uniQure Announces First Quarter 2024 Financial Results and
Highlights Recent Company Progress
~ On track to initiate FDA interaction
regarding AMT-130 in second quarter of 2024 and provide a clinical
update from the Phase I/II trials in mid-2024 ~
~ Clinical trial initiation for Fabry disease
on track to begin in second quarter of 2024, followed by refractory
mesial temporal lobe epilepsy and SOD1-ALS in third quarter of 2024
~
~ Comprehensive review of operations and
options to reduce expenses underway and expected to be completed in
mid-2024 ~
LEXINGTON, Mass. and AMSTERDAM, May 07, 2024
(GLOBE NEWSWIRE) -- 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 2024 and highlighted recent progress across
its business.
“In the first quarter, we made solid progress
across three key business priorities – clarifying the road ahead
for AMT-130 in Huntington’s disease, initiating three new Phase
I/II clinical trials, and prudently conserving our capital,” stated
Matt Kapusta, chief executive officer of uniQure. “We are on track
to initiate FDA interactions on AMT-130 this quarter and look
forward to presenting longer-term follow-up data from our Phase
I/II trials mid-year, which will include up to three years of
patient follow-up on 29 treated patients. We have also
made significant progress towards the initiation of new Phase I/II
studies. We have obtained IRB approvals for initial sites, which
are poised for imminent activation, and have also identified
prospective patients. Enrollment for the Fabry disease study is on
track to begin in the second quarter of 2024, with the SOD1-ALS and
MTLE studies expected to begin in third quarter.”
“In addition, we are actively engaged in a
comprehensive review of uniQure’s operations and options to reduce
expenses and increase shareholder value,” he added. “We believe
deeply in the significant value of our assets, as well as the need
to achieve a focused and sustainable level of investment in order
to maximize this potential. We expect to complete this evaluation
mid-2024.”
Recent Updates
- Advancing AMT-130 for the
treatment of Huntington’s disease
- The Company is on track to initiate
interactions with the U.S. Food and Drug Administration in the
second quarter of 2024 with the goal of defining the future
clinical and regulatory pathway for AMT-130. These interactions,
which are expected to continue throughout the year, are expected to
include discussions of the interim data from the ongoing Phase I/II
clinical trials and the potential to leverage natural history
comparators alongside our long-term Phase I/II clinical data. The
Company expects to provide an update on its regulatory plans as
part of the next clinical update on AMT-130 mid-year and have
greater clarity regarding a potential approval pathway for AMT-130
before the end of the year.
- In mid-2024, the Company expects to
provide an update from its ongoing Phase I/II clinical trials of
AMT-130. The data will include up to three years of follow-up data
on 29 treated patients in both the low- and higher-dose cohorts, of
which 21 patients will have a minimum of two years of
follow-up.
- 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, 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.
- Initiating new Phase I/II
clinical studies
- AMT-191 for the treatment of
Fabry disease - Patient enrollment in a Phase I/IIa clinical
trial is expected to begin in the second quarter of 2024. The Phase
I/IIa clinical trial will be a U.S.-based, multi-center, open-label
trial consisting of two cohorts enrolling up to six adult male
patients each. The study is designed to evaluate safety,
tolerability, and early signs of efficacy.
- AMT-162 for the treatment of
SOD1 amyotrophic lateral sclerosis (ALS) – Patient enrollment
in a Phase I/II clinical trial is expected to begin in the third
quarter of 2024. The Phase I/II clinical trial will be a
U.S.-based, multi-center, open-label trial consisting of three
cohorts with up to four patients each receiving a one-time
intrathecal infusion with immunosuppression. The study is designed
to evaluate safety, tolerability, and early signs of efficacy.
- AMT-260 for the treatment of
refractory mesial temporal lobe epilepsy (MTLE) – Patient
enrollment in a Phase I/IIa clinical trial is expected to begin in
third quarter of 2024. The Phase I/II will be a U.S.-based
consisting of two parts. The first part is a multicenter,
open-label trial with two dosing cohorts of six patients each to
assess safety, tolerability, and first signs for efficacy of
AMT-260 in patients with refractory MTLE. The second part is
expected to be a randomized, controlled trial to generate proof of
concept (POC) data.
Upcoming Investor Events
- RBC Capital Markets 2024 Global
Healthcare Conference, May 15th – New York, NY
Financial Highlights
Cash position: As of March 31,
2024, the Company held cash and cash equivalents and investment
securities of $555.7 million, compared to $617.9 million as of
December 31, 2023. The Company expects cash, cash equivalents and
investment securities will fund operations into the second quarter
of 2027.
Revenues: Revenue for the three
months ended March 31, 2024 was $8.5 million, compared to $5.3
million in the same period in 2023. The net increase of $3.2
million in revenue resulted from an increase of $2.9 million from
collaboration revenue and an increase of $1.2 million in license
revenue partially offset by a decrease of $0.9 million from
contract manufacturing of HEMGENIX® for
CSL.
Cost of contract manufacturing
revenues: Cost of contract manufacturing revenues were
$9.1 million for the three months ended March 31, 2024, compared to
$2.4 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 $40.7 million for the three months ended
March 31, 2024, compared to $60.8 million during the same period in
2023. The $20.1 million decrease was primarily related to an $8.1
million decrease in external spend for the ALS SOD1 program, as
prior year costs included a $10.0 million one-time cost for the
acquisition of the program from Apic Bio. Additionally, there was a
$7.0 million decrease in preclinical and other program spend, a
$2.0 million decrease in costs incurred related to preclinical
supplies and a $2.7 million decrease in employee related
expenses.
SG&A expenses: Selling,
general and administrative expenses were $13.9 million for the
three months ended March 31, 2024, compared to $17.8 million during
the same period in 2023. The $3.9 million decrease was primarily
related to a $1.7 million decrease in professional and intellectual
property fees, as well as a reduction of expenses for information
technology.
Other non-operating items,
net:
Other non-operating items, net was an expense of $10.7 million for
the three months ended March 31, 2024, compared to $4.3 million for
the same period in 2023. The $6.5 million increase in other
non-operating items, net was primarily related to an increase in
non-cash interest expense of $12.5 million related to the royalty
agreement that the Company entered into in May 2023, which
partially was offset by an increase of $4.8 million in interest
income earned on investment securities and cash on hand.
Net loss:
The net loss for the three months ended March 31, 2024, was $65.6
million, or $1.36 basic and diluted loss per ordinary share,
compared to $77.2 million net loss for the same period in 2023, or
$1.63 basic and diluted loss per ordinary share.
About uniQure
uniQure’s mission is to reimagine the future of
medicine by delivering innovative cures that transform lives. The
recent approvals of our gene therapy for hemophilia B – a 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. We are now leveraging our modular and validated
technology and manufacturing platform to advance a pipeline of
proprietary gene therapies for the treatment of patients with
Huntington's disease, refractory mesial temporal lobe epilepsy,
amyotrophic lateral sclerosis (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 into the second quarter of 2027; the
Company’s ongoing review of its operations and options to reduce
expenses, the Company’s expectations that such review will lead to
a strategy that, if effectively executed, will reduce operating
expenses and increase shareholder value, and the expected timing of
the completion of such review; the Company’s plans to announce
additional follow-up data from its ongoing U.S. and European Phase
I/II clinical studies of AMT-130; the Company’s plans to initiate
interactions with the FDA regarding the further development of
AMT-130, the timing of such interactions and expectations regarding
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 in the second quarter of
2024, and to initiate patient enrollment for AMT-260 and AMT-162 in
the third quarter of 2024. 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 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; 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: |
|
|
|
Maria E. Cantor Direct: 339-970-7536
Mobile: 617-680-9452
m.cantor@uniQure.com
|
Chiara Russo Direct: 617-306-9137
Mobile: 617-306-9137
c.russo@uniQure.com
|
Tom Malone
Direct: 339-970-7558
Mobile:339-223-8541
t.malone@uniQure.com |
uniQure N.V.
UNAUDITED CONSOLIDATED BALANCE SHEETS |
|
|
|
|
|
March
31, |
|
December 31, |
|
2024 |
|
2023 |
|
(in
thousands, except share and per share amounts) |
Current assets |
|
|
|
Cash and cash equivalents |
$ |
243,062 |
|
$ |
241,360 |
Current
investment securities |
|
312,621 |
|
|
376,532 |
Accounts
receivable and contract asset |
|
10,717 |
|
|
4,193 |
Inventories |
|
7,672 |
|
|
12,024 |
Prepaid
expenses |
|
18,839 |
|
|
15,089 |
Other
current assets and receivables |
|
3,092 |
|
|
2,655 |
Total current assets |
|
596,003 |
|
|
651,853 |
Non-current assets |
|
|
|
Property,
plant and equipment, net |
|
44,554 |
|
|
46,548 |
Operating
lease right-of-use assets |
|
27,695 |
|
|
28,789 |
Intangible
assets, net |
|
59,111 |
|
|
60,481 |
Goodwill |
|
25,795 |
|
|
26,379 |
Deferred tax
assets, net |
|
11,594 |
|
|
12,276 |
Other
non-current assets |
|
5,298 |
|
|
5,363 |
Total non-current assets |
|
174,047 |
|
|
179,836 |
Total assets |
$ |
770,050 |
|
$ |
831,689 |
Current liabilities |
|
|
|
Accounts
payable |
$ |
5,231 |
|
$ |
6,586 |
Accrued
expenses and other current liabilities |
|
22,658 |
|
|
30,534 |
Current
portion of contingent consideration |
|
27,587 |
|
|
28,211 |
Current
portion of operating lease liabilities |
|
7,997 |
|
|
8,344 |
Total current liabilities |
|
63,473 |
|
|
73,675 |
Non-current liabilities |
|
|
|
Long-term
debt |
|
102,120 |
|
|
101,749 |
Liability
from royalty financing agreement |
|
405,398 |
|
|
394,241 |
Operating
lease liabilities, net of current portion |
|
26,983 |
|
|
28,316 |
Contingent
consideration, net of current portion |
|
14,625 |
|
|
14,795 |
Deferred tax
liability, net |
|
7,376 |
|
|
7,543 |
Other
non-current liabilities |
|
3,321 |
|
|
3,700 |
Total non-current liabilities |
|
559,823 |
|
|
550,344 |
Total liabilities |
|
623,296 |
|
|
624,019 |
Shareholders' equity |
|
|
|
Total shareholders' equity |
|
146,754 |
|
|
207,670 |
Total liabilities and shareholders' equity |
$ |
770,050 |
|
$ |
831,689 |
uniQure N.V.
UNAUDITED CONSOLIDATED STATEMENTS OF
OPERATIONS |
|
|
|
|
|
Three months ended March 31, |
|
|
2024 |
|
|
|
2023 |
|
|
(in
thousands, except share and per share amounts) |
Total revenues |
$ |
8,485 |
|
|
$ |
5,325 |
|
Operating expenses: |
|
|
|
Cost of
license revenues |
|
(150 |
) |
|
|
— |
|
Cost of
contract manufacturing revenues |
|
(9,076 |
) |
|
|
(2,435 |
) |
Research and
development expenses |
|
(40,692 |
) |
|
|
(60,809 |
) |
Selling,
general and administrative expenses |
|
(13,937 |
) |
|
|
(17,848 |
) |
Total operating expenses |
|
(63,855 |
) |
|
|
(81,092 |
) |
Other
income |
|
1,376 |
|
|
|
1,811 |
|
Other
expense |
|
(234 |
) |
|
|
(216 |
) |
Loss
from operations |
|
(54,228 |
) |
|
|
(74,172 |
) |
Non-operating items, net |
|
(10,734 |
) |
|
|
(4,262 |
) |
Loss
before income tax (expense) / benefit |
$ |
(64,962 |
) |
|
$ |
(78,434 |
) |
Income tax
(expense) / benefit |
|
(656 |
) |
|
|
1,207 |
|
Net
loss |
$ |
(65,618 |
) |
|
$ |
(77,227 |
) |
|
|
|
|
Basic and
diluted net loss per ordinary share |
$ |
(1.36 |
) |
|
$ |
(1.63 |
) |
Weighted
average shares used in computing basic and diluted net loss per
ordinary share |
|
48,384,510 |
|
|
|
47,436,335 |
|
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