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 2023 and highlighted recent progress across its business.
“uniQure continued its momentum from a historic
2022 into the first quarter of 2023 as we make progress across all
of our corporate objectives,” stated Matt Kapusta, chief executive
officer of uniQure. “Our priorities for 2023 include supplying
CSL’s launch of HEMGENIX®, advancing the development of AMT-130 in
Huntington’s disease, and preparing for the initiation of two new
clinical studies in SOD1-ALS and refractory temporal lobe epilepsy.
Later in this second quarter of 2023, we look forward to providing
a clinical update from our ongoing U.S. Phase I/II study of
AMT-130, the first investigational AAV gene therapy in clinical
development for Huntington’s disease. This update will help to
further guide our clinical development of AMT-130 as we work
diligently to bring a disease-modifying treatment option to
patients with this devastating neurodegenerative
disease.”
Recent Updates
- Commercial launch 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 HEMGENIX® from uniQure in May 2021 and is
solely responsible for its commercialization. Following the first
commercial sale of HEMGENIX® in the United States, uniQure is
entitled to receive a $100.0 million payment from CSL Behring. The
Company is also eligible to receive a $75.0 million payment from
CSL Behring following the first product sale in one of five major
European countries if achieved prior to July 2, 2023.
- Advancing AMT-130 for the treatment
of Huntington’s disease
- The Company
plans to announce one to two-years of follow up data from its 26
patient, randomized, controlled and double-blinded U.S. Phase I/II
clinical study in the second quarter of 2023. The data update is
expected to include safety and tolerability, biomarker, imaging and
functional data across both high and low dose cohorts.
- Three of the six control patients
in the higher-dose cohort have now crossed over to treatment. Two
of these three patients have received the higher dose of AMT-130.
Control patients in the higher-dose cohort have the option to cross
over to treatment if they meet the study’s eligibility criteria
after unblinding. The company expects to complete any additional
crossover procedures by the end of the second quarter of 2023.
- All six
patients have been enrolled in the lower-dose cohort of the
European, open-label Phase I/II clinical trial of AMT-130. The
Company continues to enroll the second, higher-dose cohort of nine
patients. Patient enrollment in the higher-dose cohort is expected
to complete in mid- 2023 with one-year of clinical data from the
lower-dose cohort expected 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
- American Society of Gene and Cell
Therapy (ASGCT) 2023 – The company will have a major presence at
the annual ASGCT meeting being held in Los Angeles, CA from May
16-20, 2023. Twelve abstracts have been accepted for
presentation, including two oral presentations, that will feature
innovation in expanding the Company’s research pipeline and
advancements in AAV gene-therapy manufacturing.
- 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 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 the third quarter of
2022, the Company initiated a GLP toxicology study of AMT-260 in
non-human primates. The Company expects to submit an
investigational new drug (IND) 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 the third quarter of 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 March 31, 2023, the Company
had cash and cash equivalents and investment securities of $315.3
million. The Company expects cash and cash equivalents and
investment securities will fund operations into 2025 assuming the
achievement of $100.0 million of first commercial sale milestone in
the United States, 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.
Financial Highlights
Cash position: As of March 31,
2023, the Company held cash and cash equivalents and investment
securities of $315.3 million, compared to $392.8 million as of
December 31, 2022.
Revenues: Revenue for the three
months ended March 31, 2023 was $5.3 million, compared to $1.8
million in the same period in 2022. The increase is primarily a
result of contract manufacturing revenues of $4.9 million
recognized in the current period related to contract manufacturing
HEMGENIX® for CSL Behring partially offset by a decrease in
collaboration revenues of $1.4 million.
R&D expenses: Research and
development expenses were $60.8 million for the three months ended
March 31, 2023, compared to $45.0 million in the same period in
2022. The increase was primarily related to the $10.0 million
payment made to Apic Bio to acquire ABP-102, now AMT-162, 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 a licensor upon EMA approval of HEMGENIX®.
SG&A expenses: Selling,
general and administrative expenses were $17.8 million for the
three months ended March 31, 2023, compared to $11.0 million in the
same period in 2022. The increase was primarily related to an
increase in personnel and contractor related expenses to support
the growth of the Company and an increase in professional fees
incurred in the current period, primarily driven by an increase in
professional fees related to the Apic Bio transaction.
Other non-operating items, net:
Other non-operating items net was an expense of $4.3 million for
the three months ended March 31, 2023, compared to net income of
$6.8 million for the same period in 2022. The decrease in other
non-operating items, net was primarily related to recognizing
foreign currency losses, net of $2.4 million in the current period
compared to recognizing foreign currency gains, net of $8.6 million
in the prior period.
Net loss:The net loss for the
three months ended March 31, 2023, was $77.2 million, or $1.63
basic and diluted loss per ordinary share, compared to $46.7
million net loss for the same period in 2022, or $1.00 basic and
diluted loss 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,
whether there will be continued progress on the commercialization
of HEMGENIX®, 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 that data will help to further guide our ongoing
clinical development of AMT-130, 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 and the Quarterly Report on Form 10-Q filed May 9, 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 INVESTORS: |
|
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 |
|
|
|
|
|
|
March
31, |
|
December
31, |
|
|
2023 |
|
|
2022 |
|
|
(in
thousands, except share and per share amounts) |
|
Current assets |
|
|
|
|
Cash and
cash equivalents |
$ |
153,851 |
|
$ |
228,012 |
|
Current
investment securities |
|
161,487 |
|
|
124,831 |
|
Accounts
receivable and contract asset |
|
104,793 |
|
|
102,376 |
|
Inventories |
|
7,477 |
|
|
6,924 |
|
Prepaid
expenses |
|
12,567 |
|
|
11,817 |
|
Other
current assets and receivables |
|
2,468 |
|
|
2,814 |
|
Total current assets |
|
442,643 |
|
|
476,774 |
|
Non-current assets |
|
|
|
|
Property,
plant and equipment, net |
|
50,072 |
|
|
50,532 |
|
Non-current
investment securities |
|
- |
|
|
39,984 |
|
Operating
lease right-of-use assets |
|
32,135 |
|
|
32,726 |
|
Intangible
assets, net |
|
59,704 |
|
|
58,778 |
|
Goodwill |
|
25,998 |
|
|
25,581 |
|
Deferred tax
assets, net |
|
14,331 |
|
|
14,528 |
|
Other
non-current assets |
|
6,121 |
|
|
6,061 |
|
Total non-current assets |
|
188,361 |
|
|
228,190 |
|
Total assets |
$ |
631,004 |
|
$ |
704,964 |
|
Current liabilities |
|
|
|
|
Accounts
payable |
$ |
8,885 |
|
$ |
10,984 |
|
Accrued
expenses and other current liabilities |
$ |
22,529 |
|
$ |
30,571 |
|
Current
portion of contingent consideration |
|
27,253 |
|
|
25,982 |
|
Current
portion of operating lease liabilities |
|
7,669 |
|
|
8,382 |
|
Total current liabilities |
|
66,336 |
|
|
75,919 |
|
Non-current liabilities |
|
|
|
|
Long-term
debt |
|
103,253 |
|
|
102,791 |
|
Operating
lease liabilities, net of current portion |
|
31,075 |
|
|
31,719 |
|
Contingent
consideration, net of current portion |
|
9,641 |
|
|
9,334 |
|
Deferred tax
liability, net |
|
6,970 |
|
|
8,257 |
|
Other
non-current liabilities |
|
958 |
|
|
935 |
|
Total non-current liabilities |
|
151,897 |
|
|
153,036 |
|
Total liabilities |
|
218,233 |
|
|
228,955 |
|
Shareholders' equity |
|
|
|
|
Total shareholders' equity |
|
412,771 |
|
|
476,009 |
|
Total liabilities and shareholders' equity |
$ |
631,004 |
|
$ |
704,964 |
|
|
|
|
|
|
|
uniQure N.V.UNAUDITED CONSOLIDATED
STATEMENTS OF OPERATIONS |
|
|
Three months ended March 31, |
|
|
2023 |
|
|
|
2022 |
|
|
(in
thousands, except share and per share amounts) |
Total revenues |
$ |
5,325 |
|
|
$ |
1,792 |
|
Operating expenses: |
|
|
|
Cost of
contract manufacturing revenues |
|
(2,435 |
) |
|
|
- |
|
Research and
development expenses |
|
(60,809 |
) |
|
|
(45,003 |
) |
Selling,
general and administrative expenses |
|
(17,848 |
) |
|
|
(10,987 |
) |
Total operating expenses |
|
(81,092 |
) |
|
|
(55,990 |
) |
Other
income |
|
1,811 |
|
|
|
311 |
|
Other
expense |
|
(216 |
) |
|
|
(193 |
) |
Loss
from operations |
|
(74,172 |
) |
|
|
(54,080 |
) |
Non-operating items, net |
|
(4,262 |
) |
|
|
6,786 |
|
Loss
before income tax benefit / (expense) |
$ |
(78,434 |
) |
|
$ |
(47,294 |
) |
Income tax
benefit / (expense) |
|
1,207 |
|
|
|
616 |
|
Net
loss |
$ |
(77,227 |
) |
|
$ |
(46,678 |
) |
|
|
|
|
Basic and
diluted net loss per ordinary share |
$ |
(1.63 |
) |
|
$ |
(1.00 |
) |
Weighted
average shares used in computing basic and diluted net loss per
ordinary share |
|
47,436,335 |
|
|
|
46,599,114 |
|
|
|
|
|
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