uniQure Announces First 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 first quarter
of 2021 and highlighted recent progress across its business.
“We have made great strides with our clinical
trial execution across both our hemophilia and Huntington’s disease
programs, with all patients in the HOPE-B pivotal trial completing
their 52-week follow-up visits and all ten patients in the first
dose cohort of our U.S. clinical trial in Huntington’s disease
completing their procedures,” stated Matt Kapusta, chief executive
officer at uniQure. “Looking at important near-terms catalysts, we
look forward to announcing top-line, 52-week follow-up data from
the HOPE-B pivotal trial, initiating our collaboration with CSL
Behring and holding an R&D Day to provide updates on the
research pipeline, including new product candidates,” he added. “In
the second half of the year, we expect to begin patient enrollment
in the second cohort of AMT-130 and to initiate our open-label
European study of AMT-130. Later this year, we expect to submit the
BLA for etranacogene dezaparvovec and announce early data on the
first four patients in the Phase I/II study of AMT-130.”
Recent Company Progress
- Advancing late-stage development of
etranacogene dezaparvovec (AMT-061) for the treatment of hemophilia
B
- The Company expects to announce
top-line data during the second quarter of 2021 related to
additional clinical trial endpoints in the HOPE-B pivotal trial of
etranacogene dezaparvovec in severe to moderately severe hemophilia
B patients, including 52-week annualized bleeding rates and FIX
activity.
- 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.
- In April 2021, the FDA removed the
clinical hold on the Company’s hemophilia B gene therapy program
regarding a diagnosis of hepatocellular carcinoma (HCC), a form of
liver cancer, in one patient in the HOPE-B trial. This followed a
review of data from a comprehensive investigation that found it was
highly unlikely etranacogene dezaparvovec was the cause of the HCC
in this patient.
- Advancing the clinical development
of AMT-130 for the treatment of Huntington’s disease
- The ten-patient, first dose cohort
of the Phase I/II clinical trial of AMT-130 was fully enrolled in
the second quarter of 2021. The second, higher-dose
cohort is expected to begin enrollment in the third quarter of 2021
after a review by the independent Data Safety Monitoring Board
(DSMB). Early imaging and biomarker data from the first four
enrolled patients in this first-in-human AAV-gene therapy trial in
Huntington’s disease are anticipated by year-end 2021.
- In February 2021, the DSMB
recommended proceeding with patient enrollment in the Phase I/II
clinical trial of AMT-130 after finding no significant safety
concerns based on its review of the six-month data from the first
two enrolled patients and the 90-day data from the next two
enrolled patients.
- The Company also announced plans 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 February 2021, the Company began an expansion of its
Amsterdam site to build additional laboratories to support growing
research and development activities, as well as a cleanroom
designed for manufacturing cGMP materials at a 500-liter
scale.
- The Company plans to host a virtual
Research & Development Day on June 22, 2021 to highlight new
investments in its expanding gene therapy pipeline focusing on CNS
and rare, liver-directed disorders, as well as new advancements in
platform technology and manufacturing.
- Expansion of the leadership team
- Pierre Caloz will join the
leadership team as Chief Operating Officer, effective May 17, 2021,
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. Most recently, he served as Senior Vice President and
General Manager of European Union and Asia Pacific Operations at
CSL Behring where he managed a team of more than 4,000 FTEs and an
operating budget of more than EUR 1 billion. Mr. Caloz earned
a BSc degree from the University of Geneva, a MSc degree from Swiss
Federal Institute of Technology, and an EMBA from the Ashridge
Business School.
- Strong cash position to advance the
Company’s programs
- As of March 31, 2021, the Company’s
cash position was $260.8 million, which did not include any
financial impact associated with the commercialization and license
agreement with CSL Behring. Including the receipt from CSL Behring
of the $450 million upfront payment, the Company expects cash and
cash equivalents will be sufficient to fund operations into the
second half of 2024.
Upcoming Investor Events (each to be
conducted virtually)
- 2021 RBC Capital Markets Global
Healthcare Conference, May 18-20, 2021
- Jefferies Virtual Healthcare
Conference, June 1-4, 2021
- 42nd Annual Goldman Sachs Global
Healthcare Conference, June 8-10, 2021
- Virtual Raymond James Human Health
Innovations Conference, June 21-23, 2021
- uniQure Research & Development
Day, June 22, 2021
Financial Highlights
Cash Position: As of March 31,
2021, the Company held cash and cash equivalents of $260.8 million,
compared to $244.9 million as of December 31, 2020. 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 March 31, 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. Through March 31, 2021, the Company sold 859,885
ordinary shares pursuant to this agreement for gross proceeds of
approximately $28.7 million.
Revenues: Revenue for the three
months ended March 31, 2021 was $0.5 million, compared to $0.1
million during the same period in 2020.
R&D Expenses: Research and
development expenses were $32.7 million for the three months ended
March 31, 2021, compared to $26.0 million during the same period in
2020. The change was primarily related to increased activities
associated with our ongoing clinical studies of Huntington’s
disease, increased activity in programs in preclinical development
and the recruitment of personnel to support the development of our
product candidates.
SG&A Expenses: Selling,
general and administrative expenses were $12.4 million for the
three months ended March 31, 2021, compared to $9.1 million during
the same period in 2020. The change was primarily related to
increases in personnel and consulting expenses to support our
growth, increases in professional fees, and increased share-based
compensation.
Other non-operating items, net:
Other income, net was $3.1 million for the three months ended March
31, 2021, compared to other income, net of $6.5 million during the
same period in 2020. The decrease is primarily due to the
termination of the Bristol-Myers Squibb warrants in December 2020,
resulting in no recognition of changes in fair value of the
derivative financial liability in the current period compared to a
recognized in the same period in 2020.
Net Loss: The net loss for the
three months ended March 31, 2021 was $41.6 million, or $0.91 loss
per share, compared to $28.0 million, or $0.63 loss per share
during the same period 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 employment of a Chief Operating
Officer, the potential financial compensation we may be paid
pursuant to our agreement with CSL Behring, the planned R&D Day
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: |
|
|
|
|
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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
MaloneDirect: 339-970-7558Mobile:339-223-8541
t.malone@uniQure.com |
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uniQure N.V.
UNAUDITED CONSOLIDATED BALANCE
SHEETS
|
|
|
|
|
March
31, |
|
December
31, |
2021 |
|
2020 |
|
|
|
(in
thousands, except share and per share amounts) |
Current assets |
|
|
|
Cash and cash equivalents |
$ |
260,813 |
|
$ |
244,932 |
Accounts
receivables |
|
5,445 |
|
|
6,618 |
Prepaid
expenses |
|
9,186 |
|
|
4,337 |
Other
current assets |
|
6,886 |
|
|
3,024 |
Total current assets |
|
282,330 |
|
|
258,911 |
Non-current assets |
|
|
|
Property,
plant and equipment, net |
|
33,862 |
|
|
32,328 |
Operating
lease right-of-use assets |
|
25,313 |
|
|
26,086 |
Intangible
assets, net |
|
2,908 |
|
|
3,361 |
Goodwill |
|
518 |
|
|
542 |
Restricted
cash |
|
2,716 |
|
|
2,748 |
Deferred tax
asset |
|
16,206 |
|
|
16,419 |
Total non-current assets |
|
81,523 |
|
|
81,484 |
Total assets |
$ |
363,853 |
|
$ |
340,395 |
Current liabilities |
|
|
|
Accounts
payable |
$ |
5,749 |
|
$ |
3,772 |
Accrued
expenses and other current liabilities |
|
20,896 |
|
|
18,038 |
Current
portion of operating lease liabilities |
|
5,457 |
|
|
5,524 |
Total current liabilities |
|
32,102 |
|
|
27,334 |
Non-current liabilities |
|
|
|
Long-term
debt |
|
70,467 |
|
|
35,617 |
Operating
lease liabilities, net of current portion |
|
29,487 |
|
|
30,403 |
Other
non-current liabilities |
|
3,107 |
|
|
3,136 |
Total non-current liabilities |
|
103,061 |
|
|
69,156 |
Total liabilities |
$ |
135,163 |
|
$ |
96,490 |
Shareholders' equity |
|
|
|
Total shareholders' equity |
|
228,690 |
|
|
243,905 |
Total liabilities and shareholders' equity |
$ |
363,853 |
|
$ |
340,395 |
|
|
|
|
uniQure N.V.
UNAUDITED CONSOLIDATED STATEMENTS OF
OPERATIONS
|
|
|
|
|
Three months ended March 31, |
|
2021 |
|
2020 |
|
|
|
(in
thousands, except share and per share amounts) |
Total revenues |
$ |
454 |
|
|
$ |
104 |
|
Operating expenses: |
|
|
|
Research and
development expenses |
|
(32,656 |
) |
|
|
(26,013 |
) |
Selling,
general and administrative expenses |
|
(12,375 |
) |
|
|
(9,072 |
) |
Total operating expenses |
|
(45,031 |
) |
|
|
(35,085 |
) |
Other
income |
|
352 |
|
|
|
857 |
|
Other
expense |
|
(233 |
) |
|
|
(339 |
) |
Loss
from operations |
|
(44,458 |
) |
|
|
(34,463 |
) |
Non-operating items, net |
|
3,115 |
|
|
|
6,464 |
|
Loss
before income tax expense |
|
(41,343 |
) |
|
|
(27,999 |
) |
Income tax
expense |
|
(213 |
) |
|
|
- |
|
Net
loss |
$ |
(41,556 |
) |
|
$ |
(27,999 |
) |
|
|
|
|
Basic and
diluted net loss per ordinary share |
$ |
(0.91 |
) |
|
$ |
(0.63 |
) |
Weighted
average shares used in computing basic and diluted net loss per
ordinary share |
|
45,468,485 |
|
|
|
44,279,456 |
|
|
|
|
|
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