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 third quarter
of 2017 and highlighted recent progress across its business.
“In recent months, we have made tremendous
progress advancing our gene therapy candidates in hemophilia B and
Huntington’s disease, and continue to build momentum as we near the
end of the year,” stated Matthew Kapusta, chief executive officer
of uniQure. “Based on recent meetings with the FDA and EMA, we now
have what we believe is a clear pathway to initiate a pivotal study
in 2018 with AMT-061, an AAV5-Padua gene therapy. We believe
AMT-061 has the potential to provide consistent, long-term benefits
to nearly all patients with hemophilia B, without the complications
often associated with immune responses to the capsid.
Preparations for the pivotal study are underway and the
manufacturing of AMT-061 for clinical use has been initiated.”
“Regarding AMT-130 in Huntington’s disease, we
received Orphan Drug Designation from the FDA and presented new
preclinical data demonstrating functional and survival benefit,”
added Mr. Kapusta. “We are also pleased to announce that we
recently completed dosing of a toxicology study that will support
an IND filing in 2018. For the remainder of this year, we
remain focused on the execution across our programs and look
forward to sharing updates on our progress in the near future.”
Third Quarter 2017 and Recent Company
Progress:
• Achieved significant progress with
hemophilia B gene therapy program by introducing the Padua
transgene
- Based on recent meetings with the U.S. Food and Drug
Administration (FDA) and the European Medicines Agency (EMA), the
Company plans to initiate a pivotal study in 2018 with AMT-061, an
AAV5-Padua FIX gene therapy.
- The FDA agreed that AMT-061 will fall under the existing
Breakthrough Therapy Designation and Investigational New Drug (IND)
for AMT-060, and the EMA also agreed that AMT-061 will fall under
the current PRIME designation. The Company also achieved general
agreement with the FDA and EMA on the AMT-061 comparability plan
and proposed pivotal trial design.
- A Good Laboratory Practices (GLP), nonclinical study of AMT-061
performed in non-human primates demonstrated a strong correlation
between dose and human FIX (hFIX) expression levels, as well as the
biological activity of the expressed hFIX protein. AMT-061
demonstrated substantial increases in hFIX clotting activity
compared to AMT-060, and was well-tolerated with no evidence of any
significant toxicological findings.
- uniQure has initiated production of multiple clinical-grade
batches of AMT-061 in its state-of-the-art Lexington, MA
manufacturing facility. Production is at commercial scale and
utilizing current Good Manufacturing Practices. Data reviewed
to date support comparability between AMT-061 and AMT-060.
• Acquired exclusive patent family
covering the use of Padua in gene therapy for hemophilia B
- The intellectual property broadly covers the Padua FIX variant
(R338L) and its use in gene therapy and includes a patent issued in
the U.S., as well as pending patent applications in Europe.
- The patent family was acquired from Professor Paolo Simioni, a
renowned hemophilia expert at the University of Padua, Italy, who
is widely recognized as the first to identify the mutation.
Professor Simioni is serving as an advisor and consultant
exclusively to uniQure for the development of gene therapy products
using his invention.
• Continued progress on AMT-130 gene
therapy in Huntington’s disease
- Preclinical data presented at the European Society of Gene and
Cell Therapy (ESGCT) 25th Anniversary Congress in Berlin, Germany
demonstrated substantial improvement in motor symptoms and median
survival following a single administration of AMT-130 (AAV5-miHTT)
gene therapy in a mouse model with highly aggressive disease.
- The Company has completed the dosing of non-human primates in a
GLP toxicology study of AMT-130. This study will support the
filing of an IND in 2018, with the goal of being the first one-time
administered gene therapy to enter a clinical study for
Huntington’s disease.
- Orphan Drug Designation (ODD) from the FDA was granted to
AMT-130 in October 2017.
• Added talent to leadership team and
Board of Directors
- On August 7, the Company announced the appointments of Dr.
Scott McMillan as Chief Operating Officer and Dr. Sander van
Deventer as Chief Scientific Officer & General Manager,
Amsterdam.
- On September 14, the Company’s shareholders approved the
appointments of Dr. Jeremy Springhorn and Madhavan Balachandran to
the Board of Directors.
• Completed follow-on offering
- On October 27, the Company completed an underwritten public
offering of 5,000,000 of its ordinary shares at a public offering
price of $18.25 per share. The gross proceeds from the offering,
before deducting the underwriting discounts and commissions and
estimated offering expenses, were approximately $91,250,000.
uniQure has granted to the underwriters a 30-day option to purchase
up to 750,000 additional ordinary shares at the public offering
price, less underwriting discounts and commissions.
Upcoming Anticipated
Milestones
- Presentation of nonclinical data of AMT-061 at the 59th
American Society of Hematology (ASH) Meeting & Exposition
- Presentation of up to two year follow-up data of AMT-060 from
an ongoing Phase I/II clinical trial in hemophilia B at the 59th
ASH Meeting & Exposition
- Completion of the comparability analysis for AMT-061 and
submission of data to regulatory agencies for review
- Submission of the IND amendment for AMT-061
- Commencement of patient enrollment in the AMT-061 dose
confirmation and pivotal Phase III studies
- Submission of the IND for AMT-130 in Huntington’s disease
- Initiation of a preclinical therapeutic heart study for
AMT-126, an S100A1 gene therapy targeting congestive heart
failure
Financial Highlights
Cash Position: As of
September 30, 2017, the Company held cash and cash equivalents of
$88.9 million, compared to $132.5 million as of December 31,
2016. The decrease in cash was primarily related to the
advancement of its clinical and preclinical gene therapy targets,
general corporate activities and capital expenditures related to
its facilities.
Revenues: Revenues for the
three months ended September 30, 2017 were $2.3 million compared to
$7.2 million for the same period in 2016. Collaboration revenues
for the third quarter of 2017 were $1.1 million, compared to $6.0
million for the comparable period in 2016. The decrease in
collaboration revenue was primarily due to the termination of the
Chiesi co-development agreement in July 2017, as well as
nonrecurring revenue recognized in the prior year period associated
with the production of AMT-126 product supplies.
R&D Expenses: Research and
development expenses for the three months ended September 30, 2017
were $20.1 million compared to $16.6 million for the same period in
2016. The increase primarily results from non-cash changes in fair
value of the contingent consideration related to the Company’s 2014
acquisition of InoCard.
SG&A Expenses: Selling,
general and administrative expenses for the three months ended
September 30, 2017 were $5.6 million compared to $5.1 million for
the same period in 2016. The increase was primarily related to
larger share-based compensation expenses in the current year
period.
Other income: Other income for
the three months ended September 30, 2017 were $14.4 million
compared to $0.3 million for the same period in 2016. The current
year period includes the full amortization of the outstanding
deferred revenue of $13.8 million following the termination of the
Company’s collaboration with Chiesi in July 2017.
Net Loss: The net loss for the
third quarter of 2017 was $10.2 million, or $0.40 per share,
compared to $15.3 million, or $0.61 per share, for the third
quarter of 2016.
About uniQureuniQure 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 and partnered gene therapies to treat patients with
liver/metabolic, central nervous system and cardiovascular
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, the development of our
gene therapy product candidates, the transition to our AMT-061
product candidate, the success of our collaborations and the risk
of cessation, delay or lack of success of any of our ongoing or
planned clinical studies and/or development of our product
candidates, and the scope of protection provided by our patent
portfolio. Our actual results could differ materially from those
anticipated in these forward-looking statements for many reasons,
including, without limitation, risks associated with our and our
collaborators’ clinical development activities, collaboration
arrangements, corporate reorganizations and strategic shifts,
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
Quarterly Report on Form 10-Q filed on August 8, 2017. Given these
risks, uncertainties and other factors, you should not place undue
reliance on these forward-looking statements, and we assume no
obligation to update these forward-looking statements, even if new
information becomes available in the future.
uniQure Contacts:
FOR INVESTORS:
Maria E. CantorDirect: 339-970-7536Mobile:
617-680-9452m.cantor@uniQure.com
Eva M. MulderDirect: +31 20 240 6103Mobile: +31
6 52 33 15 79e.mulder@uniQure.com
FOR MEDIA:
Tom MaloneDirect: 339-970-7558Mobile:
339-223-8541t.malone@uniQure.com
uniQure N.V. |
|
UNAUDITED CONSOLIDATED BALANCE SHEETS |
|
|
September 30, |
|
December 31, |
2017 |
|
2016 |
|
in thousands, except share and per share
amounts |
Current assets |
|
|
|
Cash and
cash equivalents |
$ |
88,934 |
|
$ |
132,496 |
Accounts
receivables and accrued income |
|
1,945 |
|
|
9,180 |
Prepaid
assets and other current assets |
|
1,436 |
|
|
2,270 |
Total current assets |
|
92,315 |
|
|
143,946 |
Non-current assets |
|
|
|
Property, plant and equipment, net |
|
34,653 |
|
|
35,702 |
Intangible assets and goodwill |
|
9,549 |
|
|
8,789 |
Other
non-current assets |
|
2,469 |
|
|
1,828 |
Total non-current
assets |
|
46,671 |
|
|
46,319 |
Total assets |
$ |
138,986 |
|
$ |
190,265 |
Current liabilities |
|
|
|
Accounts
payable |
$ |
2,987 |
|
$ |
5,524 |
Accrued
expenses and other current liabilities |
|
10,165 |
|
|
9,766 |
Current
portion of long-term debt |
|
6,232 |
|
|
605 |
Current
portion of deferred rent |
|
724 |
|
|
684 |
Current
portion of deferred revenue |
|
4,249 |
|
|
6,142 |
Current
portion of contingent consideration |
|
1,017 |
|
|
- |
Total current liabilities |
|
25,374 |
|
|
22,721 |
Non-current liabilities |
|
|
|
Long-term debt, net of current portion |
|
14,353 |
|
|
19,631 |
Deferred
rent, net of current portion |
|
8,829 |
|
|
6,781 |
Deferred
revenue, net of current portion |
|
67,863 |
|
|
75,612 |
Contingent consideration, net of current portion |
|
2,593 |
|
|
1,838 |
Other
non-current liabilities |
|
367 |
|
|
51 |
Total non-current liabilities |
|
94,005 |
|
|
103,913 |
Total liabilities |
|
119,379 |
|
|
126,634 |
Total shareholders' equity |
|
19,607 |
|
|
63,631 |
Total liabilities and shareholders' equity |
$ |
138,986 |
|
$ |
190,265 |
uniQure N.V. |
|
|
|
UNAUDITED CONSOLIDATED STATEMENTS OF
OPERATIONS |
|
|
|
|
Three months ended September 30, |
|
|
2017 |
|
2016 |
|
|
in thousands, except share and per share
amounts |
|
Total revenues |
$ |
2,260 |
|
|
$ |
7,221 |
|
|
Operating expenses: |
|
|
|
|
Research
and development expenses |
|
(20,103 |
) |
|
|
(16,604 |
) |
|
Selling,
general and administrative expenses |
|
(5,584 |
) |
|
|
(5,113 |
) |
|
Total operating expenses |
|
(25,687 |
) |
|
|
(21,717 |
) |
|
Other
income |
|
14,413 |
|
|
|
336 |
|
|
Other
expense |
|
(261 |
) |
|
|
- |
|
|
Loss from operations |
|
(9,275 |
) |
|
|
(14,160 |
) |
|
Non
operating items, net |
|
(1,248 |
) |
|
|
(935 |
) |
|
Loss before income tax expense |
|
(10,523 |
) |
|
|
(15,095 |
) |
|
Income
tax benefit / (expense) |
|
278 |
|
|
|
(177 |
) |
|
Net loss |
$ |
(10,245 |
) |
|
$ |
(15,272 |
) |
|
|
|
|
|
|
Basic
and diluted net loss per common share |
$ |
(0.40 |
) |
|
$ |
(0.61 |
) |
|
Weighted
average shares used in computing basic and diluted net loss per
common share |
|
25,632,642 |
|
|
|
25,142,660 |
|
|
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