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 2020 and highlighted recent progress across its business.
“The first half of 2020 has been extremely
productive for uniQure with significant achievements across our
clinical-stage programs,” stated Matt Kapusta, chief executive
officer at uniQure. “The initiation of patient dosing in our Phase
I/II study of AMT-130 marked the first AAV gene therapy for
patients with Huntington’s disease to enter the clinic, and we
remain highly focused on advancing development of this important
program. We also completed the dosing phase of our HOPE-B pivotal
study for etranacogene dezaparvovec in patients with hemophilia B
and remain on track to present topline data on all patients by the
end of 2020 and submit a BLA in 2021.”
“Importantly, in June we announced a landmark
global license agreement with CSL Behring to develop and
commercialize our hemophilia B gene therapy candidate, with uniQure
eligible following regulatory approval to receive more than $2
billion of economics, including upfront cash, milestone, and
royalty payments,” he added. “The collaboration ideally positions
us to deliver our highly innovative gene therapy to the largest
number of hemophilia B patients as quickly as possible, and we
expect it will provide an exceptionally strong balance sheet to
aggressively expand our pipeline, invest in technology innovation
and further scale our manufacturing capabilities.”
Recent Company Progress
- Advancing late-stage development of etranacogene dezaparvovec
(AMT-061) for the treatment of hemophilia B
- In June 2020, the Company and CSL Behring entered into a
licensing agreement providing CSL Behring with exclusive global
rights to etranacogene dezaparvovec, the Company’s
investigational gene therapy for patients with hemophilia B.
Under the terms of the agreement, which is subject first to
regulatory review in the United States, Australia and the United
Kingdom, the Company will receive a $450 million upfront cash
payment and be eligible to receive up to $1.6 billion in payments
based on regulatory and commercial milestones. the Company will
also be eligible to receive tiered double-digit royalties in a
range of up to a low-twenties percentage of net product sales
arising from the collaboration. The agreement is one of the
largest gene therapy deals announced to date and leverages CSL
Behring’s global hematology capabilities and infrastructure to
benefit hemophilia B patients around the world.
- The Company continues to conduct the HOPE-B pivotal trial of
etranacogene dezaparvovec and remains on track to provide 26-weeks
of Factor IX data for all 54 patients before the end of this
year.
- Manufacturing process validation for etranacogene dezaparvovec
continues in anticipation of the submission of a biologics license
application in the United States and a marketing authorization
application in the European Union, which the Company expects will
be submitted in 2021.
- Advancing AMT-130 into clinical development for the treatment
of Huntington’s disease
- In June 2020, the first two patients were enrolled in the Phase
I/II clinical trial of AMT-130 for the treatment of
Huntington’s disease. The Phase I/II study is a double-blinded,
randomized clinical trial being conducted in the United
States.
- The next two patients are expected to be enrolled in the fourth
quarter of 2020, following a 90-day safety evaluation by the
clinical trial’s Data Safety Monitoring Board.
- Advancing research pipeline of gene therapy candidates toward
the clinic
- The Company expects to initiate IND-enabling studies for its
Spinocerebellar Ataxia Type 3 (SCA3) program in the third quarter
of 2020, which is expected to support the submission of an IND
application planned for 2021. SCA3 patients experience brain
degeneration that results in movement disorders, rigidity, muscular
atrophy and paralysis. There is currently no treatment available
that slows the progressive course of this lethal disease. AMT-150
is a one-time administered AAV gene therapy incorporating the
Company’s proprietary miQURE™ silencing technology, similar to the
technology used in the Company’s AMT-130 program for the treatment
for Huntington’s disease, and is designed to halt ataxia in early
manifest SCA3 patients.
- In May 2020, the Company had a significant presence at the
American Society of Gene and Cell Therapy (ASGCT) Virtual 2020
Annual Meeting, with 22 data presentations: five virtual oral
presentations and 17 poster presentations.
- The Company presented in vivo preclinical data on AMT-150 for
the treatment of SCA3 in six, non-human primates (NHP) that
received a one-time injection of AMT-150 via the cisterna
magna. The goal of the study was to assess expression and
distribution with samples taken after eight weeks showing
widespread transduction of the brain and spinal cord.
- The Company also presented additional preclinical studies
evaluating AMT-150 in SCA3 mouse models and human induced
pluripotent stem cell (iPSC)-derived neurons and astrocytes which
represent the most disease-relevant cell type for therapeutic
targeting of AMT-150. The goal of these studies was to evaluate
potential off-target effects of the AAV5-miATXN3. A clear
dose-dependent expression of miATXN3 was observed in the
iPSC-derived neurons and astrocytes. Mature miATXN3 molecules were
also associated with extracellular vesicles that strongly
correlated with the dose and miATXN3 expression, suggesting the
potential therapeutic spread of the engineered miATXN3.
Additionally, AMT-150 demonstrated ATXN3 knockdown in human neurons
and various SCA3 mouse models with subsequent neuropathology
improvement.
- On June 24, 2020, the Company held a conference call in which
it announced its plans to aggressively advance and expand its
pipeline by accelerating internal research with a focus on CNS
disorders and other rare, liver-directed disorders, as well as by
evaluating business development opportunities. Additionally, the
Company announced plans to invest in technology innovation and
continue to further scale manufacturing capabilities.
- Appointment of experienced R&D expert to the board
- In June 2020, Leonard E. Post, Ph.D. was appointed to the
Company’s Board of Directors and will serve as Chair of the
Company’s Research & Development Committee. Dr. Post has more
than 35 years of research and development leadership, serving
currently as Chief Scientific Officer of Vivace Therapeutics and
its sister company, Virtuoso Therapeutics, both of which are
developing oncology therapeutics. From 2010 until 2016, Dr. Post
served in various positions at BioMarin (NASDAQ:
BMRN), including Chief Scientific Officer, during which
time he oversaw the initiation of BioMarin's first gene therapy
project for hemophilia A. Prior to that, Dr. Post served as
Chief Scientific Officer of LEAD Therapeutics, Senior Vice
President of Research & Development at Onyx Pharmaceuticals and
Vice President of Discovery Research at Parke-Davis
Pharmaceuticals. He is also currently an advisor to Canaan
Partners.
- Strong cash position to advance the Company’s programs
- As of June 30, 2020, the Company’s cash position was $314.3
million, which is expected to fund the Company operations into
2022. This does not include any financial impact associated with
the pending collaboration and license agreement with CSL Behring.
Assuming the receipt from CSL Behring of the $450 million payment
due at the closing, 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)
- Citi’s 15th Annual BioPharma Virtual Conference, September
9-10, 2020
- Wells Fargo Global Healthcare Conference, September 9-10,
2020
- H.C. Wainwright 22nd Annual Global Investment Conference,
September 14-15, 2020
- Cantor Fitzgerald Virtual Global Healthcare Conference,
September 15-17, 2020
- SVB Leerink CybeRx Series: Rare & Genetics, October 1,
2020
- Jefferies Virtual Gene Therapy/Editing Summit, October 1-2,
2020
- Chardan’s Virtual 4th Annual Genetic Medicines Conference,
October 5-6, 2020
Financial Highlights
Cash Position: As of June 30,
2020, the Company held cash and cash equivalents of $314.3 million,
compared to $342.0 million as of March 31, 2020.
Revenues: Revenue for the three
months ended June 30, 2020 was $1.5 million, compared to $2.5
million during the same period in 2019.
R&D Expenses: Research and
development expenses were $28.4 million for the three months ended
June 30, 2020, compared to $24.2 million during the same period in
2019. The change was primarily related to increased activities
associated with our ongoing clinical studies of etranacogene
dezaparvovec and AMT-130, increased share-based compensation, the
recruitment of personnel to support the development of our product
candidates and increased investments in our facilities.
SG&A Expenses: Selling,
general and administrative expenses were $11.5 million for the
three months ended June 30, 2020, compared to $7.9 million during
the same period in 2019. The change was primarily related to
increases in personnel and consulting expenses, professional fees,
and share-based compensation expenses.
Other non-operating items, net:
Other expense was $4.3 million for the three months ended June 30,
2020, compared to other expense of $2.1 million during the same
period in 2019.
Net Loss: The net loss for the
three months ended June 30, 2020 was $42.6 million, or $0.96 loss
per share, compared to $31.4 million, or $0.83 loss per share
during the same period in 2019.
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
about its cash runway, certain statements concerning the
transaction between CSL Behring and uniQure, including whether the
parties will successfully complete the review under applicable
antitrust laws or otherwise close the transaction, whether uniQure
will receive more than $2 billion of economics, including upfront
cash, milestone, and royalty payments, whether uniQure will expand
its pipeline, invest in technology innovation or further scale its
manufacturing capabilities, whether uniQure will submit a biologics
license application in the United States or a marketing
authorization application in the European Union for etranacogene
dezaparvovec in 2021 or ever, whether uniQure will present topline
data on all patients in its Phase III clinical trial of
etranacogene dezaparvovec by the end of 2020 or ever, whether the
next two patients will be enrolled in uniQure’s Phase I/II clinical
trial of AMT-130 for the treatment of Huntington’s disease in
the fourth quarter of 2020 or ever, whether uniQure initiates
IND-enabling studies for its Spinocerebellar Ataxia Type 3 (SCA3)
program in the third quarter of 2020 or ever, and whether uniQure
submits an IND application for SCA3 in 2021 or ever. 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, the regulatory approval of that transaction, 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 2, 2020 and Quarterly Report on Form 10-Q
filed on April 29, 2020. 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, |
|
2020 |
|
2019 |
|
|
|
|
|
|
(in
thousands, except share and per share amounts) |
|
Current assets |
|
|
|
|
Cash and cash equivalents |
$ |
314,265 |
|
$ |
377,793 |
|
Accounts
receivable and accrued income from related party |
|
222 |
|
|
947 |
|
Prepaid
expenses |
|
4,082 |
|
|
4,718 |
|
Other
current assets |
|
1,066 |
|
|
748 |
|
Total current assets |
|
319,635 |
|
|
384,206 |
|
Non-current assets |
|
|
|
|
Property,
plant and equipment, net |
|
29,301 |
|
|
28,771 |
|
Operating
lease right-of-use assets |
|
26,139 |
|
|
26,797 |
|
Intangible
assets, net |
|
7,087 |
|
|
5,427 |
|
Goodwill |
|
496 |
|
|
496 |
|
Restricted
cash |
|
2,683 |
|
|
2,933 |
|
Total non-current assets |
|
65,706 |
|
|
64,424 |
|
Total assets |
$ |
385,341 |
|
$ |
448,630 |
|
Current liabilities |
|
|
|
|
Accounts
payable |
$ |
4,942 |
|
$ |
5,681 |
|
Accrued
expenses and other current liabilities |
|
13,250 |
|
|
12,457 |
|
Current
portion of operating lease liabilities |
|
5,495 |
|
|
5,865 |
|
Current
portion of deferred revenue |
|
6,153 |
|
|
7,627 |
|
Total current liabilities |
|
29,840 |
|
|
31,630 |
|
Non-current liabilities |
|
|
|
|
Long-term
debt, net of current portion |
|
35,373 |
|
|
36,062 |
|
Operating
lease liabilities, net of current portion |
|
30,279 |
|
|
31,133 |
|
Deferred
revenue, net of current portion |
|
23,048 |
|
|
23,138 |
|
Derivative
financial instruments related party |
|
832 |
|
|
3,075 |
|
Other
non-current liabilities |
|
464 |
|
|
534 |
|
Total non-current liabilities |
|
89,996 |
|
|
93,942 |
|
Total liabilities |
$ |
119,836 |
|
$ |
125,572 |
|
Shareholders' equity |
|
|
|
|
Total shareholders' equity |
|
265,505 |
|
|
323,058 |
|
Total liabilities and shareholders' equity |
$ |
385,341 |
|
$ |
448,630 |
|
|
|
|
|
|
|
|
|
|
|
uniQure N.V.
UNAUDITED CONSOLIDATED STATEMENTS OF
OPERATIONS
|
|
|
|
|
Three months ended June 30, |
|
2020 |
|
|
2019 |
|
|
(in
thousands, except share and per share amounts) |
Total revenues |
$ |
1,535 |
|
|
$ |
2,474 |
|
Operating expenses: |
|
|
|
Research and
development expenses |
|
(28,401 |
) |
|
|
(24,154 |
) |
Selling,
general and administrative expenses |
|
(11,511 |
) |
|
|
(7,870 |
) |
Total operating expenses |
|
(39,912 |
) |
|
|
(32,024 |
) |
Other
income |
|
669 |
|
|
|
566 |
|
Other
expense |
|
(500 |
) |
|
|
(347 |
) |
Loss
from operations |
|
(38,208 |
) |
|
|
(29,331 |
) |
Non
operating items, net |
|
(4,343 |
) |
|
|
(2,068 |
) |
Net
loss |
$ |
(42,551 |
) |
|
$ |
(31,399 |
) |
|
|
|
|
Basic and
diluted net loss per ordinary share |
$ |
(0.96 |
) |
|
$ |
(0.83 |
) |
Weighted
average shares used in computing basic and diluted net loss per
ordinary share |
|
44,387,463 |
|
|
|
37,824,928 |
|
|
|
|
|
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