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uniQure Announces 2019 Financial Results and Highlights Recent Company Progress ~ Patient Dosing Nearly Completed in Phase III HOPE-B Trial of E tranacogene Dezaparvovec in Hemophilia B ~ ~ First Patient Procedure in Pha

Key Takeaway: uniQure Announces 2019 Financial Results and Highlights Recent Company Progress Patient Dosing Nearly Completed in Phase III HOPE-B Trial of Etranacogene Dezaparvovec ~ First Patient Procedure in Phase I/II Clinical Trial of AMT-130 in Huntington's Disease Expected to Occur

Full Press Release Details

uniQure Announces 2019 Financial Results
and Highlights Recent Company Progress
Patient Dosing Nearly Completed in Phase III HOPE-B Trial of Etranacogene Dezaparvovec
~ 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
~ Strong Balance Sheet Expected to Fund
Operations into 2022 ~
MA and Amsterdam, the Netherlands, March 2, 2020 - uniQure N.V. (NASDAQ:
QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported
its financial results for 2019 and highlighted recent progress across its business.
past year was marked by strong execution in our pivotal, Phase III HOPE-B study of etranacogene dezaparvovec in
hemophilia B for which we achieved full enrollment and expect to announce top-line data before the end of this year," stated
Matt Kapusta, chief executive officer of uniQure. "We believe etranacogene
dezaparvovec has a potential best-in-class profile as a one-time treatment for patients with hemophilia
B, and our primary goal is to advance this program for regulatory review and potential approval."
"We have also successfully screened
patients in the randomized Phase I/II clinical trial of AMT-130 in Huntington's disease and expect the first procedure to
occur around the end of this first quarter of 2020," he added. "In addition, we have continued to advance our research
pipeline of innovative AAV gene therapy candidates and further build on our proprietary, commercial-scale manufacturing capabilities.
We expect this year to be a transformational one for uniQure in which we announce new clinical data and advance our internally
discovered investigational gene therapies."
Recent Company Progress
Upcoming Investor Events
Financial Highlights
Cash Position: As of December 31,
2019, the Company held cash and cash equivalents of $377.8 million, compared to $234.9 million as of December 31, 2018. The
Company currently expects cash and cash equivalents will be sufficient to fund operations into 2022.
Revenues: Revenue for the year ended
December 31, 2019 was $7.3 million, compared to $11.3 million for the year ended December 31, 2018. The decrease in 2019 reflects
the termination of the S100A1 program for the treatment of congestive heart failure under our collaboration with Bristol-Myers
Expenses: Research and development expenses were $94.7 million for the year ended December 31, 2019, compared to $74.8 million
for the year ended December 31, 2018. The change was primarily related to increased activities associated with our ongoing clinical
studies of etranacogene dezaparvovec, the recently initiated
Phase I/II study of AMT-130, increased share-based compensation and the hiring of additional clinical and operations staff at our
SG&A Expenses: Selling, general
and administrative expenses were $33.5 million for the year ended December 31, 2019, compared to $25.3 million for the year ended
December 31, 2018. The change was primarily related to increases in personnel and consulting expenses, professional fees and share-based
compensation expenses.
Other income, net: Other expense
was $0.1 million for 2019, compared to other income of $0.6 million for 2018.
Net Loss: The net loss for the full
years 2019 and 2018 was $124.2 million, or $3.11 per share and $83.3 million, or $2.34 per share, respectively.
uniQure is delivering on the promise of
gene therapy - single treatments with potentially curative results. We are leveraging our modular and validated technology
platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, hemophilia A,
Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. www.uniQure.com
uniQure Forward-Looking Statements
press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking
statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate,"
"expect," "goal," "intend," "look forward to", "may," "plan," "potential,"
"predict," "project," "should," "will," "would" and similar expressions. Forward-looking
statements are based on management's beliefs and assumptions and on information available to management only as of the date of
this press release. These forward-looking statements include, but are not limited to, the ability to provide top-line data or any
further clinical updates and data with respect to our pivotal Phase III study of AMT-061 before the end of 2020 or ever, the ability
to submit a BLA for marketing authorization of etranacogene dezaparvovec in 2021, or ever, the risk of cessation, delay or lack
of success of any ongoing or planned clinical studies, the ability to dose the first sentinel patient in the AMT-130 Phase
I/II clinical study by around the end of the first quarter of 2020, or ever, the ability to fund
operations into 2022, whether AMT-061 proves to have a best-in-class profile as a one-time treatment for patients with hemophilia,
whether 2020 will be a transformational year for uniQure in which new clinical data is announced or investigational gene therapies
are advanced, whether we can provide a safety update for AMT-130 this year, whether the Data Safety Monitoring Board for AMT-130
will convene later in 2021, whether important imaging and biomarker data from the AMT-130 trial will be obtained in 2021, or ever,
whether IND-enabling studies for Spinocerebellar Ataxia Type 3 (SCA3) are initiated in 2020, and whether an IND application for
SCA3 will be filed in 2021, or ever. Our actual results could differ materially from those anticipated in these forward-looking
statements for many reasons, including, without limitation, risks associated with our clinical development activities, clinical
results, collaboration arrangements, regulatory oversight, product commercialization and intellectual property claims, as well
as the risks, uncertainties and other factors described under the heading "Risk Factors" in uniQure's Annual
Report on Form 10-K filed on March 2, 2020. Given these risks, uncertainties and other factors, you should not place undue reliance
on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information
becomes available in the future.
FOR INVESTORS: FOR MEDIA:
Maria E. Cantor Eva M. Mulder Tom Malone
Direct: 339-970-7536 Direct: +31 20 240 6103 Direct: 339-970-7558
Mobile: 617-680-9452 Mobile: +31 6 52 33 15 79 Mobile:339-223-8541
m.canto@uniQure.com e.mulder@uniQure.com t.malone@uniQure.com
UNAUDITED CONSOLIDATED BALANCE SHEETS
UNAUDITED CONSOLIDATED STATEMENTS OF
Last updated: Mar 2, 2020