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 2022 and
highlighted recent progress across its business.
“We had a strong ending to 2022 with the
historic approval of the world’s first gene therapy for hemophilia
B for which we led the multi-year research and clinical
development, and through our partnership with CSL Behring,
HEMGENIX® will now be available to patients in the United States
and European Union,” stated Matt Kapusta, chief executive officer
of uniQure. “We are immensely proud of our efforts that led to this
product approval, and of the innovation and leadership in genomic
medicine that it represents. At the same time, we are working with
urgency to advance AMT-130, the first AAV gene therapy being
clinically investigated in Huntington’s disease, and are on track
to provide a clinical update from our U.S. Phase I/II study in the
second quarter of 2023. We are also preparing for the initiation of
two new Phase I/II studies in refractory temporal lobe epilepsy and
SOD1-ALS in the second half of this year, and readying for the
submission of an investigational new drug application in Fabry
disease in 2023.”
“Throughout this progress, we have established
cutting-edge capabilities in the field of genetic medicines – from
how we discover and develop our gene therapies, to how we
successfully navigate complex clinical and regulatory pathways, to
how we manufacture these novel gene therapies globally for clinical
and commercial usage,” said Mr. Kapusta. “We believe these
capabilities position us well for continued growth and success as
we look forward to developing more life-altering gene therapy
products for patients in need.”
Recent Updates
- Preparing global commercial supply
of HEMGENIX® (etranacogene dezaparvovec-drbl) for
the treatment of hemophilia B in the United States and European
Union
- In February 2023, the European
Commission granted conditional marketing approval for HEMGENIX®,
the first and only one-time gene therapy for the treatment of
adults 18 years of age and older living with severe and moderately
severe hemophilia B. This follows the November 2022 U.S. marketing
approval of HEMGENIX® in adults living with hemophilia B.
- CSL Behring licensed the exclusive
global rights to etranacogene dezaparvovec from uniQure in May
2021 and is now solely responsible for the further development,
registration, and commercialization of the therapy. uniQure is
responsible for the global commercial supply of etranacogene
dezaparvovec and manufactures the product at its cGMP facility in
Lexington, MA.
- uniQure is eligible to receive a
$100.0 million payment from CSL Behring following the first sale of
HEMGENIX® in the United States, as well as a $75.0 million payment
from CSL Behring following the first product sale in one of five
major European countries if prior to July 2, 2023.
- Advancing AMT-130 for the treatment
of Huntington’s disease
All 26 patients have been enrolled in the first two cohorts of
the randomized, controlled and double-blinded U.S. Phase I/II study
of AMT-130, including 10 patients in the lower-dose cohort (6
treated patients and 4 control patients) and 16 patients in the
higher-dose cohort (10 treated patients and 6 control patients).
The 6 control patients in the higher-dose cohort will have the
option to cross over to treatment if they meet the study’s
eligibility criteria. In the first quarter of 2023, a second
control patient was crossed over to treatment and received the
higher dose of AMT-130.
- The Company
plans to announce one to two-years of follow up data from the U.S.
Phase I/II clinical study in the second quarter of 2023. The data
update is expected to include safety and tolerability, biomarker,
functional and imaging data across both dose cohorts.
- Patient
enrollment in the European, open-label Phase I/II clinical trial of
AMT-130 is expected to be complete in the first half of 2023.
Fifteen patients across the two dose cohorts will be treated with
AMT-130, including six patients receiving the lower dose and nine
patients receiving the higher dose. Clinical data from the
lower-dose cohort is expected to be presented in the second half of
2023.
- A third cohort of patients is
expected to begin enrollment in the U.S. in the second half of 2023
to explore the feasibility of certain surgical adaptations aimed at
enhancing procedure efficiency.
- Expanding the Pipeline and Progress
Towards Investigational New Drug (IND) Applications
- AMT-162 for the treatment of SOD1
amyotrophic lateral sclerosis (ALS) - In January 2023, the Company
entered into a global licensing agreement with Apic Bio for
ABP-102, now AMT-162 for the treatment of superoxide dismutase 1
(SOD1) amyotrophic lateral sclerosis (ALS), a rare, genetic form of
ALS. The licensing of AMT-162 enhances the Company’s CNS pipeline
and is highly complementary to AMT-161, a gene therapy candidate
for ALS caused by mutations in the c9orf72 gene, allowing the
Company to potentially address most inherited forms of ALS. AMT-162
has been granted both Orphan Drug and Fast Track designations and
has a cleared IND. The Company expects to initiate a Phase I/II
clinical study of AMT-162 in the second half of 2023.
- AMT-260 for the treatment of
refractory temporal lobe epilepsy (rTLE) – In November, 2022, the
Company hosted an investor research and development event that
explained the unmet medical need in rTLE and the impact of disease
on patients, along with preclinical data on AMT-260 and plans for
its early stage clinical development. The Company expects to submit
an investigational new drug application and initiate a Phase I/II
clinical study of AMT-260 in the second half of 2023.
- AMT-191 for the treatment of Fabry
disease – In August 2022, the Company initiated a GLP toxicology
study of AMT-191 in non-human primates which is expected to support
an IND submission in 2023.
- Strong cash position to advance the
Company’s programs
- As of December 31, 2022, the
Company had cash and cash equivalents and investment securities of
$392.8 million. The Company expects cash and cash equivalents and
investment securities will fund operations into 2025 assuming the
achievement of $100.0 million of a first commercial sale milestone
for HEMGENIX® in the U.S. and into the first half of 2025 if the
$75.0 million first commercial sale milestone for HEMGENIX® in any
of the five contractually defined European countries is achieved
prior to July 2, 2023.
Upcoming Investor Events
- Cowen 43rd Annual Health Care
Conference, Monday, March 6, 2023, Boston
- Stifel Virtual 2023 CNS Days,
Tuesday, March 28, 2023
- Guggenheim Virtual Healthcare
Talks: Genomic Medicines and Rare Disease, Tuesday, April 4,
2023
Financial Highlights
Cash position: As of December
31, 2022, the Company held cash and cash equivalents and investment
securities of $392.8 million, compared to $556.3 million as of
December 31, 2021. As of December 31, 2022, the Company held
investments in debt securities of $164.8 million, compared to nil
as of December 31, 2021. The investments in debt securities have
remaining maturities ranging from three to 14 months.
Revenues: Revenue for the year
ended December 31, 2022 was $106.5 million, compared to $524.0
million in the same period in 2021. In 2022, the Company recognized
$100.0 million of license revenue related to a milestone payment
the Company expects to receive following the first sale of HEMGENIX
in the U.S. In 2021, the Company recognized $462.4 million of
license revenue upon closing of the CSL Behring transaction in May
2021 as well as $55.0 million of license revenue for milestone
payments related to CSL Behring’s BLA and MAA submissions in the
first months of 2022.
R&D expenses: Research and
development expenses were $197.6 million for the year ended
December 31, 2022, compared to $143.5 million during the same
period in 2021. The increase was primarily related to advancing the
clinical development of the Company’s Huntington’s disease gene
therapy program, the preclinical development of the temporal lobe
epilepsy program (AMT-260), an increase in personnel and contractor
related expenses to support the growth of the Company, and
contractual payments owed to licensors upon FDA approval of
HEMGENIX and a valid patent claim granted within the EU.
SG&A expenses: Selling,
general and administrative expenses were $55.1 million for the year
ended December 31, 2022, compared to $56.3 million during the same
period in 2021. The increase in personnel and contractor related
expenses to support the growth of the Company was offset by a
reduction in professional and financial advisory fees we had
incurred in 2021 related to the closing of the CSL Behring
transaction.
Other non-operating items, net:
Other non-operating income, net was income of $14.9 million for the
year ended December 31, 2022, compared to other non-operating
income, net of $22.2 million for the same period in 2021. The
decrease in other non-operating income, net was primarily related
to a decrease in net foreign currency gains of $6.4 million, an
increase of $3.8 million in interest expense related to the
long-term debt with Hercules Capital, Inc. and a $2.9 million
increase in other non-operating gains as a result of releasing a
financial liability.
Net loss:The net loss for the
year ended December 31, 2022, was $126.8 million, or $2.71 basic
and diluted loss per ordinary share, compared to $329.6 million net
income for the same period in 2021, or $7.17 basic net income per
ordinary share and $7.04 diluted net income per ordinary
share.
About
HEMGENIX®
HEMGENIX® is a gene therapy that reduces the
rate of abnormal bleeding in eligible people with hemophilia B by
enabling the body to continuously produce factor IX, the deficient
protein in hemophilia B. It uses AAV5, a non-infectious viral
vector, called an adeno-associated virus (AAV). The AAV5 vector
carries the Padua gene variant of Factor IX (FIX-Padua) to the
target cells in the liver, generating factor IX proteins that are
5x-8x more active than normal. These genetic instructions remain in
the target cells, but generally do not become a part of a person’s
own DNA. Once delivered, the new genetic instructions allow
the cellular machinery to produce stable levels of factor IX.
HEMGENIX® is a registered trademark of CSL
Behring.
About uniQure
uniQure is delivering on the promise of gene
therapy – single treatments with potentially curative results. The
recent approvals of our gene therapy for hemophilia B – an historic
achievement based on more than a decade of research and clinical
development – represents a major milestone in the field of genomic
medicine and ushers in a new treatment approach for patients living
with hemophilia. We are now leveraging our modular and validated
technology platform to advance a pipeline of proprietary gene
therapies for the treatment of patients with Huntington's disease,
refractory temporal lobe epilepsy, ALS, Fabry disease, and other
severe 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," “establish,” "estimate,"
"expect," "goal," "intend," "look forward to", "may," "plan,"
"potential," "predict," "project," “seek,” "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,
statements about whether a third cohort of patients in our AMT-130
clinical trial for Huntington’s Disease will begin enrollment in
the U.S. in the second half of 2023, whether the Company will
announce one to two-years of follow up data from the U.S. Phase
I/II clinical study of AMT-130 in the second quarter of 2023,
whether clinical data from the lower-dose cohort for AMT-130 is
expected to be presented in the second half of 2023, whether the
Company will submit an investigational new drug application for
Fabry disease in 2023, whether the company will initiate a Phase
I/II clinical study of AMT-162 for the treatment of ALS in the
second half of 2023, and whether the Company will submit an
investigational new drug application or initiate a Phase I/II
clinical study of AMT-260 for rTLE in the second half of 2023. 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 regulatory
approval and commercial launch of HEMGENIX®, our clinical trial for
Huntington’s disease, the impact of financial and geopolitical
events 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 27, 2023. 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.
uniQure Contacts: |
|
FOR MEDIA: |
|
|
|
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, |
|
|
2022 |
|
|
2021 |
|
|
(in
thousands, except share and per share amounts) |
|
Current assets |
|
|
|
|
Cash and
cash equivalents |
$ |
228,012 |
|
$ |
556,256 |
|
Current
investment securities |
|
124,831 |
|
|
— |
|
Accounts
receivable and contract asset |
|
102,376 |
|
|
58,768 |
|
Inventories |
|
6,924 |
|
|
— |
|
Prepaid
expenses |
|
11,817 |
|
|
10,540 |
|
Other
current assets and receivables |
|
2,814 |
|
|
2,675 |
|
Total current assets |
|
476,774 |
|
|
628,239 |
|
Non-current assets |
|
|
|
|
Property,
plant and equipment, net |
|
50,532 |
|
|
43,505 |
|
Non-current
investment securities |
|
39,984 |
|
|
— |
|
Operating
lease right-of-use assets |
|
32,726 |
|
|
25,573 |
|
Intangible
assets, net |
|
58,778 |
|
|
62,686 |
|
Goodwill |
|
25,581 |
|
|
27,633 |
|
Deferred tax
assets, net |
|
14,528 |
|
|
15,647 |
|
Other
non-current assets |
|
6,061 |
|
|
5,897 |
|
Total non-current assets |
|
228,190 |
|
|
180,941 |
|
Total assets |
$ |
704,964 |
|
$ |
809,180 |
|
Current liabilities |
|
|
|
|
Accounts
payable |
$ |
10,984 |
|
$ |
2,502 |
|
Accrued
expenses and other current liabilities |
$ |
30,571 |
|
$ |
28,487 |
|
Current
portion of contingent consideration |
|
25,982 |
|
|
— |
|
Current
portion of operating lease liabilities |
|
8,382 |
|
|
5,774 |
|
Total current liabilities |
|
75,919 |
|
|
36,763 |
|
Non-current liabilities |
|
|
|
|
Long-term
debt |
|
102,791 |
|
|
100,963 |
|
Operating
lease liabilities, net of current portion |
|
31,719 |
|
|
28,987 |
|
Contingent
consideration, net of current portion |
|
9,334 |
|
|
29,542 |
|
Deferred tax
liability, net |
|
8,257 |
|
|
12,913 |
|
Other
non-current liabilities |
|
935 |
|
|
4,236 |
|
Total non-current liabilities |
|
153,036 |
|
|
176,641 |
|
Total liabilities |
|
228,955 |
|
|
213,404 |
|
Shareholders' equity |
|
|
|
|
Total shareholders' equity |
|
476,009 |
|
|
595,776 |
|
Total liabilities and shareholders' equity |
$ |
704,964 |
|
$ |
809,180 |
|
|
|
|
|
|
|
|
|
|
|
Balancing
check |
|
- |
|
|
- |
|
uniQure N.V.
UNAUDITED CONSOLIDATED STATEMENTS OF
OPERATIONS
|
|
|
|
|
|
|
Years ended December 31, |
|
|
2022 |
|
|
|
2021 |
|
|
|
2020 |
|
|
(in
thousands, except share and per share amounts) |
Total revenues |
$ |
106,483 |
|
|
$ |
524,002 |
|
|
$ |
37,514 |
|
Operating expenses: |
|
|
|
|
|
Cost of
license revenues |
|
(1,254 |
) |
|
|
(24,976 |
) |
|
|
- |
|
Cost of
contract manufacturing revenues |
|
(2,089 |
) |
|
|
- |
|
|
|
- |
|
Research and
development expenses |
|
(197,591 |
) |
|
|
(143,548 |
) |
|
|
(122,400 |
) |
Selling,
general and administrative expenses |
|
(55,059 |
) |
|
|
(56,290 |
) |
|
|
(42,580 |
) |
Total operating expenses |
|
(255,993 |
) |
|
|
(224,814 |
) |
|
|
(164,980 |
) |
Other
income |
|
7,171 |
|
|
|
12,306 |
|
|
|
3,342 |
|
Other
expense |
|
(820 |
) |
|
|
(876 |
) |
|
|
(1,302 |
) |
(Loss) / income from operations |
|
(143,159 |
) |
|
|
310,618 |
|
|
|
(125,426 |
) |
Non-operating items, net |
|
14,900 |
|
|
|
22,188 |
|
|
|
(16,017 |
) |
(Loss) / income before income tax benefit /
(expense) |
$ |
(128,259 |
) |
|
$ |
332,806 |
|
|
$ |
(141,443 |
) |
Income tax
benefit / (expense) |
|
1,470 |
|
|
|
(3,217 |
) |
|
|
16,419 |
|
Net
(loss) / income |
$ |
(126,789 |
) |
|
$ |
329,589 |
|
|
$ |
(125,024 |
) |
Earnings per ordinary share - basic |
|
|
|
|
|
Basic net (loss) / income per ordinary share |
$ |
(2.71 |
) |
|
$ |
7.17 |
|
|
$ |
(2.81 |
) |
Earnings per ordinary share - diluted |
|
|
|
|
|
Diluted net (loss) / income per ordinary share |
$ |
(2.71 |
) |
|
$ |
7.04 |
|
|
$ |
(2.81 |
) |
Weighted
average shares - basic |
|
46,735,045 |
|
|
|
45,986,467 |
|
|
|
44,466,365 |
|
Weighted
average shares - diluted |
|
46,735,045 |
|
|
|
46,840,972 |
|
|
|
44,466,365 |
|
|
|
|
|
|
|
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