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.
“Despite 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
• Advancing late-stage development of
etranacogene dezaparvovec (AMT-061) for the treatment of hemophilia
B
- In the first quarter, the Company
announced the achievement of targeted dosing of patients in the
HOPE-B pivotal trial of etranacogene dezaparvovec (AMT-061), an
investigational AAV5-based gene therapy incorporating the
patent-protected FIX-Padua variant for the treatment of patients
with severe and moderately severe hemophilia B. A total of 54
patients now have received the one-time dose of etranacogene
dezaparvovec.
- The Company remains on track to
provide 26-weeks of Factor IX data for all 54 patients in the
HOPE-B trial before the end of this year and to file an application
for marketing authorization in 2021.
- The manufacturing process
validation for etranacogene dezaparvovec is ongoing, and the
Company believes it is able to produce material at commercial
scale.
• Advancing AMT-130 into clinical
development for the treatment of Huntington’s
disease
- In March 2020, the Company
announced that the first two patients in the Phase I/II clinical
trial of AMT-130 in Huntington’s disease have been enrolled after
successfully meeting all screening and eligibility criteria. Due to
the impact of the coronavirus pandemic, the two procedures that
were scheduled to occur in late March have been temporarily
postponed.
- The Company continues its work to
resume treatment in the Phase I/II trial as soon as it is
clinically appropriate and will provide further updates on the
program at that time.
• Advancing research pipeline of gene
therapy candidates and the Company’s proprietary
technologies
- Earlier this month, the Company
announced its significant scientific presence at the upcoming
American Society of Gene and Cell Therapy Virtual Annual Meeting,
to be held May 12-15, 2020. In total, 22 data presentations will be
delivered by the Company’s researchers and scientists, including
five oral presentations.
- The data presentations will
highlight the Company’s industry-leading research and technology
capabilities and feature new preclinical findings on the Company’s
gene therapy candidates for hemophilia A, spinocerebellar ataxia
type 3 and Fabry disease.
• Strong cash position to advance the
Company’s programs
- As of March 31, 2020, the Company
held cash and cash equivalents of $342.0 million, which is expected
to be sufficient to fund the Company’s operations into 2022.
Upcoming Investor Events
• SunTrust Robinson Humphrey
(STRH) Virtual Life Sciences Summit, May 5 – 6, 2020.
• American Society of Gene and
Cell Therapy Virtual Annual Meeting, May 12 -15, 2020.
• Huntington's Disease
Regulatory Science Consortium Virtual Annual Meeting, May 18 – 19,
2020.
• Goldman Sachs Virtual Annual
Healthcare Conference, June 9 – 11, 2020.
• Credit Suisse 2020 Summer
Biotech Conference, June 22 -24, 2020.
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 Squibb (“BMS”).
R&D 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 candidates.
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.
About uniQure
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
This 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.
uniQure Contacts: |
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FOR INVESTORS: |
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FOR MEDIA: |
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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.cantor@uniQure.com |
e.mulder@uniQure.com |
t.malone@uniQure.com |
uniQure N.V.
UNAUDITED CONSOLIDATED BALANCE
SHEETS
|
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|
|
|
|
|
March
31, |
|
December
31, |
|
2020 |
|
2019 |
|
|
(in
thousands, except share and per share amounts) |
|
Current assets |
|
|
|
|
Cash and cash equivalents |
$ |
342,029 |
|
$ |
377,793 |
|
Accounts
receivable and accrued income from related party |
|
258 |
|
|
947 |
|
Prepaid
expenses |
|
5,738 |
|
|
4,718 |
|
Other
current assets |
|
1,576 |
|
|
748 |
|
Total current assets |
|
349,601 |
|
|
384,206 |
|
Non-current assets |
|
|
|
|
Property,
plant and equipment, net of accumulated depreciation of $29.8
million as of March 31, 2020 and $28.6 million as of December 31,
2019 respectively |
|
27,736 |
|
|
28,771 |
|
Operating
lease right-of-use assets |
|
26,288 |
|
|
26,797 |
|
Intangible
assets, net |
|
7,211 |
|
|
5,427 |
|
Goodwill |
|
486 |
|
|
496 |
|
Restricted
cash |
|
2,921 |
|
|
2,933 |
|
Total non-current assets |
|
64,642 |
|
|
64,424 |
|
Total assets |
$ |
414,243 |
|
$ |
448,630 |
|
Current liabilities |
|
|
|
|
Accounts
payable |
$ |
4,989 |
|
$ |
5,681 |
|
Accrued
expenses and other current liabilities |
|
9,507 |
|
|
12,457 |
|
Current
portion of operating lease liabilities |
|
5,900 |
|
|
5,865 |
|
Current
portion of deferred revenue |
|
6,732 |
|
|
7,627 |
|
Total current liabilities |
|
27,128 |
|
|
31,630 |
|
Non-current liabilities |
|
|
|
|
Long-term
debt |
|
36,209 |
|
|
36,062 |
|
Operating
lease liabilities, net of current portion |
|
30,518 |
|
|
31,133 |
|
Deferred
revenue, net of current portion |
|
23,713 |
|
|
23,138 |
|
Derivative
financial instruments related party |
|
1,004 |
|
|
3,075 |
|
Other
non-current liabilities |
|
524 |
|
|
534 |
|
Total non-current liabilities |
|
91,968 |
|
|
93,942 |
|
Total liabilities |
$ |
119,096 |
|
$ |
125,572 |
|
Total shareholders' equity |
|
295,147 |
|
|
323,058 |
|
Total liabilities and shareholders' equity |
$ |
414,243 |
|
$ |
448,630 |
|
|
|
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uniQure N.V.
UNAUDITED CONSOLIDATED STATEMENTS OF
OPERATIONS
|
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Three months ended March 31, |
|
|
2020 |
|
2019 |
|
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(in
thousands, except share and per share amounts) |
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Total revenues |
$ |
104 |
|
$ |
1,136 |
|
Operating expenses: |
|
|
|
|
Research and
development expenses |
|
(26,013) |
|
|
(20,537) |
|
Selling,
general and administrative expenses |
|
(9,072) |
|
|
(8,067) |
|
Total operating expenses |
|
(35,085) |
|
|
(28,604) |
|
Other
income |
|
857 |
|
|
313 |
|
Other
expense |
|
(339) |
|
|
(349) |
|
Loss
from operations |
|
(34,463) |
|
|
(27,504) |
|
Non
operating items, net |
|
6,464 |
|
|
(268) |
|
Net
loss |
$ |
(27,999) |
|
$ |
(27,772) |
|
|
|
|
|
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Basic and
diluted net loss per ordinary share |
$ |
(0.63) |
|
$ |
(0.74) |
|
Weighted
average shares used in computing basic and diluted net loss per
ordinary share |
|
44,279,456 |
|
|
37,676,172 |
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