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Dec 31, 2019
uniQure Q4 2019 Earnings Report
uniQure reported its financial results for 2019 and highlighted recent progress across its business.
Key Takeaways
uniQure reported a net loss of $124.2 million for the year ended December 31, 2019. The company held cash and cash equivalents of $377.8 million as of December 31, 2019, which is expected to fund operations into 2022.
Patient dosing nearly completed in Phase III HOPE-B trial of Etranacogene Dezaparvovec in Hemophilia B.
First patient procedure in Phase I/II clinical trial of AMT-130 in Huntington’s Disease expected to occur around the end of the first quarter of 2020.
Strong balance sheet expected to fund operations into 2022.
Expect to announce top-line data before the end of this year for Phase III HOPE-B study.
uniQure
uniQure
Forward Guidance
uniQure anticipates a transformational year in 2020 with new clinical data announcements and advancement of internally discovered investigational gene therapies.
Positive Outlook
- Full enrollment achieved in Phase III HOPE-B study of etranacogene dezaparvovec in hemophilia B.
- Top-line data from the HOPE-B study expected before the end of the year.
- Potential best-in-class profile for etranacogene dezaparvovec as a one-time treatment for hemophilia B.
- Patient screening successfully completed in the Phase I/II clinical trial of AMT-130 in Huntington’s disease.
- First procedure in the AMT-130 trial expected around the end of the first quarter of 2020.
Challenges Ahead
- Risk of cessation, delay, or lack of success of ongoing or planned clinical studies.
- Uncertainty regarding the ability to provide top-line data or any further clinical updates and data with respect to the pivotal Phase III study of AMT-061 before the end of 2020.
- Uncertainty regarding the ability to submit a BLA for marketing authorization of etranacogene dezaparvovec in 2021.
- Risks associated with clinical development activities, clinical results, collaboration arrangements, regulatory oversight, product commercialization, and intellectual property claims.
- Potential for actual results to differ materially from those anticipated in forward-looking statements.