In a release issued under the same headline earlier today by
uniQure N.V. (NASDAQ:QURE), the tables were not included. The
corrected release follows:
uniQure N.V. (NASDAQ:QURE), a leader in human gene therapy,
today announced audited results for the fourth quarter and year
ending December 31, 2015, and provided a corporate overview on its
pipeline programs and operations.
"Over the past several months, we announced proof-of-concept
results from our Phase I/II clinical trials in Sanfilippo B and
hemophilia B. Both studies demonstrated not only encouraging safety
and efficacy data, but also validated our proprietary AAV5
technology and insect cell, baculovirus production system in both
the brain and the liver," said Dan Soland, CEO of uniQure. "In
2016, we will move closer to advancing these lead programs into
pivotal studies and initiate Investigational New Drug
(IND)-enabling studies on additional product candidates that are
expected to enter human clinical trials in 2017. We are
particularly looking forward to sharing longer follow-up data from
our ongoing Phase I/II clinical study of AMT-060 in hemophilia B
and from the Sanfilippo B patients. Since joining uniQure in
December, I am increasingly convinced that our validated technology
platform, commercial-scale manufacturing capabilities and broad
clinical and preclinical programs will continue to drive our
leadership in gene therapy."
Corporate Highlights
Liver/Metabolism Therapeutic Focus Area
- Reported Encouraging Top-line Data from Low-Dose Cohort
in Phase I/II Study of AMT-060 – On January 7, 2016,
uniQure announced preliminary topline results from the first,
low-dose cohort in the ongoing AMT-060-01 Phase I/II Hemophilia B
study. Initial results showed that AMT-060 was generally well
tolerated and the first two patients that completed at least 12
weeks of follow-up showed promising Factor IX expression levels of
5.5% and 4.5% of normal. Additionally, four out of five patients
discontinued recombinant FIX prophylaxis as of January 6, 2016.
These early data validate successful transduction of the liver
using uniQure's proprietary AAV5 vector. uniQure expects to report
results on all five patients from the low-dose cohort at a
scientific conference in the second quarter of 2016.
- Initiated Dosing of AMT-060 in High-Dose
Cohort – On March 14, 2016, the Company announced that the
first patient in the high-dose cohort of the AMT-060-01 trial had
been treated. As of today, 2 patients in the high-dose cohort
have been treated. All 8 patients screened so far in the Phase I/II
trial have tested negative for anti-AAV5 antibodies.
CNS Therapeutic Focus Area
- Positive Results from Phase I/II Study of AMT-110 in
Sanfilippo B – On September 19, 2015, encouraging results
from an academic-sponsored Phase I/II trial in Sanfilippo B using
uniQure's novel AAV5-based gene therapy were presented at the
European Society of Gene and Cell Therapy (ESGCT) in Helsinki,
Finland. In all four patients, researchers verified the restoration
of catalytical activity of the NaGlu protein in the cerebrospinal
fluid (CSF) from 0% at baseline up to 14-17% of normal at three
months and maintained further at 12 months. The trial also
demonstrated that incremental cognitive development was maintained
in all four patients with no progression of brain atrophy detected
via MRI scans. These data validate the effective transmission of
the NaGlu gene into the brain with uniQure's proprietary AAV5 viral
vector. uniQure expects to present 30-month follow-up data
from the four patients in early 2017.
- Sponsorship of Extension Protocol for Phase I/II Study
of AMT-110 to Transition to uniQure – In January 2016,
uniQure and the academic consortium comprised of Institut Pasteur,
the French Muscular Dystrophy Association, Vaincre les Maladies
Lysosomales and Institut National de la Santé et de la Recherche
Médicale (INSERM), executed a term sheet reflecting the license of
certain data and intellectual property from the
consortium-sponsored Phase I/II study of AMT-110 in Sanfilippo B.
uniQure and the consortium are currently negotiating the terms of a
definitive agreement. Additionally, uniQure has assumed the
sponsorship of the Phase I/II extension study, enabling the Company
to continue the follow-up of the four patients treated to
date.
- Phase I Study in Parkinson's Disease to Complete
Enrolling Second Cohort – A Phase I clinical study in
Parkinson's disease led by Krystof Bankiewicz, MD, PhD of the
University of California at San Francisco together with John D.
Heiss, MD, and colleagues from the National Institutes of Heath,
using an AAV-glial cell line-derived neurotrophic factor (GDNF)
product licensed from AMGEN for gene therapy applications, has
completed enrollment of its first of four six-patient cohorts and
is expected to complete the treatment of the second cohort later in
the year.
- Preclinical Proof-of-Concept Achieved in Huntington's
Disease – On March 22, 2016, uniQure announced the
publication of preclinical data in the March 2016 edition of
Molecular Therapy-Nucleic Acids which showed that AMT-130, a novel
AAV5-based gene therapy candidate for Huntington's disease,
achieved preclinical proof-of-concept. The studies, which
were conducted in vitro and in a humanized mouse model,
demonstrated silencing of the mutated Huntingtin gene with
therapeutic microRNAs delivered via an AAV5 vector. uniQure
has selected its lead candidate and has initiated IND-enabling
studies.
Cardiovascular Therapeutic Focus Area
- Four Targets Designated by BMS;
Advancing S100A1 Gene Therapy Towards IND Filing –
On April 6, 2015, uniQure announced a collaboration with
Bristol-Myers Squibb in which the two companies would develop gene
therapies for cardiovascular diseases. In total, the parties may
collaborate on up to ten targets, of which four have been
designated. uniQure and BMS are currently conducting safety
and toxicology studies for the S100A1 gene therapy using uniQure's
proprietary insect-cell, baculovirus expression system and are
advancing towards an IND filing.
Technology and Manufacturing
- Lexington Facility Operational and in Process of
Ramping Up to Commercial-Scale Production – In 2015,
uniQure completed the build-out of its 53,000 sq. ft. fully
scalable gene therapy manufacturing plant in Lexington,
Massachusetts. The facility is operational and currently producing
research batches. Scale-up of AMT-060 production is currently
underway and expected to be completed in 2016 in order to supply
clinical GMP material for a pivotal study in hemophilia B.
- Designation of AAV Capsid Variants with 4D; Initiating
Preclinical Validation – In the fourth quarter of 2015,
uniQure designated specific synthetic AAV capsid variants derived
from 4D's proprietary capsid library for further improvement of
efficacy in both the CNS and Liver/Metabolism Therapeutic Focus
Areas. uniQure will initiate preclinical validation of the
designated capsid variants in non-human primates in 2016.
- Designation of Synthetic Liver-Specific Promoters from
Synpromics Ltd; Initiating Preclinical Validation– As part
of the cooperation and license agreement signed in January 2015,
Synpromics delivered a series of synthetic liver-specific promoters
derived from their rationally-designed promoter library technology.
uniQure will use these promoters to further improve the therapeutic
activity of AAV-vectors in the liver. uniQure will initiate
preclinical testing of these promoters in non-human primates during
2016.
Corporate Finance
- Strong Financial Position – As of December 31,
2015, uniQure had €203.5 million of cash on hand, which is expected
to fund operations into the second half of 2018. To date,
uniQure has received approximately $140 million in upfront and
other consideration from BMS. In addition, on April 7, 2015,
uniQure closed a follow-on public offering of 3,000,000 ordinary
shares at an offering price of $29.50 per share raising a total of
$83.2 million.
Human Resources
- Expanding Key Talent – Over the course of 2015
uniQure has strengthened its management team with experienced
industry leaders in its two key locations in Lexington,
Massachusetts and Amsterdam, including the appointment of Dan
Soland as CEO in December 2015, Matt Kapusta as CFO in January
2015, Dr. Charlie Richard as SVP, R&D Neuroscience in July 2015
and Dr. Deya Corzo, SVP, R&D Liver/Metabolism in July
2015.
Financial Highlights
As of December 31, 2015, the Company held cash and cash
equivalents of €203.5 million, compared with €53.2 million as of
December 31, 2014. The increase was due to the consideration
received from BMS during the period and the Company's follow-on
offering in April 2015, offset in part by cash used in research,
development and general corporate activities.
Revenue for 2015 was €9.4 million, compared with €4.7 million in
2014. These revenues are primarily related to the Company's
collaboration agreements with Bristol-Myers Squibb and Chiesi, as
well as Glybera product sales, which commenced in the third quarter
of 2015.
Cost of goods sold in 2015 was €0.6 million, compared with zero
in 2014. Cost of goods sold includes the cost of Glybera
product sales and amortization of specific Glybera-related license
agreements, which were required to be expensed upon the initiation
of commercialization in the third quarter of 2015.
Research and development expenses were €46.8 million in 2015,
compared to €33.9 million in 2014. The increase is related to the
continuation of uniQure's Phase I/II clinical study of AMT-060 in
hemophilia B, the continued progression of uniQure's other product
candidates and increased activity in the Company's U.S.
facility.
Selling, general and administrative expenses were €19.3 million
in 2015, compared with €11.2 million in 2014. The increase was
primarily due to expenses related to consultants and professional
fees associated with business development, expenses related to the
Company's follow-on offering conducted in April 2015 and other
general and administrative activities.
Other gains/losses were a loss of €0.2 million for 2015,
compared to a gain of €5.8 million in 2014. The loss was
primarily attributable to the impact of foreign currency exchange
rates on the Company's dollar-denominated deposits.
In the third quarter of 2015, the Company incurred a one-time,
non-recurring impairment charge on certain Glybera-related
intangible assets of €11.6 million related to the revision of the
Company's forecasted number of patients treated with Glybera and
certain reimbursement-related developments in Europe.
The net loss for the fourth quarter of 2015 was €14.1 million,
or €0.58 per share, compared with €11.2 million, or €0.62 per
share, for the fourth quarter of 2014. The net loss for the
full years 2015 and 2014 was €71.5 million, or €3.24 per share and
€37.0 million, or €2.16 per share, respectively.
For further financial information for the period ending December
31, 2015, please refer to the financial statements appearing at the
end of this release.
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 and partnered gene therapies to treat
patients with CNS, liver/metabolic 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, statements regarding the
development of our gene therapiesthe 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. Our actual results could differ materially from
those anticipated in these forward-looking statements for many
reasons, including, without limitation, risks associated with
collaboration arrangements, our and our collaborators' clinical
development activities, 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 2014 Annual Report on Form 20-F filed
with the Securities and Exchange Commission on April 25, 2014 and
its 2015 Annual Report on Form 20-F to be filed with the Securities
and Exchange Commission on or about the date hereof. 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
N.V. |
|
Consolidated Statements
of Financial Position |
(€ in thousands) |
|
|
|
|
|
NOTE |
DECEMBER 31,
2014 |
DECEMBER 31,
2015 |
Assets |
|
|
|
Non-current assets |
|
|
|
Goodwill |
2, 5, 6 |
1,342 |
442 |
Intangible assets other than Goodwill |
5, 6 |
16,368 |
7,209 |
Property, plant and equipment |
7 |
19,667 |
23,820 |
Other non-current assets |
8 |
1,022 |
1,142 |
Total non-current assets |
|
38,399 |
32,613 |
Current assets |
|
|
|
Receivables from related parties |
9 |
2,426 |
3,792 |
Trade and other receivables |
9 |
1,542 |
1,730 |
Inventories |
10 |
200 |
435 |
Cash and cash equivalents |
11 |
53,219 |
203,532 |
Total current assets |
|
57,387 |
209,489 |
Total assets |
|
95,786 |
242,102 |
Equity |
|
|
|
Share capital |
|
905 |
1,216 |
Share premium |
|
206,111 |
344,803 |
Other reserves |
|
17,149 |
26,026 |
Accumulated deficit |
|
(181,081) |
(252,561) |
Total equity |
12 |
43,084 |
119,484 |
Liabilities |
|
|
|
Non-current liabilities |
|
|
|
Borrowings |
14 |
16,418 |
13,434 |
Derivative financial instruments- related
parties |
14 |
-- |
530 |
Financial lease liabilities |
15 |
134 |
-- |
Deferred rent |
27 |
5,658 |
5,737 |
Deferred revenue |
17 |
15,387 |
75,852 |
Deferred tax liabilities |
5, 23 |
1,379 |
-- |
Contingent considerations |
5 |
1,454 |
2,687 |
Total non-current
liabilities |
|
40,430 |
98,240 |
Current liabilities |
|
|
|
Trade and other payables |
15, 16 |
9,617 |
11,220 |
Derivative financial instruments - related
parties |
14 |
645 |
992 |
Borrowings |
14 |
-- |
5,124 |
Borrowings - derivative |
14 |
207 |
238 |
Deferred rent |
27 |
475 |
579 |
Deferred revenue |
17 |
1,328 |
6,225 |
Total current
liabilities |
|
12,272 |
24,378 |
Total liabilities |
|
52,702 |
122,618 |
Total equity and
liabilities |
|
95,786 |
242,102 |
uniQure
N.V. |
|
|
|
|
|
Consolidated Statements
of Comprehensive Loss |
(€ in thousands, except share
and per share data) |
|
|
|
|
|
|
TWELVE MONTHS
ENDED |
|
DECEMBER
31, |
|
NOTE |
2013 |
2014 |
2015 |
License revenues |
17 |
440 |
883 |
2.854 |
Collaboration revenues |
17 |
2.503 |
3.802 |
6.271 |
Product sales |
17 |
-- |
-- |
300 |
Total revenues |
|
2.943 |
4.685 |
9.425 |
Cost of goods sold |
18 |
(800) |
-- |
(584) |
Other income |
21 |
585 |
773 |
708 |
Research and development expenses |
18, 19 |
(13.182) |
(33.932) |
(46.781) |
Selling, general and administrative
expenses |
18, 20 |
(11.628) |
(11.167) |
(19.317) |
Impairment of intangible assets |
6 |
-- |
-- |
(11.640) |
Other gains / losses, net |
|
(453) |
5.807 |
(248) |
Total operating costs |
|
(25.478) |
(38.519) |
(77.862) |
Operating result |
|
(22.535) |
(33.834) |
(68.437) |
Finance income |
|
102 |
254 |
549 |
Finance expense |
22 |
(4.387) |
(3.46) |
(4.023) |
Finance
income/(expense)---net |
|
(4.285) |
(3.206) |
(3.474) |
Result before corporate income
tax |
|
(26.82) |
(37.04) |
(71.911) |
Corporate income taxes |
23 |
-- |
-- |
431 |
Net loss |
|
(26.82) |
(37.04) |
(71.480) |
Items that may be subsequently reclassified
to profit or loss |
|
|
|
|
Currency translation differences on foreign
operations |
|
12 |
1.149 |
1.25 |
Other comprehensive income/(loss) |
24 |
12 |
1.149 |
1.25 |
Total comprehensive
loss |
|
(26.808) |
(35.891) |
(70.230) |
Loss per share attributable to the equity
holders of the |
|
|
|
|
Company during the year: |
|
|
|
|
Basic and diluted loss per
share |
25 |
(2,48) |
(2,16) |
(3,24) |
uniQure
N.V. |
|
|
|
|
|
|
|
Consolidated Statements
of Changes in (Deficit)/Equity |
(€ in thousands) |
|
|
|
|
|
|
|
|
|
Total |
|
|
|
|
|
|
Share |
Share |
Other |
Accumulated |
Total |
|
Note |
Capital |
Premium |
Reserves |
Deficit |
Equity/Deficit |
Balance at January 1,
2013 |
|
483 |
114,795 |
1,508 |
(117,234) |
(448) |
Result for the period |
|
-- |
-- |
-- |
(26,820) |
(26,820) |
Other comprehensive income/(loss) |
12 |
-- |
-- |
-- |
12 |
12 |
Total comprehensive
loss |
|
-- |
-- |
-- |
(26,808) |
(26,808) |
Capital contributions |
|
127 |
27,664 |
-- |
-- |
27,791 |
Result on conversion of loan |
|
-- |
-- |
3,005 |
-- |
3,005 |
Share based payment/expense |
13 |
-- |
-- |
2,023 |
-- |
2,023 |
Balance at December 31,
2013 |
|
610 |
142,459 |
6,536 |
(144,041) |
5,564 |
Result for the period |
|
-- |
-- |
-- |
(37,040) |
(37,040) |
Other comprehensive income |
|
-- |
-- |
1,149 |
-- |
1,149 |
Total comprehensive
loss |
|
-- |
-- |
1,149 |
(37,040) |
(35,891) |
Capital contributions |
12 |
295 |
64,320 |
-- |
-- |
64,615 |
Share issuance costs |
|
-- |
(668) |
-- |
-- |
(668) |
Share based payment/expense |
13 |
-- |
-- |
9,464 |
-- |
9,464 |
Balance at December 31,
2014 |
12 |
905 |
206,111 |
17,149 |
(181,081) |
43,084 |
Result for the period |
|
-- |
-- |
-- |
(71,480) |
(71,480) |
Other comprehensive income |
24 |
-- |
-- |
1,250 |
-- |
1,250 |
Total comprehensive
loss |
|
-- |
-- |
1,250 |
(71,480) |
(70,230) |
Capital contributions |
12 |
311 |
139,304 |
-- |
-- |
139,615 |
Share issuance costs |
|
-- |
(612) |
-- |
-- |
(612) |
Share based payment/expense |
13 |
-- |
-- |
7,627 |
-- |
7,627 |
Balance at December 31,
2015 |
12 |
1,216 |
344,803 |
26,026 |
(252,561) |
119,484 |
uniQure
N.V. |
|
|
|
|
|
Consolidated Statements
of Cash Flows |
(€ in thousands) |
|
|
|
|
|
|
|
|
YEARS |
|
|
|
|
ENDED |
|
|
|
|
DECEMBER 31, |
|
|
NOTE |
2013 |
2014 |
2015 |
Cash flow from operating
activities |
|
|
|
|
Net loss |
|
(26,820) |
(37,040) |
(71,480) |
Adjustments for: |
|
|
|
|
Depreciation |
7 |
535 |
1,539 |
3,982 |
Amortization on intangibles assets |
6 |
-- |
-- |
460 |
Impairment of intangible assets |
6 |
-- |
-- |
11,640 |
Change in deferred tax liability |
23 |
-- |
-- |
(479) |
Lease incentive |
27 |
134 |
5,452 |
183 |
Loss/(gain) on derivatives |
14 |
3,446 |
(87) |
(440) |
Loss/(gain) on foreign exchanges |
14 |
49 |
(4,692) |
1,332 |
Changes in contingent consideration |
|
-- |
153 |
1,232 |
Share-based expenses |
13 |
2,023 |
9,464 |
7,627 |
Changes in other non-current assets |
|
(923) |
-- |
-- |
Changes in trade and other receivables |
|
(1,439) |
(952) |
(1,554) |
Movement in inventories |
|
(865) |
664 |
(235) |
Changes in other current liabilities |
|
3,655 |
1,540 |
(1,199) |
Changes in deferred revenue |
17 |
16,958 |
(242) |
65,361 |
Intial recognition of warrants |
3 |
-- |
-- |
2,622 |
Cash (used in) / generated by
operations |
|
(3,247) |
(24,201) |
19,052 |
Interest paid |
|
(889) |
(1,224) |
(1,878) |
Net cash (used in) / generated by
operating activities |
|
(4,136) |
(25,425) |
17,174 |
Cash flow from investing
activities |
|
|
|
|
Purchases of property, plant and
equipment |
7 |
(1,336) |
(15,769) |
(5,671) |
Purchases of intangible assets |
6 |
(4,652) |
(3,367) |
(2,940) |
Interest received |
|
17 |
148 |
91 |
Acquisition of businesses |
5 |
-- |
(1,463) |
-- |
Net cash used in investing
activities |
|
(5,971) |
(20,451) |
(8,520) |
Cash flow from financing
activities |
|
|
|
|
Proceeds from shares issued |
12 |
14,294 |
63,097 |
138,342 |
Share issuance cost |
12 |
-- |
(668) |
(612) |
Convertible loans drawn down |
|
11,999 |
-- |
-- |
Proceeds from Borrowings |
14 |
7,492 |
7,184 |
-- |
Payments of finance lease |
16 |
(143) |
(156) |
(168) |
Net cash generated from financing
activities |
|
33,642 |
69,457 |
137,562 |
Net increase in cash, cash
equivalents and bank overdrafts |
|
23,535 |
23,581 |
146,216 |
Currency effect cash and cash
equivalents |
|
12 |
5,828 |
4,097 |
Cash, cash equivalents and bank overdrafts at
beginning of the period |
|
263 |
23,810 |
53,219 |
Cash, cash equivalents and bank
overdrafts at end of the period |
|
23,810 |
53,219 |
203,532 |
CONTACT: uniQure:
Eva Mulder
Direct: +31 20 240 6103
Main: +31 20 240 6000
e.mulder@uniQure.com
Media inquiries:
Gretchen Schweitzer
MacDougall Biomedical Communications
Direct: +49 172 861 8540
Main: +49 89 2424 3494 or +1 781 235 3060
gschweitzer@macbiocom.com
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