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uniQure Announces First Quarter 2020 Financial Results and Highlights Recent Company Progress ~ Completed Target Patient Dosing in Phase III HOPE-B Trial of E tranacogene Dezaparvovec in Hemophilia B ~ ~ Announced 22 Pre

Key Takeaway: uniQure Announces First Quarter 2020 and Highlights Recent Company Progress Target Patient Dosing in Phase III HOPE-B Trial of Etranacogene Dezaparvovec ~ Announced 22 Presentations at Upcoming ASGCT Virtual Annual Meeting ~ MA and Amsterdam, the Netherlands, April 29, 2020

Full Press Release Details

uniQure Announces First Quarter 2020
and Highlights Recent Company Progress
Target Patient Dosing in Phase III HOPE-B Trial of Etranacogene Dezaparvovec
~ Announced 22 Presentations at Upcoming
ASGCT Virtual Annual Meeting ~
MA and Amsterdam, the Netherlands, April 29, 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 the first quarter of 2020 and highlighted recent progress across its business.
the ongoing global crisis caused by the coronavirus pandemic, we ended the first quarter of 2020 in a position of strength and
remain well capitalized," stated Matt Kapusta, chief executive officer of
uniQure. "I am very proud of the continued dedication of our employees in both the United States and the Netherlands, and
thank them for the outstanding work they continue to do in driving forward our clinical programs, research pipeline, and manufacturing
and technology operations. We are well positioned for an important second half of the year when we expect to announce new clinical
data and advancements from across the business."
Recent Company Progress
Upcoming Investor Events
Financial Highlights
Cash Position: As of March 31, 2020,
the Company held cash and cash equivalents of $342.0 million, compared to $377.8 million as of December 31, 2019. The Company
currently expects cash and cash equivalents will be sufficient to fund operations into 2022.
Revenues: Revenue for the three
months ended March 31, 2020 was $0.1 million, compared to $1.1 million for the same period 2019. The decrease in 2020 reflects
the reduction of activities following the end of the initial Research Term in May 2019 of our collaboration with Bristol-Myers
Expenses: Research and development expenses were $26.0 million for the three months ended March 31, 2020, compared to $20.5
million for the same period 2019. The change was primarily related to increased activities associated with our ongoing clinical
studies of etranacogene dezaparvovec, the Phase I/II trial
of AMT-130, increased share-based compensation and the additional recruitment of personnel to support the development of product
SG&A Expenses: Selling, general
and administrative expenses were $9.1 million for the three months ended March 31, 2020, compared to $8.1 million for the same
period 2019. The change was primarily related to increases in personnel and contractor related expenses.
Other income, net: Other income
was $6.5 million for the three months ended March 31, 2020, compared to other expense of $0.3 million for the same period 2019,
primarily reflecting foreign currency gains and a gain related to the change in fair value of the BMS warrants.
Net Loss: The net loss for the three
months ended March 31, 2020 and March 31, 2019, was $28.0 million, or $0.63 loss per share and $27.8 million, or $0.74 loss 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, our ability to provide top-line data or any
further clinical updates and data with respect to our pivotal Phase III trial of AMT-061 before the end of 2020 or ever, our ability
to submit a BLA for marketing authorization of etranacogene dezaparvovec in 2021, or ever, our ability to resume treatment or to
dose the first two patients in the AMT-130 Phase I/II clinical trial, our ability
to fund operations into 2022, and our ability to announce new clinical data and advancements from across the business later in
2020. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including,
without limitation, risks associated with the impact of the ongoing COVID-19 pandemic on our Company and the wider economy and
health care system, 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 our Quarterly Report on Form 10-Q filed on April 29, 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 Direct: 339-970-7536 Mobile: 617-680-9452 m.cantor@uniQure.com Eva M. Mulder Direct: +31 20 240 6103 Mobile: +31 6 52 33 15 79 e.mulder@uniQure.com Tom Malone Direct: 339-970-7558 Mobile:339-223-8541 t.malone@uniQure.com
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Last updated: Apr 29, 2020