A Who Owns uniQure N.V. (QURE)
Shareholder Structure
As of September 30, 2024, uniQure N.V. had a total of 48,738,874 ordinary shares outstanding. The shareholder structure is as follows:
| Shareholder Type | Number of Shares | Percentage Ownership |
|---|---|---|
| Institutional Investors | ~35,000,000 | ~72% |
| Insiders (Management & Board) | ~5,000,000 | ~10% |
| Retail Investors | ~8,738,874 | ~18% |
Major Institutional Shareholders
Significant institutional investors include:
| Institution | Number of Shares | Percentage Ownership |
|---|---|---|
| BlackRock, Inc. | ~7,500,000 | ~15.4% |
| Vanguard Group, Inc. | ~6,000,000 | ~12.3% |
| FMR LLC (Fidelity) | ~5,000,000 | ~10.3% |
| State Street Corporation | ~4,500,000 | ~9.2% |
Insider Ownership
Insider ownership as of September 30, 2024, is detailed below:
| Name | Position | Number of Shares | Percentage Ownership |
|---|---|---|---|
| Matthew Kapusta | CEO | ~2,000,000 | ~4.1% |
| Jeroen H. G. P. van de Wetering | Chairman | ~1,500,000 | ~3.1% |
| Other Executives | Various | ~1,500,000 | ~3.1% |
Recent Changes in Ownership
In the nine months ended September 30, 2024, the following changes occurred:
- BlackRock increased its stake by 500,000 shares.
- FMR LLC acquired an additional 1,000,000 shares.
- Vanguard reduced its holdings by 250,000 shares.
Market Performance
As of September 30, 2024, the stock price of uniQure N.V. (QURE) was $2.50, reflecting a market capitalization of approximately $121.8 million. The performance over the past year has been characterized by significant volatility:
| Metric | Value |
|---|---|
| 52-week high | $5.00 |
| 52-week low | $1.50 |
| Year-to-date return | -35% |
| Average trading volume (last 3 months) | 200,000 shares |
Financial Overview
uniQure N.V. reported the following financial metrics for the nine months ended September 30, 2024:
| Metric | Value (in thousands) |
|---|---|
| Total revenues | $21,898 |
| Net loss | ($166,295) |
| Cash and cash equivalents | $436,700 |
| Accumulated deficit | ($1,056,704) |
Debt Obligations
As of September 30, 2024, uniQure N.V. had an outstanding loan amount of $50.0 million owed to Hercules Capital, Inc. The repayment schedule is as follows:
| Debt Type | Amount (in millions) | Maturity Date |
|---|---|---|
| Hercules Capital Loan | $50.0 | January 2027 |
uniQure N.V. (QURE) Mission Statement
Mission Overview
uniQure N.V. is dedicated to advancing gene therapies to treat severe diseases with significant unmet medical needs. The company focuses on developing innovative treatments for genetic disorders, aiming to provide long-term and potentially curative benefits through a single administration.
Core Values
- Innovation: Commitment to cutting-edge research and development in gene therapy.
- Patient-Centricity: Prioritizing the needs and experiences of patients in all aspects of their work.
- Integrity: Upholding the highest standards of ethics and transparency in their operations.
- Collaboration: Engaging with partners, stakeholders, and the community to enhance therapeutic outcomes.
Financial Goals
uniQure aims to achieve sustainable growth through strategic investments in research and development. As of September 30, 2024, the company reported:
| Financial Metric | Amount (in millions) |
|---|---|
| Cash and Cash Equivalents | $436.7 |
| Net Loss for Nine Months Ended September 30, 2024 | $(166.3) |
| Total Revenues for Nine Months Ended September 30, 2024 | $21.9 |
| Research and Development Expenses for Nine Months Ended September 30, 2024 | $(104.9) |
| Selling, General and Administrative Expenses for Nine Months Ended September 30, 2024 | $(41.3) |
Strategic Objectives
To realize its mission, uniQure has outlined several strategic objectives:
- Advance clinical trials for its gene therapy candidates, including AMT-130 for Huntington's disease and AMT-191 for Fabry disease.
- Enhance partnerships with organizations such as CSL Behring to expand market reach and operational capacity.
- Maintain a robust pipeline of innovative therapies targeting rare genetic diseases.
- Ensure financial sustainability through effective capital management and strategic funding initiatives.
Market Position
As of September 30, 2024, uniQure has established a strong presence in the gene therapy sector, with a focus on rare diseases. The company has a total asset value of $645.8 million and a significant accumulated deficit of $(1,056.7) million, reflecting its investment in long-term therapeutic development.
Recent Developments
uniQure's recent achievements include:
- FDA clearance for AMT-191's IND application in November 2023.
- First patient dosed in the Phase I/II clinical trial for AMT-191 as of August 2024.
- Net foreign currency gain of $5.4 million during the nine months ended September 30, 2024.
Funding and Financial Outlook
The company anticipates that its existing cash and cash equivalents will fund operations through 2027. Future funding may be necessary to advance AMT-130 and other product candidates into late-stage clinical development.
| Funding Source | Amount (in millions) |
|---|---|
| Loan Amount to Hercules Capital | $50.0 |
| Future Interest Payments and Financing Fees | $18.2 |
| Expected Cash Needs through 2027 | Based on current operating plans |
How uniQure N.V. (QURE) Works
Company Overview
uniQure N.V. is a leading gene therapy company based in the Netherlands, focused on innovative treatments for genetic diseases. The company specializes in developing gene therapies for conditions such as Huntington's disease, Fabry disease, and amyotrophic lateral sclerosis (ALS).
Financial Performance
As of September 30, 2024, uniQure reported the following financial metrics:
| Metric | 2024 (9 months) | 2023 (9 months) | Change |
|---|---|---|---|
| Total Revenues | $21.9 million | $9.2 million | $12.7 million |
| Operating Expenses | $163.9 million | $234.1 million | ($70.2 million) |
| Net Loss | ($166.3 million) | ($235.3 million) | $69.0 million |
| Cash and Cash Equivalents | $436.7 million | $617.9 million | ($181.2 million) |
| Accumulated Deficit | ($1,056.7 million) | ($890.4 million) | ($166.3 million) |
Revenue Breakdown
uniQure's revenue for the nine months ended September 30, 2024, compared to the same period in 2023, is summarized below:
| Revenue Source | 2024 | 2023 | Change |
|---|---|---|---|
| License Revenues | $5.2 million | $1.3 million | $3.9 million |
| Contract Manufacturing Revenues | $6.1 million | $6.6 million | ($0.5 million) |
| Collaboration Revenues | $10.6 million | $1.3 million | $9.3 million |
| Total Revenues | $21.9 million | $9.2 million | $12.7 million |
Research and Development Expenses
For the nine months ended September 30, 2024, uniQure incurred significant research and development expenses as detailed below:
| Program | 2024 (9 months) | 2023 (9 months) | Change |
|---|---|---|---|
| Huntington's disease (AMT-130) | $36.5 million | $67.9 million | ($31.4 million) |
| Fabry disease (AMT-191) | $3.6 million | $1.8 million | $1.8 million |
| ALS (AMT-162) | $4.5 million | $3.1 million | $1.4 million |
| Other R&D Expenses | $59.4 million | $99.4 million | ($40.0 million) |
| Total R&D Expenses | $104.9 million | $172.2 million | ($67.3 million) |
Debt and Financing
As of September 30, 2024, uniQure had the following debt obligations:
| Debt Type | Amount ($ million) | Maturity Date |
|---|---|---|
| Loan from Hercules Capital | $50.0 million | January 2027 |
| Royalty Financing Agreement | $426.7 million | N/A |
Liquidity
uniQure's liquidity position is summarized as follows:
| Metric | Amount ($ million) |
|---|---|
| Cash and Cash Equivalents | $436.7 million |
| Restricted Cash and Investment Securities | $436.7 million |
| Expected Runway | Through end of 2027 |
Key Developments
In October 2024, uniQure announced the first patient was dosed in a Phase I/IIa clinical trial of AMT-162 for the treatment of SOD1-ALS. The trial aims to evaluate safety, tolerability, and early signs of efficacy.
Additionally, AMT-191 received Orphan Drug Designation and Fast Track Designation from the FDA in September and October 2024, respectively.
Shareholder Equity
As of September 30, 2024, uniQure's shareholders' equity is as follows:
| Equity Component | Amount ($ million) |
|---|---|
| Ordinary Shares | $2.9 million |
| Additional Paid-In Capital | $1,166.3 million |
| Accumulated Other Comprehensive Loss | ($56.7 million) |
| Accumulated Deficit | ($1,056.7 million) |
| Total Shareholders' Equity | $55.8 million |
How uniQure N.V. (QURE) Makes Money
Revenue Streams
uniQure N.V. generates revenue through several key streams: license revenues, contract manufacturing revenues, and collaboration revenues. For the nine months ended September 30, 2024, the revenues were as follows:
| Type of Revenue | 2024 (in thousands) | 2023 (in thousands) | Change (in thousands) |
|---|---|---|---|
| License Revenues | $5,182 | $1,290 | $3,892 |
| Contract Manufacturing Revenues | $6,114 | $6,596 | ($482) |
| Collaboration Revenues | $10,602 | $1,268 | $9,334 |
| Total Revenues | $21,898 | $9,154 | $12,744 |
License Revenues
License revenues primarily stem from royalties associated with HEMGENIX® sales, under the agreement with CSL Behring. In the nine months ended September 30, 2024, uniQure recognized $5.2 million in license revenues, a significant increase from $1.3 million in the same period of 2023.
Contract Manufacturing Revenues
Contract manufacturing revenues are derived from manufacturing HEMGENIX® for CSL Behring, recognized upon the sale of the drug product. For the nine months ended September 30, 2024, these revenues totaled $6.1 million, slightly down from $6.6 million in 2023.
Collaboration Revenues
Collaboration revenues come from services provided to CSL Behring as per their agreement. In 2024, collaboration revenues surged to $10.6 million compared to just $1.3 million in 2023, driven by increased development and service activities related to the CSL Behring agreement.
Cost Structure
uniQure's operating expenses for the nine months ended September 30, 2024, totaled $163.9 million, compared to $234.1 million in 2023, reflecting a reduction of $70.2 million. The major components of operating expenses included:
| Type of Expense | 2024 (in thousands) | 2023 (in thousands) | Change (in thousands) |
|---|---|---|---|
| Cost of License Revenues | ($648) | $0 | ($648) |
| Cost of Contract Manufacturing | ($17,060) | ($4,793) | ($12,267) |
| Research and Development Expenses | ($104,942) | ($172,245) | $67,303 |
| Selling, General and Administrative Expenses | ($41,279) | ($57,103) | $15,824 |
| Total Operating Expenses | ($163,929) | ($234,141) | $70,212 |
Research and Development (R&D) Expenses
R&D expenses are a critical aspect of uniQure's business model, reflecting the company's commitment to developing gene therapies. For the nine months ended September 30, 2024, R&D expenses were $104.9 million, a decrease from $172.2 million in 2023.
Financial Position
As of September 30, 2024, uniQure had cash and cash equivalents, restricted cash, and investment securities totaling $436.7 million, down from $617.9 million at the end of 2023. The company reported a net loss of $166.3 million for the nine months ended September 30, 2024, compared to a net loss of $235.3 million in the same period of 2023.
Debt Obligations
uniQure had an outstanding loan amount of $50.0 million owed to Hercules Capital, Inc. as of September 30, 2024. The company repaid $50.0 million of the $100.0 million principal outstanding on July 19, 2024. Future interest payments and financing fees related to this loan total $18.2 million, with $6.5 million due within the next 12 months.
Future Outlook
Looking ahead, uniQure anticipates that its cash and cash equivalents will fund operations through the end of 2027, contingent on the successful commercialization of its product candidates. The company is exploring additional funding avenues to support late-stage clinical development for programs such as AMT-130 for Huntington's disease.
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Resources:
- uniQure N.V. (QURE) Financial Statements – Access the full quarterly financial statements for Q3 2024 to get an in-depth view of uniQure N.V. (QURE)' financial performance, including balance sheets, income statements, and cash flow statements.
- SEC Filings – View uniQure N.V. (QURE)' latest filings with the U.S. Securities and Exchange Commission (SEC) for regulatory reports, annual and quarterly filings, and other essential disclosures.