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 fourth quarter
and full year 2023 and highlighted recent progress across its
business.
“We are pleased with the progress made across
the company in 2023 and are now laser-focused on execution across
multiple clinical programs,” stated Matt Kapusta, chief executive
officer of uniQure. “Our top priorities are to engage with
regulatory authorities to clarify the approval pathway for AMT-130
in Huntington’s disease and to initiate patient enrollment across
three additional clinical trials as expeditiously as possible.”
“While we enter 2024 with a strong balance
sheet, we remain disciplined on the prudent and efficient
allocation of our capital,” he continued. “Any decision to advance
AMT-130 into late-stage development will necessitate a clear and
timely approval pathway and financial feasibility, including
through a partnership which we would secure before beginning any
Phase III trial.”
Recent Updates
- Advancing AMT-130 for the treatment
of Huntington’s disease
- In December 2023, the Company
announced updated clinical data from the ongoing U.S. and European
Phase I/II studies of AMT-130 for the treatment of early-stage
Huntington’s disease.
- Patients treated with AMT-130
continued to show evidence of preserved neurological function with
potential dose-dependent clinical benefits relative to a
non-concurrent criteria-matched natural history of the disease.
When compared to the expected rate of decline from an inclusion
criteria-matched natural history data set, AMT-130 showed favorable
trends in composite Unified Huntington’s Disease Rating Scale
(cUHDRS), Total Functional Capacity (TFC) and Total Motor Score
(TMS) key clinical rating scales designed to assess disease
progression.
- Further declines in neurofilament
light chain (NfL), an exploratory biomarker for the measurement of
neuronal degradation and disease progression were observed among
patients treated with AMT-130. Mean NfL in the cerebrospinal fluid
(CSL) for low-dose patients remained below baseline at 30 months of
follow-up and high-dose patients were near baseline at 18
months.
- AMT-130 continued to be generally
well-tolerated with a manageable safety profile across both
doses.
- In the fourth quarter of 2023, the
Company initiated patient dosing in a third cohort of up to 12
patients to further investigate both doses of AMT-130 in
combination with perioperative immunosuppression, with a focus on
evaluating near-term safety and tolerability. Enrollment in this
cohort is expected to be completed in the second half of 2024.
- In the second quarter of 2024, the
Company expects to initiate regulatory interactions with the U.S.
Food and Drug Administration (FDA) to discuss data from the ongoing
Phase I/II studies and potential strategies for the further
development of AMT-130. By the end of 2024, the Company expects to
have greater clarity regarding a potential approval pathway for
AMT-130.
- In mid-2024, the Company expects to
provide an interim update from the ongoing Phase I/II studies of
AMT-130, including up to 24- and 36-month follow-up data from all
treated patients in the U.S. and European trials.
- Advancing additional programs into
the clinic
- AMT-260 for the treatment of
refractory mesial temporal lobe epilepsy (rMTLE) – In September
2023, the Company announced the clearance of an IND for the Phase
I/IIa clinical study of AMT-260. Site initiation is underway, and
patient enrollment is expected to begin in the first half of
2024.
- 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 known as AMT-162, for the treatment of superoxide
dismutase 1 (SOD1) ALS, a rare, genetic form of ALS. Patient
enrollment in a Phase I/II clinical trial is expected to begin in
the first half of 2024.
- AMT-191 for the treatment of Fabry
disease – In November 2023, the Company announced the clearance of
an IND for the Phase I/IIa clinical study of AMT-191. Patient
enrollment is expected to begin in the first half of 2024.
- Generating value from the
commercial launch of HEMGENIX®
- The Company continues to provide
commercial supply of etranacogene dezaparvovec (HEMGENIX®) to its
partner CSL Behring and manufactures the product at its cGMP
facility in Lexington, MA. The Company is one of a select number of
gene therapy companies with a qualified facility producing routine
commercial manufacturing for the market.
Upcoming Investor Events
- 44th Annual Cowen Health Care
Conference, March 5, 2024 – Boston, MA
- Leerink Global Biopharma Conference
2024, March 12, 2024 – Miami, FL
- UBS Virtual CNS Day, March 18,
2024
Financial Highlights
Cash position: As of December
31, 2023, the Company held cash and cash equivalents and investment
securities of $617.9 million, compared to $392.8 million as of
December 31, 2022. The Company entered into a royalty agreement in
May 2023 and received an upfront payment of $375.0 million, and
collected $100.0 million in July 2023 for a milestone due from CSL
following the first sale of HEMGENIX® in the U.S. The Company
expects cash, cash equivalents and investment securities will fund
operations into the second quarter of 2027.
Revenues: Revenue for the year
ended December 31, 2023 was $15.8 million, compared to $106.5
million in the same period in 2022. Revenues in 2022 included
$100.0 million of license revenue related to the U.S. first sale
milestone payment of HEMGENIX® that the Company expected to receive
in 2023. Revenue from contract manufacturing HEMGENIX® for CSL
increased $9.1 million in 2023 compared to 2022.
Cost of contract manufacturing
revenues: Cost of contract manufacturing revenues were
$13.6 million for the year ended December 31, 2023, compared to
$2.1 million for the same period in 2022. The increase relates to
an increase in activities related to contract manufacturing
HEMGENIX® for CSL.
R&D expenses: Research and
development expenses were $214.9 million for the year ended
December 31, 2023, compared to $197.6 million during the same
period in 2022. The $17.2 million increase was primarily related to
a $5.9 million net increase of external research and development
expenses including a $10.0 million payment made to Apic Bio to
acquire AMT-162, an $8.8 million non-cash increase related to the
fair value of contingent consideration associated with the
Company’s acquisition of Corlieve Therapeutics in 2021 as well as a
$1.4 million non-cash impairment loss related to our Lexington
research facility.
SG&A expenses: Selling,
general and administrative expenses were $74.6 million for the year
ended December 31, 2023, compared to $55.1 million during the same
period in 2022. The $19.5 million increase was primarily related to
a $9.7 million increase of professional, financial advisory and
intellectual property fees, a $3.7 million increase in information
technology expenses as well as a $3.7 million increase in personnel
and contractor-related expenses.
Other non-operating items, net:
Other non-operating items, net was an expense of $23.7 million for
the year ended December 31, 2023, compared to net income of $14.9
million for the same period in 2022. The $38.6 million decrease in
other non-operating items, net was primarily related to a decrease
in foreign currency gains, net of $24.9 million and an increase in
non-cash interest expense of $26.9 million related to the royalty
agreement that the Company entered into in May 2023, which
partially was offset by an increase of $19.0 million in interest
income earned on investment securities and cash on hand.
Net loss:The net loss for the
year ended December 31, 2023, was $308.5 million, or $6.47 basic
and diluted loss per ordinary share, compared to $126.8 million net
loss for the same period in 2022, or $2.71 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 expected cost savings related to its strategic
reorganization; the Company’s plans to announce additional follow
up data from the ongoing U.S. and European Phase I/II clinical
studies of AMT-130; the Company’s plans to initiate interactions
with regulatory authorities regarding the further development of
AMT-130 and the timing of regulatory clarity from such
interactions; and the Company’s plans to initiate patient
enrollment for AMT-260, AMT-162 and AMT-191 in the first half 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 |
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, |
|
|
2023 |
|
|
2022 |
|
|
(in thousands, except share and per share
amounts) |
|
Current assets |
|
|
|
|
Cash and cash equivalents |
$ |
241,360 |
|
$ |
228,012 |
|
Current investment securities |
|
376,532 |
|
|
124,831 |
|
Accounts receivable and contract asset |
|
4,193 |
|
|
102,376 |
|
Inventories, net |
|
12,024 |
|
|
6,924 |
|
Prepaid expenses |
|
15,089 |
|
|
11,817 |
|
Other current assets and receivables |
|
2,655 |
|
|
2,814 |
|
Total current assets |
|
651,853 |
|
|
476,774 |
|
Non-current assets |
|
|
|
|
Property, plant and equipment, net |
|
46,548 |
|
|
50,532 |
|
Non-current investment securities |
|
— |
|
|
39,984 |
|
Operating lease right-of-use assets |
|
28,789 |
|
|
32,726 |
|
Intangible assets, net |
|
60,481 |
|
|
58,778 |
|
Goodwill |
|
26,379 |
|
|
25,581 |
|
Deferred tax assets, net |
|
12,276 |
|
|
14,528 |
|
Other non-current assets |
|
5,363 |
|
|
6,061 |
|
Total non-current assets |
|
179,836 |
|
|
228,190 |
|
Total assets |
$ |
831,689 |
|
$ |
704,964 |
|
Current liabilities |
|
|
|
|
Accounts payable |
$ |
6,586 |
|
$ |
10,984 |
|
Accrued expenses and other current liabilities |
$ |
30,534 |
|
$ |
30,571 |
|
Current portion of contingent consideration |
|
28,211 |
|
|
25,982 |
|
Current portion of operating lease liabilities |
|
8,344 |
|
|
8,382 |
|
Total current liabilities |
|
73,675 |
|
|
75,919 |
|
Non-current liabilities |
|
|
|
|
Long-term debt |
|
101,749 |
|
|
102,791 |
|
Liability from royalty financing agreement |
|
394,241 |
|
|
— |
|
Operating lease liabilities, net of current portion |
|
28,316 |
|
|
31,719 |
|
Contingent consideration, net of current portion |
|
14,795 |
|
|
9,334 |
|
Deferred tax liability, net |
|
7,543 |
|
|
8,257 |
|
Other non-current liabilities |
|
3,700 |
|
|
935 |
|
Total non-current liabilities |
|
550,344 |
|
|
153,036 |
|
Total liabilities |
|
624,019 |
|
|
228,955 |
|
Shareholders' equity |
|
|
|
|
Total shareholders' equity |
|
207,670 |
|
|
476,009 |
|
Total liabilities and shareholders' equity |
$ |
831,689 |
|
$ |
704,964 |
|
|
|
|
|
|
|
|
|
|
|
Balancing check |
|
- |
|
|
- |
|
uniQure N.V.UNAUDITED CONSOLIDATED
STATEMENTS OF OPERATIONS |
|
Years ended December 31, |
|
|
2023 |
|
|
|
2022 |
|
|
|
2021 |
|
|
(in thousands, except share and per share
amounts) |
License revenues |
$ |
2,758 |
|
|
$ |
100,000 |
|
|
$ |
517,400 |
|
Contract manufacturing revenues |
|
10,835 |
|
|
|
1,717 |
|
|
|
- |
|
Collaboration revenues |
|
2,250 |
|
|
|
4,766 |
|
|
|
6,602 |
|
Total revenues |
|
15,843 |
|
|
|
106,483 |
|
|
|
524,002 |
|
Operating expenses: |
|
|
|
|
|
Cost of license revenues |
|
(65 |
) |
|
|
(1,254 |
) |
|
|
(24,976 |
) |
Cost of contract manufacturing revenues |
|
(13,563 |
) |
|
|
(2,089 |
) |
|
|
- |
|
Research and development expenses |
|
(214,864 |
) |
|
|
(197,591 |
) |
|
|
(143,548 |
) |
Selling, general and administrative expenses |
|
(74,591 |
) |
|
|
(55,059 |
) |
|
|
(56,290 |
) |
Total operating expenses |
|
(303,083 |
) |
|
|
(255,993 |
) |
|
|
(224,814 |
) |
Other income |
|
6,059 |
|
|
|
7,171 |
|
|
|
12,306 |
|
Other expense |
|
(1,690 |
) |
|
|
(820 |
) |
|
|
(876 |
) |
(Loss) / income from operations |
|
(282,871 |
) |
|
|
(143,159 |
) |
|
|
310,618 |
|
Non-operating items, net |
|
(23,686 |
) |
|
|
14,900 |
|
|
|
22,188 |
|
(Loss) / income before income tax (expense) /
benefit |
$ |
(306,557 |
) |
|
$ |
(128,259 |
) |
|
$ |
332,806 |
|
Income tax (expense) / benefit |
|
(1,921 |
) |
|
|
1,470 |
|
|
|
(3,217 |
) |
Net (loss) / income |
$ |
(308,478 |
) |
|
$ |
(126,789 |
) |
|
$ |
329,589 |
|
Earnings per ordinary share - basic |
|
|
|
|
|
Basic net (loss) / income per ordinary share |
$ |
(6.47 |
) |
|
$ |
(2.71 |
) |
|
$ |
7.17 |
|
Earnings per ordinary share - diluted |
|
|
|
|
|
Diluted net (loss) / income per ordinary share |
$ |
(6.47 |
) |
|
$ |
(2.71 |
) |
|
$ |
7.04 |
|
Weighted average shares - basic |
|
47,670,986 |
|
|
|
46,735,045 |
|
|
|
45,986,467 |
|
Weighted average shares - diluted |
|
47,670,986 |
|
|
|
46,735,045 |
|
|
|
46,840,972 |
|
|
|
|
|
|
|
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