uniQure Announces 2021 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 2021 and
highlighted recent progress across its business.
“We made tremendous progress in 2021 across all
our strategic imperatives, including advancing our clinical-stage
programs, expanding our research pipeline, and preparing for the
submission of marketing applications planned for etranacogene
dezaparvovec during the first half of 2022,” stated Matt Kapusta,
chief executive officer at uniQure. “With positive data from the
HOPE-B pivotal study we look forward to working closely with our
partner, CSL Behring to bring this potentially life-changing gene
therapy to patients living with hemophilia B.”
“During 2022, we are also keenly focused on
maintaining our strong momentum in enrolling two ongoing Phase I/II
studies in Huntington’s disease. Importantly, we look forward to
sharing additional safety and biomarker data from all ten patients
in the lower dose cohort, including mutant HTT protein (mHTT) and
neurofilament light chain (NfL), during the second quarter of
2022,” he added. “We also are poised to advance our gene therapy
product candidates for Fabry disease and refractory temporal lobe
epilepsy into IND-enabling toxicology studies, and we expect to
initiate at least two new gene therapy programs targeting the liver
and CNS during the year. As we continue to advance and grow our
pipeline, we are also expanding our manufacturing footprint with a
second cGMP facility in Amsterdam, which we expect will come online
in 2022.”
Recent Key Accomplishments
- Advancing the late-stage
development of etranacogene dezaparvovec (AMT-061) for the
treatment of hemophilia B
- In December 2021, the Company and
CSL Behring announced the achievement of primary and secondary
endpoints from the HOPE-B pivotal trial of etranacogene
dezaparvovec in severe and moderately severe hemophilia B patients.
The primary endpoint of non-inferiority in annualized bleeding rate
(ABR) 18-months following administration compared to baseline
Factor IX (FIX) prophylactic therapy was achieved, as was a
secondary superiority endpoint on ABR.
- Data from the HOPE-B trial
demonstrated that etranacogene dezaparvovec produced mean FIX
activity of 39.0 percent of normal at six months and 36.9 percent
of normal at 18 months post infusion. After the six-month lead-in
period post-infusion, the adjusted annualized bleeding rate (ABR)
(1.51) for all bleeds was reduced by 64 percent (p=0.0002) and all
FIX-treated bleeds was reduced by 77 percent (3.65 to 0.83;
p<0.0001) over months seven to 18. In addition, 98 percent of
subjects treated with a full dose of etranacogene dezaparvovec
discontinued use of prophylaxis, with an overall 97 percent
reduction in mean unadjusted annualized FIX consumption of
257,338.8 IU/yr/participant to 8,486.6 IU/yr/participant (from
lead-in period to months 13-18). ABR for all bleeds after stable
FIX expression, assessed at 18 months, was 1.51 compared with the
ABR of 4.19 for the lead-in period of at least six months
(p=0.0002). ABR for investigator-adjudicated FIX-treated bleeds was
0.83 compared with lead-in ABR of 3.65 (p<0.0001).
- The Company and CSL Behring are
making significant progress on the preparation of marketing
applications for etranacogene dezaparvovec, which are expected to
be submitted by CSL Behring in the U.S. and the European Union
during the first half of 2022. As part of these efforts, the
Company successfully competed validation of its commercial
manufacturing process in December 2021.
- Advancing the clinical development
of AMT-130 for the treatment of Huntington’s disease
- In December 2021, initial
observations were announced on the first four patients in the
low-dose cohort of the double-blinded and randomized U.S. Phase
I/II study of AMT-130. AMT-130 was generally well tolerated with no
serious adverse events related to AMT-130. NfL increased as
expected immediately following the AMT-130 surgical procedure and
returned to baseline in the two treated patients. NfL remained
relatively constant in the two untreated control patients.
Structural magnetic resonance imaging did not reveal any clinically
meaningful safety findings in either treated or control patients at
one year of follow-up.
- During the second quarter of 2022,
the Company expects to provide a clinical update on all 10 patients
in the lower dose cohort, including safety, mHTT and NfL biomarker
data. Volumetric MRI and functional data is expected to be
available during the first half of 2023, after all patients in the
two dose cohorts are unblinded.
- Patient enrollment in a third
cohort is expected to begin in the second half of 2022 to explore
the use of alternative stereotactic navigation systems to simplify
placement of catheters for infusions of AMT-130. This cohort is
planned to include up to 18 additional randomized patients,
approximately 12 of whom will receive the higher dose of
6x1013 vg.
- In February 2022, the first two
patients were enrolled in a European open-label Phase Ib/II study
of AMT-130. This study will enroll 15 patients with early manifest
Huntington’s disease across the same doses being explored in the
U.S study. Together with the ongoing U.S. study, the European study
is intended to establish safety, proof of concept, and the optimal
dose of AMT-130 to take forward into Phase III development, or into
a confirmatory study should an accelerated registration pathway be
feasible.
- In total, 59 patients are expected
to be enrolled in the U.S. and European Phase 1/II trials of
AMT-130.
- Advancing our research pipeline
into the clinic
- Refractory Temporal Lobe Epilepsy
(TLE) - The Company expects to initiate a GLP toxicology study in
non-human primates in the second half of 2022, which is expected to
support an IND submission in 2023. AMT-260 employs a miRNA
silencing technology to suppress aberrantly expressed kainite
receptors in the hippocampus of patients with TLE. TLE represents a
large unmet clinical need affecting approximately 1.3 million
people in the U.S. and Europe alone, of which approximately 800,000
patients are unable to adequately control acute seizures with
currently approved anti-epileptic therapies.
- Fabry disease – The Company expects
to initiate a GLP toxicology study of AMT-191 by the middle of
2022, which is expected to support an IND submission in 2023.
AMT-191 is a one-time administered AAV5 gene therapy incorporating
an α-galactosidase A (GLA) transgene. In preclinical studies,
AMT-191 has shown the potential for cross correction in the kidney
and heart, and potentially into the brain. It also offers the
possibility for re-dosing as shown in a study of non-human
primates.
- With the goal of prioritizing
resources and accelerating the advancement of our programs in
refractory TLE and Fabry disease into the clinic, the Company has
deprioritized the preclinical development of AMT-150 in
spinocerebellar ataxia type 3.
- Expanding our manufacturing
capabilities
- The Company recently completed the
expansion of its Amsterdam facility providing additional
laboratories for new research and development activities, as well
as the construction of a cleanroom designed for the manufacturing
of cGMP materials at a 500-liter scale. This expansion now allows
for increased speed and agility across product development.
- Strong cash position to advance the
Company’s programs
- As of December 31, 2021, the
Company had cash and cash equivalents of $556.3 million. The
Company expects that its cash and cash equivalents will be
sufficient to fund operations into the first half of 2025 assuming
achievement of the BLA and MAA submission as well as first
commercial sales milestones under the CSL Behring
Agreement.
Upcoming Investor Events (each to be
conducted virtually)
- Cowen 42nd Annual Health Care
Conference, March 7-9, 2022
- Stifel 2022 CNS Days, March 28-29,
2022
- Guggenheim Genomic Medicines and
Rare Disease Day, March 31 & April 1, 2022
Financial Highlights
Cash Position: As of December
31, 2021, the Company held cash and cash equivalents of $556.3
million, compared to $244.9 million as of December 31, 2020. Upon
the CSL Behring Agreement becoming fully effective on May 6, 2021,
the Company received $462.4 million of payments. In January and
December 2021, the Company and Hercules amended the debt facility
agreement, under which the Company drew down an additional $35.0
million in January 2021 and a further $30.0 million for a total of
$100.0 million outstanding under the facility as of December 31,
2021. The Company also extended the maturity from June 2023 to
December 2025. In March and April 2021, the Company sold 921,730
ordinary shares for gross proceeds of approximately $29.6 million
under an Open Market Sale Agreement with SVB Leerink LLC. In July
2021, the Company paid a net EUR 42.1 million ($49.9 million)
related to its acquisition of Corlieve.
Revenues: Revenue for the year
ended December 31, 2021, was $524.0 million, compared to $37.5
million during the same period in 2020. The increase is a
result of $462.4 million of license revenue recognized upon closing
of the CSL Behring transaction in May 2021 as well as $55.0 million
of license revenue related to milestone payments the Company
expects to collect following the submissions of a biologics license
application (“BLA”) and market authorization application (“MAA”) by
CSL Behring in the first half of 2022. In 2020, the Company had
recognized $27.8 million in non-cash license revenue as of the
December 1, 2020, effective date of the amended Bristol-Myers
Squibb collaboration and license agreement as well as revenue of
$4.4 million in December 2020 following achievement of a research
milestone for one of the four Collaboration Targets.
R&D Expenses: Research and
development expenses were $143.5 million for the year ended
December 31, 2021, compared to $122.4 million during the same
period in 2020. The change was primarily related to recruitment of
personnel to support the development of product candidates,
advancing the clinical development of the Company’s Huntington’s
disease gene therapy program as well as increased activities
associated with preclinical product candidates, and an increase
from fair value changes related to liability recorded for
contingent consideration owed in relation to the acquisition of
Corlieve.
SG&A Expenses: Selling,
general and administrative expenses were $56.3 million for the year
ended December 31, 2021, compared to $42.6 million during the same
period in 2020. The change was primarily related to recruitment of
personnel, increased share-based compensation expenses, and as a
result of financial advisory payments made in relation to our
licensing transaction with CSL Behring.
Other Income, net: Other
income, net was $11.4 million for the year ended December 31, 2021,
compared to other income, net of $2.0 million during the same
period in 2020. The increase in other income, net was primarily
related to the receipts of employee retention credits under the
U.S. CARES Act, income related to payments received from European
authorities to subsidize research and development efforts in the
Netherlands and income related to a settlement agreement that the
Company and VectorY B.V. entered into in April 2021.
Other Non-operating Items, net:
Other non-operating income, net was income of $22.2 million for the
year ended December 31, 2021, compared to other non-operating loss
of $16.0 million for the same period in 2020. The increase in other
non-operating income was primarily related to net foreign currency
gains in the current period compared to net foreign currency losses
in the same period in 2020.
Net Income: The net income for
the year ended December 31, 2021, was $329.6 million, or $7.17
basic net income per ordinary share and $7.04 diluted net income
per ordinary share, compared to a loss of $125.0 million, or $2.81
basic and diluted loss per ordinary share during the same period in
2020.
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, refractory
temporal lobe epilepsy, Fabry disease, 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 re not limited to, whether we will bring
etranacogene dezaparvovec to patients, whether we will share mutant
HTT protein (mHTT) and neurofilament light chain (NfL) or other
safety and biomarker data from any patients in our AMT-130 clinical
trials, during the second quarter of 2022, the first half of 2023
or ever, whether we will be able to advance our gene therapy
product candidates for Fabry disease or refractory temporal lobe
epilepsy into IND-enabling toxicology studies, whether we will
initiate any new gene therapy programs targeting the liver or CNS
during the year, whether our second cGMP facility in Amsterdam will
come online in 2022 or ever, whether marketing applications for
etranacogene dezaparvovec, will be submitted in the U.S. or the
European Union during the first half of 2022 or ever.. The
Company’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, 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 the Company’s periodic securities filings,
including its Annual Report on Form 10-K filed February 25, 2022.
Given these risks, uncertainties and other factors, you should not
place undue reliance on these forward-looking statements, and the
Company assumes no obligation to update these forward-looking
statements, even if new information becomes available in the
future.
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uniQure
Contacts: |
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FOR
INVESTORS: |
|
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 |
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uniQure N.V.UNAUDITED
CONSOLIDATED BALANCE SHEETS
|
|
December 31, |
|
December 31, |
|
|
2021 |
|
2020 |
|
|
(in thousands, except share and per share
amounts) |
Current
assets |
|
|
|
|
Cash and cash equivalents |
|
$ |
556,256 |
|
$ |
244,932 |
Accounts receivable and
contract asset |
|
|
58,768 |
|
|
6,618 |
Prepaid expenses |
|
|
10,540 |
|
|
4,337 |
Other current assets and
receivables |
|
|
2,675 |
|
|
3,024 |
Total current
assets |
|
|
628,239 |
|
|
258,911 |
Non-current
assets |
|
|
|
|
Property, plant and equipment,
net |
|
|
43,505 |
|
|
32,328 |
Operating lease right-of-use
assets |
|
|
25,573 |
|
|
26,086 |
Intangible assets, net |
|
|
62,686 |
|
|
3,361 |
Goodwill |
|
|
27,633 |
|
|
542 |
Deferred tax assets, net |
|
|
15,647 |
|
|
16,419 |
Other non-current assets |
|
|
5,897 |
|
|
2,748 |
Total non-current
assets |
|
|
180,941 |
|
|
81,484 |
Total
assets |
|
$ |
809,180 |
|
$ |
340,395 |
Current
liabilities |
|
|
|
|
Accounts payable |
|
$ |
2,502 |
|
$ |
3,772 |
Accrued expenses and other
current liabilities |
|
|
28,487 |
|
|
18,038 |
Current portion of operating
lease liabilities |
|
|
5,774 |
|
|
5,524 |
Total current
liabilities |
|
|
36,763 |
|
|
27,334 |
Non-current
liabilities |
|
|
|
|
Long-term debt |
|
|
100,963 |
|
|
35,617 |
Operating lease liabilities,
net of current portion |
|
|
28,987 |
|
|
30,403 |
Contingent consideration |
|
|
29,542 |
|
|
- |
Deferred tax liability,
net |
|
|
12,913 |
|
|
- |
Other non-current
liabilities |
|
|
4,236 |
|
|
3,136 |
Total non-current
liabilities |
|
|
176,641 |
|
|
69,156 |
Total
liabilities |
|
|
213,404 |
|
|
96,490 |
Shareholders'
equity |
|
|
|
|
Total shareholders'
equity |
|
|
595,776 |
|
|
243,905 |
Total liabilities and
shareholders' equity |
|
$ |
809,180 |
|
$ |
340,395 |
uniQure N.V.UNAUDITED
CONSOLIDATED STATEMENTS OF OPERATIONS
|
|
Years ended December 31, |
|
|
2021 |
|
2020 |
|
2019 |
|
|
(in thousands, except share and per share
amounts) |
Total revenues |
|
$ |
524,002 |
|
$ |
37,514 |
|
$ |
7,281 |
Operating
expenses: |
|
|
|
|
|
|
Cost of contract revenues |
|
|
(24,976) |
|
|
- |
|
|
- |
Research and development
expenses |
|
|
(143,548) |
|
|
(122,400) |
|
|
(94,737) |
Selling, general and
administrative expenses |
|
|
(56,290) |
|
|
(42,580) |
|
|
(33,544) |
Total operating
expenses |
|
|
(224,814) |
|
|
(164,980) |
|
|
(128,281) |
Other income |
|
|
12,306 |
|
|
3,342 |
|
|
1,888 |
Other expense |
|
|
(876) |
|
|
(1,302) |
|
|
(2,028) |
Income / (loss) from
operations |
|
|
310,618 |
|
|
(125,426) |
|
|
(121,140) |
Non-operating items, net |
|
|
22,188 |
|
|
(16,017) |
|
|
(3,061) |
Income / (loss) before
income tax (expense) / income |
|
$ |
332,806 |
|
$ |
(141,443) |
|
$ |
(124,201) |
Income tax (expense) /
income |
|
|
(3,217) |
|
|
16,419 |
|
|
- |
Net income /
(loss) |
|
$ |
329,589 |
|
$ |
(125,024) |
|
$ |
(124,201) |
Earnings per ordinary
share - basic |
|
|
|
|
|
|
Basic net income / (loss) per ordinary share |
|
$ |
7.17 |
|
$ |
(2.81) |
|
$ |
(3.11) |
Earnings per ordinary
share - diluted |
|
|
|
|
|
|
Diluted net income / (loss) per ordinary share |
|
$ |
7.04 |
|
$ |
(2.81) |
|
$ |
(3.11) |
Weighted average shares -
basic |
|
|
45,986,467 |
|
|
44,466,365 |
|
|
39,999,450 |
Weighted average shares -
diluted |
|
|
46,840,972 |
|
|
44,466,365 |
|
|
39,999,450 |
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