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 second quarter
of 2024 and highlighted recent progress across its business.
“We have made significant progress over the past
few months across several key business objectives, including
advancing our clinical pipeline and taking important actions to
considerably reduce our capital requirements,” stated Matt Kapusta,
chief executive officer of uniQure. “With the recent Regenerative
Medicine Advanced Therapy (RMAT) designation and the latest interim
data supporting dose-dependent slowing of Huntington’s disease
progression, we are eager to engage further with regulators to
pursue an expedited clinical development pathway for AMT-130. We’ve
also made meaningful progress across our three other clinical
programs, with patient screenings underway and enrollment expected
to begin shortly. At the same time, we’ve taken targeted measures
to substantially reduce operating expenses, streamline operations,
and extend cash runway. These actions are designed to ensure we
have the funding required to achieve key milestones and drive
shareholder value, as we endeavor to deliver transformative
medicines to patients in need.”
Recent Company Updates
- Advancing AMT-130 for the treatment
of Huntington’s disease
- In June 2024, the U.S. Food and
Drug Administration (FDA) granted Regenerative Medicine Advanced
Therapy (RMAT) designation for investigational gene therapy
AMT-130, becoming the first therapeutic candidate to receive such a
designation for Huntington’s disease. RMAT designation allows
sponsor companies to have early, close and frequent interactions
with the FDA.
- In July 2024, uniQure announced
positive interim data from the ongoing U.S. and European Phase I/II
studies of AMT-130 for the treatment of early-stage Huntington’s
disease.1
- A statistically significant,
dose-dependent slowing in disease progression measured by the
composite Unified Huntington’s Disease Rating Scale (cUHDRS) was
observed through 24 months in patients receiving the high dose of
AMT-130 compared to a propensity score-weighted external control
(p=0.007).
- A statistically significant
reduction of neurofilament light chain (NfL) in cerebrospinal fluid
(CSF) was observed at 24 months in patients treated with AMT-130
compared to baseline (p=0.02).
- AMT-130 continued to be generally
well-tolerated with a manageable safety profile across both
doses.
- The Company expects to hold a Type
B, multi-disciplinary RMAT meeting with the FDA in the second half
of 2024 with the goal of defining the future clinical and
regulatory pathway for AMT-130.
- Patient dosing is ongoing in a
third cohort of up to 12 patients to further evaluate both doses of
AMT-130 in combination with perioperative immunosuppression
regimen, with a focus on evaluating near-term safety and
tolerability. Enrollment in this third cohort is expected to be
completed in the second half of 2024.
- The Company expects to provide an
additional interim update from its ongoing Phase I/II clinical
trials of AMT-130 in mid-2025. The update will include follow-up
data on all patients treated with AMT-130 in the first two cohorts,
including three years of follow-up on 21 treated patients.
- Initiating new Phase I/II clinical
studies
- AMT-260 for the treatment of
refractory mesial temporal lobe epilepsy (mTLE) – Patient screening
in a Phase I/II clinical study has been initiated and enrollment is
expected to begin in the third quarter of 2024. The first part of
the U.S., multi-center, open-label trial Phase I/II is expected to
include up to 12 patients across two dose cohorts.
- AMT-191 for the treatment of Fabry
disease – Patient screening in a Phase I/II clinical study has been
initiated and enrollment is expected to begin in the third quarter
of 2024. The U.S., multi-center, open-label trial is expected to
include up to 12 adult male patients across two dose cohorts.
- AMT-162 for the treatment of SOD1
amyotrophic lateral sclerosis (ALS) – Patient screening in a Phase
I/II clinical study has been initiated and enrollment is expected
to begin in the third quarter of 2024. The U.S., multi-center,
open-label trial is expected to include up to 12 patients across
three dose cohorts.
Capital Preservation Initiatives
uniQure conducted and recently concluded a
detailed review of its operating expenses with the goals of
conserving capital, streamlining operations, and ensuring
sufficient cash resources to achieve multiple potentially
meaningful value creating milestones. As a result of this review,
uniQure has or will be taking the following steps:
- The sale of the Lexington,
Massachusetts manufacturing facility to Genezen announced on July
1, 2024.
- Global workforce reductions aimed
at organizational rightsizing, delayering and outsourcing sub- and
non-critical activities.
- Inclusive of the sale of the
manufacturing facility, the elimination of approximately 65% or 300
roles across the organization. Certain intended organizational
changes are subject to review and advice from the Company’s
Amsterdam-based works council, which is ongoing and expected to be
completed in the third quarter of 2024. The Company expects to
substantially complete the restructuring in the fourth quarter of
2024.
- A reduction in annual recurring
cash burn of approximately 40% or $75 million, which includes
savings in interest expense from the retirement of $50 million in
outstanding debt.
- Current balance of cash, cash
equivalents and investment securities of $524 million as of June
30, 2024 are expected to fund operations through the end 2027.
As a result of the sale of the Lexington
manufacturing facility, Pierre Caloz, chief operating officer of
uniQure will depart the company. Amin Abujoub, Ph.D. who previously
served as chief quality officer, has been appointed to the new role
of chief technical operations officer in which he will be
responsible for global oversight of contract manufacturers as well
as internal operations, facilities, process and analytical
development, and quality.
“After a comprehensive review motivated by our
patient-driven mission, we are making important changes to align
uniQure with our objectives of delivering sustainable value
creation and ensuring we are optimally positioned for the future,”
added Matt Kapusta. “We have taken great care to ensure those
impacted by these changes are supported, and greatly appreciate the
contributions they have made to the company. I particularly want to
thank Pierre for his leadership, dedication and compassion, all of
which were critical in achieving multiple HEMGENIX® approvals and
establishing world-class commercial manufacturing capabilities.
Through our relationship with Genezen, we hope to leverage these
capabilities for years to come.”
“These decisions will enable us to prioritize
investments in our Huntington’s disease, temporal lobe epilepsy and
other gene therapy programs, as well as innovating and broadening
the long-term applicability of AAV-delivered gene therapy.”
Upcoming Investor Events
- Wells Fargo 2024 Healthcare Conference, September 5 – Boston,
MA
- 2024 Cantor Global Healthcare Conference, September 18 – New
York, NY
Financial Highlights
Cash position: As of June 30,
2024, the Company held cash and cash equivalents and investment
securities of $524.4 million, compared to $617.9 million as of
December 31, 2023. Based on the Company’s current operating plan
and pending successful completion of the capital preservation
measures described above, the Company expects cash, cash
equivalents and investment securities will be sufficient to fund
operations through the end of 2027.
Revenues: Revenue for the three
months ended June 30, 2024, was $11.1 million, compared to $2.4
million in the same period in 2023. The increase of $8.7 million in
revenue resulted from an increase of $6.8 million in collaboration
revenue, an increase of $1.1 million in license revenue and an
increase of $0.8 million in revenue from contract manufacturing of
HEMGENIX® for CSL.
Cost of license revenue: Costs
of license revenues were $0.2 million for the three months ended
June 30, 2024, compared to $0.0 million for the same period in
2023. The increase primarily relates to not incurring such costs in
2023.
Cost of contract manufacturing
revenues: Costs of contract manufacturing revenues were
$7.2 million for the three months ended June 30, 2024, compared to
$1.4 million for the same period in 2023. The increase primarily
relates to expensing costs previously capitalized as inventory.
R&D expenses: Research and
development expenses were $33.7 million for the three months ended
June 30, 2024, compared to $46.0 million during the same period in
2023. The $12.3 million decrease was primarily related to a
decrease of $6.6 million in employee-related expenses partially
offset by an increase of $1.1 million severance costs related to
the sale of the Lexington facility, a $4.5 million decrease in
costs incurred related to preclinical supplies, and a decrease of
$2.0 million related to the changes in fair value of contingent
consideration.
SG&A expenses: Selling,
general and administrative expenses were $15.8 million for the
three months ended June 30, 2024, compared to $21.2 million during
the same period in 2023. The $5.4 million decrease was primarily
related to a $3.8 million decrease in financial advisory fees, a
$1.6 million decrease in intellectual property fees and information
technology expenses, and a $1.1 million decrease in
employee-related expenses compared to the prior year period. These
decreases were partially offset by a $1.6 million increase in
professional fees primarily related to the sale of the Lexington
facility.
Other non-operating items, net:
Other non-operating items, net was an expense of $11.3 million for
the three months ended June 30, 2024, compared to $3.2 million for
the same period in 2023. The $8.1 million increase in other
non-operating items, net was primarily related to an increase in
non-cash interest expense of $9.3 million related to the royalty
agreement that the Company entered into in May 2023 and an increase
in net foreign currency losses of $1.4 million, which were
partially offset by an increase of $2.6 million in interest income
earned on investment securities and cash on hand.
Net loss: The net
loss for the three months ended June 30, 2024, was $56.3 million,
or $1.16 basic and diluted loss per ordinary share, compared to
$68.5 million net loss for the same period in 2023, or $1.44 basic
and diluted loss per ordinary share.
About uniQure
uniQure is delivering on the promise of gene
therapy – single treatments with potentially curative results. The
approvals of uniQure’s gene therapy for hemophilia B – an historic
achievement based on more than a decade of research and clinical
development – represent a major milestone in the field of genomic
medicine and ushers in a new treatment approach for patients living
with hemophilia. uniQure is now advancing 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.
Examples of these forward-looking statements include, but are not
limited to, statements concerning the Company’s cash runway and its
ability to fund its operations through the end of 2027 and achieve
multiple value-generating inflection points; the Company’s
expectations regarding planned organizational changes, including
reductions in headcount and lower annual cash burn resulting from
such changes; the ability of such organizational changes to yield
the funding required to achieve key clinical and regulatory
milestones, maximize shareholder value, and deliver transformative
medicines to patients; the Company’s planned workforce reductions,
including the extent and timing thereof and the costs associated
with such workforce reductions; the Company’s plans to announce
additional interim updates from its ongoing U.S. and European Phase
I/II clinical studies of AMT-130; the Company’s plans to meet the
FDA regarding potential expedited clinical development pathways for
AMT-130, the timing of such regulatory interactions and
expectations regarding potential regulatory clarity from such
interactions; the Company’s plans regarding the third cohort in its
AMT-130 clinical trial and the timing of enrollment for such
cohort; and the Company’s plans to initiate patient enrollment for
AMT-191, AMT-260 and AMT-162 and the design of trials for the
Company’s additional clinical programs. The Company’s actual
results could differ materially from those anticipated in these
forward-looking statements for many reasons. These risks and
uncertainties include, among others: risks associated with the
implementation of the Company’s restructuring plans; risks
associated with the clinical results and the development and timing
of the Company’s programs; the Company’s interactions with
regulatory authorities, which may affect the initiation, timing and
progress of clinical trials and pathways to approval; the Company’s
ability to continue to build and maintain the company
infrastructure and personnel needed to achieve its goals following
planned workforce reductions; the Company’s effectiveness in
managing current and future clinical trials and regulatory
processes; the continued development and acceptance of gene
therapies; the Company’s ability to demonstrate the therapeutic
benefits of its gene therapy candidates in clinical trials; the
Company’s ability to obtain, maintain and protect intellectual
property; the Company’s ability to fund its operations and to raise
additional capital as needed; and the impact of global economic
uncertainty, rising inflation, rising interest rates or market
disruptions on its business. These risks and uncertainties are more
fully described under the heading "Risk Factors" in the Company’s
periodic filings with the U.S. Securities & Exchange Commission
(“SEC”), including its Annual Report on Form 10-K filed February
28, 2024 and in other filings that the Company makes with the SEC
from time to time. 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: |
|
|
Chiara Russo |
Tom Malone |
Direct: 617-306-9137 |
Direct: 339-970-7558 |
Mobile: 617-306-9137 |
Mobile:339-223-8541 |
c.russo@uniQure.com |
t.malone@uniQure.com |
|
|
|
|
uniQure
N.V. |
|
UNAUDITED
CONSOLIDATED BALANCE SHEETS |
|
|
|
June
30, |
|
December 31, |
|
|
|
|
2024 |
|
|
2023 |
|
|
|
(in
thousands, except share and per share amounts) |
|
Current assets |
|
|
|
|
|
Cash and
cash equivalents |
|
$ |
287,877 |
|
$ |
241,360 |
|
Current
investment securities |
|
|
236,553 |
|
|
376,532 |
|
Inventories,
net |
|
|
- |
|
|
12,024 |
|
Accounts
receivable |
|
|
7,850 |
|
|
4,193 |
|
Prepaid
expenses |
|
|
18,278 |
|
|
15,089 |
|
Assets held
for sale |
|
|
37,964 |
|
|
- |
|
Other
current assets |
|
|
3,446 |
|
|
2,655 |
|
Total current assets |
|
|
591,968 |
|
|
651,853 |
|
Non-current assets |
|
|
|
|
|
Property,
plant and equipment, net |
|
$ |
26,186 |
|
$ |
46,548 |
|
Operating
lease right-of-use assets |
|
|
14,925 |
|
|
28,789 |
|
Intangible
assets, net |
|
|
58,659 |
|
|
60,481 |
|
Goodwill |
|
|
23,112 |
|
|
26,379 |
|
Deferred tax
assets, net |
|
|
10,718 |
|
|
12,276 |
|
Other
non-current assets |
|
|
5,278 |
|
|
5,363 |
|
Total non-current assets |
|
|
138,878 |
|
|
179,836 |
|
Total assets |
|
$ |
730,846 |
|
$ |
831,689 |
|
Current liabilities |
|
|
|
|
|
Accounts
payable |
|
$ |
4,407 |
|
$ |
6,586 |
|
Accrued
expenses and other current liabilities |
|
|
26,491 |
|
|
30,534 |
|
Current
portion of contingent consideration |
|
|
28,060 |
|
|
28,211 |
|
Current
portion of operating lease liabilities |
|
|
3,625 |
|
|
8,344 |
|
Liabilities
held for sale |
|
|
17,885 |
|
|
- |
|
Total current liabilities |
|
|
80,468 |
|
|
73,675 |
|
Non-current liabilities |
|
|
|
|
|
Long-term
debt |
|
|
102,507 |
|
|
101,749 |
|
Liability
from royalty financing agreement |
|
|
415,940 |
|
|
394,241 |
|
Operating
lease liabilities, net of current portion |
|
|
12,369 |
|
|
28,316 |
|
Contingent
consideration, net of current portion |
|
|
12,078 |
|
|
14,795 |
|
Deferred tax
liability, net |
|
|
7,323 |
|
|
7,543 |
|
Other
non-current liabilities |
|
|
3,054 |
|
|
3,700 |
|
Total non-current liabilities |
|
|
553,271 |
|
|
550,344 |
|
Total liabilities |
|
|
633,739 |
|
|
624,019 |
|
Shareholders' equity |
|
|
|
|
|
Total shareholders' equity |
|
|
97,107 |
|
|
207,670 |
|
Total liabilities and shareholders' equity |
|
$ |
730,846 |
|
$ |
831,689 |
|
|
|
|
|
|
|
|
uniQure
N.V. |
|
UNAUDITED
CONSOLIDATED STATEMENTS OF OPERATIONS |
|
|
|
Three months ended June 30, |
|
|
|
2024 |
|
|
|
2023 |
|
|
|
(in
thousands, except share and per share amounts) |
Total revenues |
|
$ |
11,126 |
|
|
$ |
2,422 |
|
Operating expenses: |
|
|
|
|
Cost of
license revenues |
|
|
(234 |
) |
|
|
— |
|
Cost of
contract manufacturing revenues |
|
|
(7,227 |
) |
|
|
(1,352 |
) |
Research and
development expenses |
|
|
(33,655 |
) |
|
|
(46,036 |
) |
Selling,
general and administrative expenses |
|
|
(15,767 |
) |
|
|
(21,181 |
) |
Total operating expenses |
|
|
(56,883 |
) |
|
|
(68,569 |
) |
Other
income |
|
|
1,983 |
|
|
|
1,302 |
|
Other
expense |
|
|
(236 |
) |
|
|
(229 |
) |
Loss
from operations |
|
|
(44,010 |
) |
|
|
(65,074 |
) |
Non-operating items, net |
|
|
(11,341 |
) |
|
|
(3,237 |
) |
Loss
before income tax (expense) / benefit |
|
$ |
(55,351 |
) |
|
$ |
(68,311 |
) |
Income tax
(expense) / benefit |
|
|
(948 |
) |
|
|
(163 |
) |
Net
loss |
|
$ |
(56,299 |
) |
|
$ |
(68,474 |
) |
|
|
|
|
|
Basic and
diluted net loss per ordinary share |
|
$ |
(1.16 |
) |
|
$ |
(1.44 |
) |
Weighted
average shares used in computing basic and diluted net loss per
ordinary share |
|
|
48,622,440 |
|
|
|
47,649,520 |
|
|
|
|
|
|
|
|
|
|
|
________________________________________1 All
p-values are nominal and unadjusted. Statistical comparisons of
patients treated with AMT-130 to the propensity score-weighted
external control were conducted on a post-hoc basis.
uniQure NV (NASDAQ:QURE)
과거 데이터 주식 차트
부터 11월(11) 2024 으로 12월(12) 2024
uniQure NV (NASDAQ:QURE)
과거 데이터 주식 차트
부터 12월(12) 2023 으로 12월(12) 2024