uniQure Announces 2020 Financial Results and Highlights Recent
Company Progress
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 2020 and
highlighted recent progress across its business.
“Despite unprecedented challenges in 2020, the
uniQure team demonstrated extraordinary resiliency and dedication
to the patients we serve, qualities which have defined our company
for more than two decades,” stated Matt Kapusta, chief executive
officer at uniQure. “I’m very proud of uniQure’s accomplishments,
including completing patient dosing in the HOPE-B pivotal trial,
presenting positive 26-week data on 54 patients that met the
study’s first primary endpoint, and announcing a landmark
transaction with CSL Behring for the global commercialization of
etranacogene dezaparvovec, which is expected to close in the second
quarter of 2021 following the U.S. antitrust review. We also became
the first company to initiate the clinical investigation of an AAV
gene therapy for Huntington’s disease and have now enrolled six
patients in the study.”
“Looking at 2021, we are focused on important
catalysts as we prepare the filings for regulatory approval of
etranacogene dezaparvovec and advance our Phase I/II study of
AMT-130 in Huntington’s disease, with early biomarker data on the
initial patients expected as early as the end of this year. We are
also making investments to expand our pipeline of promising gene
therapies and further strengthen our AAV manufacturing platform. We
look forward to hosting a Research and Development Day mid-year
during which we will unveil new research programs and other
technology initiatives.”
Recent Company Progress
- Advancing late-stage development of
etranacogene dezaparvovec (AMT-061) for the treatment of hemophilia
B
- In December 2020, initial 26-week
Factor IX data in 54 patients in the HOPE-B pivotal trial of
etranacogene dezaparvovec in severe to moderately severe hemophilia
B patients were presented in a late-breaker oral presentation at
the 62nd Annual Meeting of the American Society of Hematology
(ASH). The data met the first primary endpoint with mean FIX
activity of 37% of normal at 26 weeks. Patients achieved
significant increases in FIX activity irrespective of pre-existing
neutralizing antibodies. During the 26-week period after dosing,
patients reported an 83% reduction in total bleeding events. Only
three bleeding events were classified as spontaneous bleeds
requiring treatment, representing a reduction of 92% of such
bleeds. Mean annualized usage of FIX replacement therapy, a
secondary endpoint in the clinical trial, declined by 96%.
- The Company expects to present
top-line data related to additional co-primary endpoints, including
52-week annualized bleeding rates and FIX activity, during the
second quarter of 2021.
- In December 2020, the FDA placed
the Company’s hemophilia B program on clinical hold following the
report of a serious adverse event associated with a diagnosis
of hepatocellular carcinoma (HCC), a form of liver cancer, in one
patient in the HOPE-B trial. The analysis from the Company’s
ongoing investigation is expected to be completed shortly and
submitted to the FDA during the first quarter of 2021. uniQure
expects to meet with the FDA early in the second quarter of 2021 to
discuss the analysis and the status of the clinical hold, after
which the Company anticipates providing an update.
- In January 2021, the Company and
CSL Behring received requests for additional information by the
Federal Trade Commission (FTC) as part of its antitrust review of a
global licensing agreement for etranacogene dezaparvovec that was
announced in June 2020. The Company expects to fulfill the request
by the end of the first quarter of 2021 and, subject to completion
of the FTC review, close the transaction during the second quarter
of 2021.
- Advancing the clinical development
of AMT-130 for the treatment of Huntington’s disease
- In February 2021, the independent Data Safety Monitoring Board
recommended proceeding with patient enrollment in the Phase I/II
clinical trial of AMT-130 after finding no significant safety
concerns based on its review of the six-month data from the first
two enrolled patients and the 90-day data from the next two
enrolled patients.
- Two additional patient procedures were completed subsequent to
the February DSMB meeting and as of March 1, 2021, a total of six
patients have been enrolled in the study. The ten-patient cohort is
on track to be fully enrolled by mid-2021. Early
imaging and biomarker data from this first-in-human AAV-gene
therapy trial in Huntington’s disease are anticipated by year-end
2021.
- Expanding the Pipeline and
Strengthening the Platform
- The Company plans to host a virtual
Research & Development Day in mid-2021 to highlight its
expanding gene therapy pipeline, focusing on CNS disorders and
other rare, liver-directed disorders, as well as new advancements
in technology and manufacturing.
- In September 2020, the Company
initiated a safety and toxicology study of AMT-150, a novel gene
therapy for the treatment of Spinocerebellar Ataxia Type 3 (SCA3).
SCA3 patients experience brain degeneration that results in
movement disorders, rigidity, muscular atrophy and paralysis. There
is currently no treatment available that slows the progressive
course of this lethal disease. AMT-150 is a one-time administered
AAV gene therapy incorporating the Company’s proprietary miQURE™
silencing technology, similar to the technology used in the
Company’s AMT-130 program for the treatment for Huntington’s
disease, and is designed to halt ataxia in early manifest SCA3
patients.
- In September 2020, the Company
selected a lead gene therapy candidate (AMT-190) for the treatment
of Fabry disease to advance into IND-enabling studies. The lead
candidate is a one-time administered AAV5 gene therapy
incorporating an α-galactosidase A (GLA) transgene. In preclinical
studies comparing multiple product candidates, AMT-190 demonstrated
the most robust and sustained increases in GLA activity.
- Milestone achieved in research
collaboration with Bristol Myers Squibb
- In December 2020, the Company earned a $4.4 million milestone
payment from Bristol Myers Squibb for achieving a pre-specified
milestone under the companies’ collaboration and license agreement
(CLA) of 2015. The milestone payment is related to Bristol
Myers Squibb’s decision to select a gene therapy product candidate
for IND-enabling studies. Under the terms of the CLA, the
Company is entitled to receive up to $217.0 million in aggregate
milestone payments for each collaboration target, as well as
royalties on net product sales.
- Also, in December 2020, the Company and Bristol Myers Squibb
amended the CLA to limit Bristol Myers Squibb to four existing
collaboration targets. The amendment also terminated two warrants
previously granted to Bristol Myers Squibb to increase Bristol
Myers Squibb’s ownership in the Company up to 19.9%. The
companies agreed that upon the consummation of a change of control
transaction of the Company, uniQure will make a pre-specified
payment to Bristol Myers Squibb, subject to certain terms and
conditions.
- Strong cash position to advance the
Company’s programs
- In January 2021, the Company
amended its current debt facility with Hercules Capital from $35
million to $135 million and drew down $35 million of that
principal, bringing the total outstanding debt to $70 million.
- As of December 31, 2020, the
Company’s cash position was $244.9 million, which together with the
Company’s current debt facility, is expected to fund operations
into the second half of 2022. This does not include any financial
impact associated with the pending collaboration and license
agreement with CSL Behring. Assuming the receipt from CSL Behring
of the $450 million payment due at the closing of the transaction,
the Company expects cash and cash equivalents will be sufficient to
fund operations into the second half of 2024.
Upcoming Investor Events (each to be
conducted virtually)
- Cowen 41st Annual Health Care
Conference, March 1-4, 2021
- H.C. Wainwright Global Life Science
Conference, March 9-10, 2021
- Stifel CNS Day, March 31, 2021
- Guggenheim Healthcare Talks - 2021
Genomic Medicines & Rare Disease, April 1, 2021
- Wells Fargo Annual Biotech
Corporate Access Days, April 6 & 8, 2021
- 20th Annual Needham Virtual
Healthcare Conference, April 12-15, 2021
- Chardan 5th Annual Genetic
Medicines Manufacturing Summit, April 26-27, 2021
Financial Highlights
Cash Position: As of December
31, 2020, the Company held cash and cash equivalents of $244.9
million, compared to $377.8 million as of December 31,
2019.
Revenues: Revenue for the year
ended December 31, 2020 was $37.5 million, compared to $7.3 million
during the same period in 2019. The change was primarily
related to an increase in non-cash license revenue that we
recognized as of the December 1, 2020 effective date of the amended
Bristol-Myers Squibb collaboration and license agreement, as well
as from revenue that we recognized in December 2020 following
achievement of a research milestone for one of the four
Collaboration Targets.
R&D Expenses: Research and
development expenses were $122.4 million for the year ended
December 31, 2020, compared to $94.7 million during the same period
in 2019. The change was primarily related to increased activities
associated with our ongoing clinical studies of etranacogene
dezaparvovec and AMT-130, increased share-based compensation,
increased license expenses and the recruitment of personnel to
support the development of our product candidates.
SG&A Expenses: Selling,
general and administrative expenses were $42.6 million for the year
ended December 31, 2020, compared to $33.5 million during the same
period in 2019. The change was primarily related to increases in
personnel and consulting expenses to support our growth, as well as
increases in professional fees.
Other non-operating items, net:
Other expenses were $16.0 million for the year ended December 31,
2020, compared to other expenses of $3.1 million during the same
period in 2019 primarily related to foreign exchange results
related to changes in the exchange rates between the Euro and the
U.S. Dollar.
Net Loss: The net loss for the
year ended December 31, 2020 was $125.0 million, or $2.81 loss per
share, compared to $124.2 million, or $3.11 loss per share during
the same period in 2019.
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, 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, uniQure’s expectations
about its cash runway, the timing of submitting data to the FDA or
providing an update related to the investigation of any safety
event related to the HOPE-B study, the enrollment of patients in,
or Data Safety Monitoring Board review of, our Phase I/II gene
therapy clinical trial of AMT-130 in Huntington’s disease, the
timing of the announcement of data in our Phase I/II gene therapy
clinical trial of AMT-130, the timing of the announcement of data
in our HOPE-B pivotal trial of etranacogene dezaparvovec, the
timing of filing for regulatory approval of etranacogene
dezaparvovec, the timing of regulatory approval of our agreement
with CSL Behring, the potential financial compensation we may be
paid pursuant to our agreement with CSL Behring, our plans to
advance or expand our pipeline, accelerate research, identify
business development opportunities, invest in technology, or scale
our manufacturing capabilities. uniQure’s 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
Commercialization and License Agreement with CSL Behring, the
regulatory approval of that transaction, 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
periodic securities filings, including its Annual Report on Form
10-K filed March 1, 2021. Given these risks, uncertainties and
other factors, you should not place undue reliance on these
forward-looking statements, and uniQure assumes 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 |
Chiara Russo |
Tom Malone |
Direct: 339-970-7536 |
Direct: 617-306-9137 |
Direct: 339-970-7558 |
Mobile: 617-680-9452 |
Mobile: 617-306-9137 |
Mobile:339-223-8541 |
m.cantor@uniQure.com |
c.russo@uniQure.com |
t.malone@uniQure.com |
uniQure N.V.
UNAUDITED CONSOLIDATED BALANCE
SHEETS
|
|
|
|
|
|
December
31, |
|
December
31, |
|
2020 |
|
2019 |
|
|
|
|
|
(in
thousands, except share and per share amounts) |
|
Current assets |
|
|
|
|
Cash and cash equivalents |
$ |
244,932 |
|
$ |
377,793 |
|
Accounts
receivables |
|
6,618 |
|
|
- |
|
Accounts
receivable from related party |
|
- |
|
|
947 |
|
Prepaid
expenses |
|
4,337 |
|
|
4,718 |
|
Other
current assets |
|
3,024 |
|
|
748 |
|
Total current assets |
|
258,911 |
|
|
384,206 |
|
Non-current assets |
|
|
|
|
Property,
plant and equipment, net |
|
32,328 |
|
|
28,771 |
|
Operating
lease right-of-use assets |
|
26,086 |
|
|
26,797 |
|
Intangible
assets, net |
|
3,361 |
|
|
5,427 |
|
Goodwill |
|
542 |
|
|
496 |
|
Restricted
cash |
|
2,748 |
|
|
2,933 |
|
Deferred tax
asset |
|
16,419 |
|
|
- |
|
Total non-current assets |
|
81,484 |
|
|
64,424 |
|
Total assets |
$ |
340,395 |
|
$ |
448,630 |
|
Current liabilities |
|
|
|
|
Accounts
payable |
$ |
3,772 |
|
$ |
5,681 |
|
Accrued
expenses and other current liabilities |
|
18,038 |
|
|
12,457 |
|
Current
portion of operating lease liabilities |
|
5,524 |
|
|
5,865 |
|
Current
portion of deferred revenue |
|
- |
|
|
7,627 |
|
Total current liabilities |
|
27,334 |
|
|
31,630 |
|
Non-current liabilities |
|
|
|
|
Long-term
debt |
|
35,617 |
|
|
36,062 |
|
Operating
lease liabilities, net of current portion |
|
30,403 |
|
|
31,133 |
|
Deferred
revenue, net of current portion |
|
- |
|
|
23,138 |
|
Derivative
financial instruments related party |
|
- |
|
|
3,075 |
|
Other
non-current liabilities |
|
3,136 |
|
|
534 |
|
Total non-current liabilities |
|
69,156 |
|
|
93,942 |
|
Total liabilities |
$ |
96,490 |
|
$ |
125,572 |
|
Shareholders' equity |
|
|
|
|
Total shareholders' equity |
|
243,905 |
|
|
323,058 |
|
Total liabilities and shareholders' equity |
$ |
340,395 |
|
$ |
448,630 |
|
|
|
|
|
|
uniQure N.V.
UNAUDITED CONSOLIDATED STATEMENTS OF
OPERATIONS
|
|
|
|
|
|
|
|
|
|
Years ended December 31, |
|
|
|
2020 |
|
2019 |
|
2018 |
|
|
|
|
|
|
|
|
|
|
|
(in
thousands, except share and per share amounts) |
|
|
Total revenues |
$ |
37,514 |
|
|
$ |
7,281 |
|
|
$ |
11,284 |
|
|
|
Operating expenses: |
|
|
|
|
|
|
|
Research and
development expenses |
|
(122,400 |
) |
|
|
(94,737 |
) |
|
|
(74,809 |
) |
|
|
Selling,
general and administrative expenses |
|
(42,580 |
) |
|
|
(33,544 |
) |
|
|
(25,305 |
) |
|
|
Total operating expenses |
|
(164,980 |
) |
|
|
(128,281 |
) |
|
|
(100,114 |
) |
|
|
Other
income |
|
3,342 |
|
|
|
1,888 |
|
|
|
2,146 |
|
|
|
Other
expense |
|
(1,302 |
) |
|
|
(2,028 |
) |
|
|
(1,548 |
) |
|
|
Loss
from operations |
|
(125,426 |
) |
|
|
(121,140 |
) |
|
|
(88,232 |
) |
|
|
Non-operating items, net |
|
(16,017 |
) |
|
|
(3,061 |
) |
|
|
5,159 |
|
|
|
Loss
before income tax income / (expense) |
|
(141,443 |
) |
|
|
(124,201 |
) |
|
|
(83,073 |
) |
|
|
Income tax
income / (expense) |
|
16,419 |
|
|
|
- |
|
|
|
(231 |
) |
|
|
Net
loss |
$ |
(125,024 |
) |
|
$ |
(124,201 |
) |
|
$ |
(83,304 |
) |
|
|
|
|
|
|
|
|
|
|
Basic and
diluted net loss per ordinary share |
$ |
(2.81 |
) |
|
$ |
(3.11 |
) |
|
$ |
(2.35 |
) |
|
|
Weighted
average shares used in computing basic and diluted net loss per
ordinary share |
|
44,466,365 |
|
|
|
39,999,450 |
|
|
|
35,639,745 |
|
|
|
|
|
|
|
|
|
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