uniQure Announces Second Quarter 2021 Financial Results and
Highlights Recent Company Progress
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 2021 and highlighted recent progress across its business.
“We had a highly productive second quarter of
this year marked by several notable achievements, including the
closing on our global commercial licensing agreement with CSL
Behring in hemophilia B and the recent presentation of 52-week data
on all patients in our HOPE-B pivotal study showing durable and
sustained increases in Factor IX levels within the non-hemophiliac
range1, as well as significant reductions in both bleeding events
and usage of replacement therapy after a one-time administration of
etranacogene dezaparvovec,” stated Matt Kapusta, chief executive
officer at uniQure. “Additionally, we continued to make strong
progress in our U.S. Phase I/II study of AMT-130 in Huntington’s
disease, including the completion of patient enrollment in the
first dose cohort and the initiation of dosing in the second
cohort.”
“We also recently hosted a Research and
Development Day where we disclosed four new promising research
programs that complemented our expanding focus in neurological
disease and larger patient populations, including the acquisition
of Corlieve Therapeutics and its gene therapy program targeting
temporal lobe epilepsy that builds on our expanding focus in
serious neurological diseases,” he added. “These investments
deliver on our commitment to leverage our strong cash position to
invest in new research programs and enabling technologies with the
potential to drive significant value. In the second half of 2021,
we expect to continue our progress across all programs, including
availability of 78-week data from the HOPE-B study, initiating
patient enrollment for our open-label European study of AMT-130 in
Huntington’s disease, and announcing initial data on the first four
randomized Huntington’s disease patients in the ongoing U.S. Phase
I/II study.”
1 Srivastava et al, Haemophilia. 2020;26(Suppl
6):1–158.
Recent Company Progress
- Advancing late-stage development of
etranacogene dezaparvovec (AMT-061) for the treatment of hemophilia
B
- In June 2021, the Company announced
initial 52-week post-administration Factor IX data and annualized
bleeding rates (ABR) in the HOPE-B pivotal trial of etranacogene
dezaparvovec in severe to moderately severe hemophilia B patients.
Sustained increase in FIX levels was observed with a mean FIX
activity of 41.5% of normal compared to 39.0% at 26-weeks post
administration. During the 52-week period after dosing, patients
reported an 80% reduction in total bleeding events requiring
treatment, an 85% reduction in spontaneous bleeding events
requiring treatment, and a 96% reduction in annualized usage of FIX
replacement therapy.
- The Company expects all patients in
the HOPE-B clinical trial to complete their 78-week follow-up
visits by the end of the third quarter of 2021, and expects the BLA
will be submitted in the first quarter of 2022.
- The Company announced the closing
of the transaction with CSL Behring for its hemophilia B gene
therapy following the expiration of the waiting period under the
Hart-Scott-Rodino Antitrust Improvements Act of 1976 on May 5,
2021. In accordance with the agreement, the upfront payment of $450
million was paid to uniQure on May 7, 2021.
- Advancing the clinical development
of AMT-130 for the treatment of Huntington’s disease
- In May 2021, the Company announced
the completion of patient enrollment of the first dose cohort of
ten patients in its ongoing Phase I/II study of AMT-130 in
Huntington’s disease. The U.S. study is a multi-center,
double-blinded, randomized and controlled trial to assess the
safety and efficacy of AMT-130. The Data Safety Monitoring Board
recommended proceeding with patient enrollment in the second dose
cohort after reviewing nine-month safety data from the first two
enrolled patients, six-month safety data from the next two enrolled
patients, and the 30-day safety data from the remaining six
patients.
- In June 2021, the Company announced
the first two patient procedures were completed in the second dose
cohort of Phase I/II clinical trial. Enrollment of the next two
patient procedures will take place after a planned DSMB meeting
expected to be held later in the third quarter of 2021.
- Initial data from the first four
enrolled patients in this first-in-human AAV-gene therapy trial are
anticipated by year-end 2021. The Company also remains on track to
initiate an open label, Phase Ib/II clinical study of AMT-130 in
Europe with enrollment beginning in the second half of 2021.
- Expanding the pipeline and
strengthening the platform
- In June, the Company announced the
planned acquisition of Corlieve Therapeutics, a pre-clinical gene
therapy company based in France. Corlieve’s lead program, now
designated as AMT-260, employs miRNA silencing technology to
suppress aberrantly expressed kainite receptors in the hippocampus
of patients with temporal lobe epilepsy (TLE). TLE represents a
large unmet clinical need affecting approximately 1.3 million
people in the U.S. and Europe alone, of which approximately 800,000
patients are unable to adequately control acute seizures with
currently approved anti-epileptic therapies. The transaction is
subject to customary closing conditions as well as review by the
French Ministry of Economy, Finance and Recovery. The acquisition
is expected to close in the third quarter of 2021.
- In June, the Company hosted a
virtual Research & Development Day to highlight new investments
in its expanding gene therapy pipeline focusing on central nervous
system and rare, liver-directed disorders, as well as new
advancements in platform technology and manufacturing. Three new,
internally developed pre-clinical programs were announced in
Parkinson’s disease (AMT-210), amyotrophic lateral sclerosis (ALS)
(AMT-160), and autosomal dominant Alzheimer’s disease (AMT-240).
Additional highlights from the event included updates on the
clinical development of AMT-130 in Huntington’s disease and
advancements in the ongoing preclinical development of AMT-150 for
spinocerebellar ataxia 3 and AMT-190 for Fabry disease.
- In February
2021, the Company began an expansion of the Amsterdam site to
include a cleanroom capable of manufacturing cGMP materials at a
500-liter scale and additional laboratories to support the
expansions of research and development activities.
- Expansion of the leadership team
- In May 2021, Pierre Caloz joined
the leadership team as Chief Operating Officer overseeing all
manufacturing operations, global CMC development and innovation,
supply chain and facilities. Mr. Caloz joins the Company with
nearly 20 years of global operations experience in the biopharma
industry, including CSL Behring, Merck-Serono, Abgenix and
Amgen.
- On June 15, 2021, Christian Klemt
was appointed as Chief Financial Officer. Mr. Klemt was the
Company’s Chief Accounting Officer from August 2017 to June 2021,
and he will continue to serve as general manager of the Amsterdam
site.
- Strong cash position to advance the
Company’s programs
- As of June 30, 2021, the Company’s
cash position was $677.3 million. The Company expects cash and cash
equivalents will be sufficient to fund operations into the first
half of 2024, excluding any of the $1.6 billion in future
milestones or royalties on net sales the Company may receive under
the CSL Behring Agreement. The receipt of approximately $300
million in milestone payments relates to regulatory submissions,
first commercial sales and certain development milestones would be
expected to extend the cash runway by approximately 18 months.
Upcoming Investor Events (each to be
conducted virtually)
- Citi’s Annual Biopharma Virtual
Conference, September 8-10, 2021
- Wells Fargo Virtual Healthcare
Conference, September 9-10, 2021
- SVB Leerink CybeRx Series Event,
September 21-23, 2021
- Cantor Fitzgerald Virtual Global
Healthcare Conference, September 27-29, 2021
- Chardan’s 5th Annual Genetic
Medicines Conference, October 4-5, 2021
Financial Highlights
Cash Position: As of June 30,
2021, the Company held cash and cash equivalents of $677.3 million,
compared to $244.9 million as of December 31, 2020. Upon the CSL
Behring Agreement becoming fully effective on May 6, 2021, the
Company received $462.4 million of payments. Additionally, in
January 2021, the Company and Hercules entered into an amended debt
facility agreement increasing the aggregate principle to up to
$135.0 million, of which the Company drew down an additional $35.0
million for a total of $70.0 million outstanding under the facility
as of June 30, 2021. In March 2021, the Company entered into an
Open Market Sale Agreement with SVB Leerink LLC providing for the
sale of up to $200.0 million of its ordinary shares from time to
time in ‘at-the-market’ offerings under a shelf registration
statement. Pursuant to this agreement, as of April 30, 2021, the
Company had sold 921,730 ordinary shares for gross proceeds of
approximately $29.6 million.
Revenues: Revenue for the three
months ended June 30, 2021, were $463.9 million, compared to $1.5
million during the same period in 2020. The increase is a
result of $462.4 million of license revenue recognized from
the CSL Behring Agreement.
Cost of Contract Revenues: Cost
of contract revenues for the three months ended June 30, 2021, was
$23.2 million compared to nil for the same period in 2020. Costs
incurred in 2021 are associated with the license revenue recognized
under the CSL Behring Agreement.
R&D Expenses: Research and
development expenses were $32.8 million for the three months ended
June 30, 2021, compared to $28.4 million during the same period in
2020. The change was primarily related to recruitment of personnel
to support the development of product candidates, advancing the
clinical developments of Huntington’s disease, and increased
activities associated with preclinical product candidates.
SG&A Expenses: Selling,
general and administrative expenses were $17.3 million for the
three months ended June 30, 2021, compared to $11.5 million during
the same period in 2020. The change was primarily related to costs
incurred for financial advisory fees in relation to the licensing
transaction with CSL Behring, increases in personnel and consulting
expenses to support growth, and increased share-based
compensation.
Other non-operating items, net:
Other income, net was $4.7 million for the three months ended June
30, 2021, compared to other expense, net of $4.3 million during the
same period in 2020. The increase in income is primarily due to
foreign currency gains in the current period compared to a net
foreign currency loss in the same period in 2020.
Net Loss: The net income for
the three months ended June 30, 2021, was $399.5 million, or $8.68
basic net income per share and $8.51 diluted net income per share,
compared to a loss of $42.6 million, or $0.96 basic and diluted
loss per share during the same periods in 2020.
About uniQure
uniQure is delivering on the promise of gene
therapy – single treatments with potentially curative results. We
are leveraging our modular and validated technology platform to
rapidly advance a pipeline of proprietary gene therapies to treat
patients with hemophilia B, Huntington's disease, Fabry disease,
spinocerebellar ataxia Type 3 and other
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," "estimate," "expect,"
"goal," "intend," "look forward to", "may," "plan," "potential,"
"predict," "project," "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, uniQure’s expectations
its plans to announce data from the HOPE-B pivotal trial, patient
enrollment in the second cohort of AMT-130, the initiation of an
open-label European study of AMT-130, the announcement of data on
the first four patients in the Phase I/II study of AMT-130, the
submission of the regulatory filing for marketing approval of
etranacogene dezaparvovec, the potential financial compensation we
may be paid pursuant to our agreement with CSL Behring, or other
updates on the research pipeline, and our plans to advance or
expand our pipeline, accelerate research, identify business
development opportunities, invest in technology, or scale our
manufacturing capabilities. uniQure’s actual results could differ
materially from those anticipated in these forward-looking
statements for many reasons, including, without limitation, risks
associated with the impact of the ongoing COVID-19 pandemic 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
uniQure’s periodic securities filings, including its Annual Report
on Form 10-K filed March 1, 2021. Given these risks, uncertainties
and other factors, you should not place undue reliance on these
forward-looking statements, and uniQure 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
|
|
|
|
|
|
June 30, |
|
December 31, |
|
2021 |
|
2020 |
|
|
(in thousands, except share and per share
amounts) |
|
Current assets |
|
|
|
|
Cash and cash equivalents |
$ |
677,330 |
|
$ |
244,932 |
|
Accounts receivables |
|
2,705 |
|
|
6,618 |
|
Prepaid expenses |
|
10,861 |
|
|
4,337 |
|
Other current assets and receivables |
|
3,328 |
|
|
3,024 |
|
Total current assets |
|
694,224 |
|
|
258,911 |
|
Non-current assets |
|
|
|
|
Property, plant and equipment, net |
|
38,371 |
|
|
32,328 |
|
Operating lease right-of-use assets |
|
26,782 |
|
|
26,086 |
|
Intangible assets, net |
|
2,637 |
|
|
3,361 |
|
Goodwill |
|
525 |
|
|
542 |
|
Deferred tax assets |
|
15,965 |
|
|
16,419 |
|
Other non-current assets |
|
5,760 |
|
|
2,748 |
|
Total non-current assets |
|
90,040 |
|
|
81,484 |
|
Total assets |
$ |
784,264 |
|
$ |
340,395 |
|
Current liabilities |
|
|
|
|
Accounts payable |
$ |
16,817 |
|
$ |
3,772 |
|
Accrued expenses and other current liabilities |
|
23,526 |
|
|
18,038 |
|
Current portion of operating lease liabilities |
|
5,752 |
|
|
5,524 |
|
Total current liabilities |
|
46,095 |
|
|
27,334 |
|
Non-current liabilities |
|
|
|
|
Long-term debt |
|
70,780 |
|
|
35,617 |
|
Operating lease liabilities, net of current portion |
|
30,534 |
|
|
30,403 |
|
Other non-current liabilities |
|
3,247 |
|
|
3,136 |
|
Total non-current liabilities |
|
104,561 |
|
|
69,156 |
|
Total liabilities |
$ |
150,656 |
|
$ |
96,490 |
|
Shareholders' equity |
|
|
|
|
Total shareholders' equity |
|
633,608 |
|
|
243,905 |
|
Total liabilities and shareholders' equity |
$ |
784,264 |
|
$ |
340,395 |
|
uniQure N.V.
UNAUDITED CONSOLIDATED STATEMENTS OF
OPERATIONS
|
|
|
|
|
|
|
|
Three months ended June 30, |
|
|
|
|
2021 |
|
|
|
2020 |
|
|
|
|
(in thousands, except share and per share
amounts) |
|
|
Total revenues |
$ |
463,868 |
|
|
$ |
1,535 |
|
|
|
Operating expenses: |
|
|
|
|
|
Cost of contract revenues |
|
(23,178 |
) |
|
|
- |
|
|
|
Research and development expenses |
|
(33,401 |
) |
|
|
(28,401 |
) |
|
|
Selling, general and administrative expenses |
|
(16,645 |
) |
|
|
(11,511 |
) |
|
|
Total operating expenses |
|
(73,224 |
) |
|
|
(39,912 |
) |
|
|
Other income |
|
7,590 |
|
|
|
669 |
|
|
|
Other expense |
|
(226 |
) |
|
|
(500 |
) |
|
|
Loss from operations |
|
398,008 |
|
|
|
(38,208 |
) |
|
|
Non-operating items, net |
|
4,718 |
|
|
|
(4,343 |
) |
|
|
Loss before income tax expense |
|
402,726 |
|
|
|
(42,551 |
) |
|
|
Income tax expense |
|
(3,258 |
) |
|
|
- |
|
|
|
Net loss |
$ |
399,468 |
|
|
$ |
(42,551 |
) |
|
|
Earnings per ordinary share - basic |
|
|
|
|
|
Basic net income/(loss) per ordinary share |
$ |
8.68 |
|
|
$ |
(0.96 |
) |
|
|
Earnings per ordinary share - diluted |
|
|
|
|
|
Diluted net income/(loss) per ordinary share |
$ |
8.51 |
|
|
$ |
(0.96 |
) |
|
|
Weighted average shares - basic |
|
46,037,900 |
|
|
|
44,387,463 |
|
|
|
Weighted average shares - diluted |
|
46,929,870 |
|
|
|
44,387,463 |
|
|
|
|
|
|
|
|
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