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Dec 31, 2020
uniQure Q4 2020 Earnings Report
uniQure reported its financial results and highlighted recent progress across its business.
Key Takeaways
uniQure reported its financial results for 2020, including a revenue of $37.5 million and a net loss of $125.0 million. The company completed patient dosing in the HOPE-B pivotal trial and announced a transaction with CSL Behring for the commercialization of etranacogene dezaparvovec.
Completed six patient procedures in Phase I/II for Huntington’s disease, with full enrollment of first cohort expected mid-2021.
Last patient in HOPE-B pivotal study to complete 52-week follow-up visit before end of the first quarter of 2021, with top-line data to be presented in the second quarter of 2021.
Analysis of the HOPE-B safety event expected to be completed and submitted to the FDA before end of the first quarter of 2021.
Research & Development Day to be held mid-2021 to announce expanded pipeline and platform advancements.
uniQure
uniQure
Forward Guidance
uniQure is focused on important catalysts as it prepares the filings for regulatory approval of etranacogene dezaparvovec and advance its Phase I/II study of AMT-130 in Huntington’s disease.
Positive Outlook
- Preparing filings for regulatory approval of etranacogene dezaparvovec.
- Advancing Phase I/II study of AMT-130 in Huntington’s disease.
- Early biomarker data on the initial patients expected as early as the end of this year.
- Making investments to expand pipeline of promising gene therapies.
- Further strengthening AAV manufacturing platform.
Challenges Ahead
- Risks associated with the impact of the ongoing COVID-19 pandemic.
- Risks related to Commercialization and License Agreement with CSL Behring.
- Risks related to the regulatory approval of that transaction.
- Risks related to clinical development activities.
- Risks related to collaboration arrangements.