uniQure N.V. (NASDAQ: QURE), a leader in human gene therapy, today
announced financial results for the third quarter and nine months
ending September 30, 2015, and provided an update on its
development programs.
"In the last quarter uniQure and our collaborators, a consortium
consisting of Institut Pasteur, INSERM, the French Muscular
Dystrophy Association and Vaincre les Maladies Lysosomales, have
clinically demonstrated proof-of-concept for safely treating a
severe lysosomal storage disease, Sanfilippo B, with a gene therapy
construct developed by uniQure. The study has shown encouraging
signals of clinical benefit. By the second week of January 2016 we
hope to announce preliminary top-line results in our Hemophilia B
trial," said Jörn Aldag, Chief Executive Officer of uniQure. "To
accelerate our pipeline development we have added two therapeutic
area leaders, Charles Richard, Senior Vice President, Neuroscience
and Deya Corzo, Senior Vice President, Liver/Metabolism, joining
Patrick Most who leads our heart failure program partnered with
BMS. Deya and Charlie will lead the preparation for two pivotal
studies in 2016."
Pipeline and Products
Hemophilia B Program Update
- The first cohort of five hemophilia B patients has been dosed
in the Phase 1/2 study of AMT-060.
- In early January 2016, the Company plans to release
preliminary, top-line data for the two patients who will have
completed at least 12 weeks of treatment follow-up. The data
readout will include safety results and FIX expression levels. No
additional data from the remaining cohort patients or other
measures of the trial will be reported at that time.
- The Company expects to report on the full results of the first
cohort at a scientific conference during 1H 2016.
Sanfilippo B Program Update
- Positive one-year follow-up results from four Sanfilippo B
patients participating in a Phase 1/2 clinical trial were presented
by Prof. Marc Tardieu representing Institut Pasteur, uniQure's
collaborator, at the ESGCT and FSGT Collaborative Congress on
September 19, 2015. The trial demonstrated durable increases in
NAGLU expression in the cerebrospinal fluid (CSF) of 14% to 17% at
12-months post treatment. It also showed that incremental cognitive
development was maintained in all four patients with no evidence of
progressive brain atrophy. The trial provided the first clinical
validation of the Company's proprietary AAV5 vector effectively
delivering a target gene into the CNS leading to durable expression
of NAGLU protein in the CNS.
- Subsequently, Prof. Tardieu presented a post-hoc biochemical
analysis of glycosaminoglycans (GAG) levels in the CSF using
samples from the same four patients. The results were presented at
the International Conference on Sanfilippo Syndrome and Related
Lysosomal Storage Diseases in Geneva on November 27, 2015. A
decrease of Heparan sulfate, a brain-predominant GAG and a
substrate for the NaGlu enzyme, could not be detected in the CSF.
This result resembles a previously published study by Ausseil and
colleagues using uniQure's AAV5 NAGLU gene therapy vector in a
canine model of Sanfilippo B, where durable NAGLU expression in the
dog brain resulted in significant reductions of brain tissue GAGs,
although CSF GAGs were unchanged (previously unpublished). Given
the encouraging positive clinical results previously reported, and
the difficulty in interpreting CSF GAGs as a biomarker predictive
of clinical benefit, uniQure is aggressively moving forward with
its plans to complete the in-license of the data from the Company's
collaborator, prepare the future pathway with regulatory
authorities and complete close follow-up of the patients through
the end of the 30-month study, expected around year-end 2016.
Glybera Program Update
- On October 28, 2015, the Charité University Clinic in Berlin,
Germany, announced the treatment of the first patient with Glybera
as a commercially-available gene therapy, enabled by uniQure's
commercialization partner in the EU, Chiesi Farmaceutici.
- During the three month period ended September 30, 2015, the
Company recorded a one-time, non-cash impairment charge of €11.6
million associated with certain intangible assets related to
Glybera. The impairment was primarily due to a reduction in the
estimated number of patients to be treated with Glybera.
- uniQure has decided not to pursue U.S. regulatory approval of
Glybera in order to maintain the Company's focus on its three core
therapeutic areas. The Company will continue to meet all of its
obligations to its partner Chiesi and all EMA obligations including
the execution of a post-approval phase IV clinical trial, and the
continuation of the patient registry (GENIALL) to assess the
long-term clinical benefit of Glybera.
Corporate Updates
Strategic Collaboration with Bristol-Myers
Squibb
- On August 10, 2015, uniQure announced the receipt of an
additional $53 million from Bristol-Myers Squibb in accordance with
the companies' collaboration agreement. Included in the total was a
$15 million target designation fee triggered by Bristol-Myers
Squibb's selection of three new collaboration targets, in addition
to S100A1 for congestive heart failure. Bristol-Myers Squibb
also acquired an additional 1.3 million ordinary shares of uniQure
priced at $29.67 per share, providing aggregate net proceeds to the
Company of approximately $38 million. The purchase price
represented an approximately 26% premium over uniQure's closing
price per ordinary share on August 7, 2015. After this second
equity closing, Bristol-Myers Squibb owns 9.9% of uniQure's
outstanding ordinary shares. To date, uniQure has received a total
of $140 million from Bristol-Myers Squibb in the context of the
agreement.
Other Corporate Developments
- Infrastructure: uniQure's 50,000 sq. ft., fully scalable gene
therapy manufacturing plant in Lexington, Massachusetts is now
operational and research batches are being produced.
- Human Resources: In July, uniQure announced the appointment of
Charles W. Richard, M.D., Ph.D., to the position of Senior Vice
President, Research and Development, Neuroscience, to lead the
Company's growing portfolio of gene therapies targeting
neurological diseases, including current clinical trials for the
treatment of Sanfilippo B syndrome and Parkinson's disease as well
as preclinical programs in Huntington's disease and other rare CNS
disorders.
- Also in July, Deya Corzo, M.D., who joined the Company in
Spring 2014 as Vice President, Medical Affairs, was promoted to
Senior Vice President, Therapeutic Area Head,
Liver/Metabolism. She leads the efforts for the hemophilia B
clinical trial program as well as research and development for
hemophilia A.
- Hans Preusting, Chief Business Officer, has decided to pursue
new entrepreneurial opportunities. He will remain available as
a consultant following his departure in January 2016.
"Hans has been an outstanding operations and business
development executive for us over the last 9 years and I have
greatly enjoyed working with him as we crafted both large scale
strategic partnerships and innovative agreements with our
scientific collaborators. All of us at uniQure wish him the
very best and thank him for all his contributions to the growth of
the company," added Mr. Aldag.
Financial Highlights
As of September 30, 2015, the Company held cash and cash
equivalents of €214.5 million, compared with €53.2 million as of
December 31, 2014. The increase was due to the consideration
received from BMS during the period, offset in part by cash used in
research, development and general corporate
activities. Revenue for the three months ended September 30,
2015 was €3.2 million, compared with €1.0 million for the
comparable period in 2014. For the nine months ended September 30,
2015, licensing and collaboration revenues were €5.9 million
compared with €3.2 million for the same period of 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.
Research and development expenses were €11.9 million for the
three months ended September 30, 2015, compared to €9.5 million for
the comparable period in 2014. For the nine months ended September
30, 2015, research and development costs were €32.7 million
compared with €23.7 million for the same period of 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 €4.8 million
for the three months ended September 30, 2015, compared with €3.2
million for the comparable period in 2014. Selling, general and
administrative costs for the nine months ended September 30, 2015
were €13.5 million, compared with €8.0 million for the comparable
period 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 €1.6 million for the three
months ended September 30, 2015, compared to a gain of €3.6 million
for the comparable period in 2014. For the nine months ended
September 30, 2015, other gains/losses were a loss of €2.5 million
compared with a gain of €3.7 million in the same period of 2014.
The loss was primarily attributable to the impact of foreign
currency exchange rates on the Company's dollar-denominated
deposits and the periodic revaluation of outstanding warrants.
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 third quarter of 2015 was €25.8 million, or
€1.08 per share, compared with €9.1 million, or €0.51 per share,
for the third quarter of 2014. The net loss for the nine
months ended September 30, 2015 and 2014 were €57.4 million, or
€2.69 per share and €25.9 million, or €1.54 per share,
respectively. Excluding the one-time impairment charge
incurred in the third quarter of 2015, the net loss for the third
quarter of 2015 was €14.2 million, or €0.67 per share, and the net
loss for the nine months ended September 30, 2015 was €45.7
million, or €2.14 per share.
For further financial information for the period ending
September 30, 2015, please refer to the financial statements
appearing at the end of this release.
Webcast
uniQure will host a webcast and conference call today at 8:30 am
ET/14:30 CET to report on current developments. To access the
conference call information and the webcast, log on to the "Events"
section on the "Media" page of uniQure's website, at
http://www.uniqure.com/news/calendar-of-events. A replay of the
webcast will be available on uniQure's website for at least 72 days
following the event.
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 Statement
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 future
development of our programs in Sanfilippo B, hemophilia B and
cardiovascular diseases, the success of our collaboration with
Bristol-Myers Squibb, 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 7, 2015. 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.
CONTACT uniQure: Aicha Diba Investor Relations Direct : +31
20 240 6110 Main: +31 20 240 6000 a.diba@uniQure.com
Media inquiries: Gretchen Schweitzer MacDougall Biomedical
Direct: +49 172 861 8540 Main: +49 89 2424 3494 or +1 781 235 3060
gschweitzer@macbiocom.com
UNIQURE N.V. |
Unaudited Condensed
Consolidated Balance Sheets |
(€ in thousands) |
|
|
|
|
|
|
DECEMBER 31, |
SEPTEMBER, 30 |
|
NOTE |
2014 |
2015 |
Assets |
|
|
|
Non-current assets |
|
|
|
Goodwill |
8,9 |
1,342 |
1,342 |
Intangible assets other than
Goodwill |
8,9 |
16,368 |
7,362 |
Property, plant and
equipment |
7 |
19,667 |
22,627 |
Other non-current assets |
10 |
1,022 |
1,108 |
Total non-current assets |
|
38,399 |
32,439 |
Current assets |
|
|
|
Receivables from related
parties |
11 |
2,426 |
5,142 |
Trade and other
receivables |
11 |
1,542 |
2,166 |
Inventories |
12 |
200 |
411 |
Cash and cash equivalents |
13 |
53,219 |
214,494 |
Total current assets |
|
57,387 |
222,213 |
Total assets |
|
95,786 |
254,652 |
Equity |
|
|
|
Share capital |
|
905 |
1,211 |
Share premium |
|
206,111 |
344,376 |
Other reserves |
|
17,149 |
23,318 |
Accumulated deficit |
|
(181,081) |
(238,450) |
Total equity |
14 |
43,084 |
130,455 |
Liabilities |
|
|
|
Non-current liabilities |
|
|
|
Borrowings |
16 |
16,418 |
13,313 |
Derivative financial
instruments - related parties |
16 |
-- |
740 |
Financial lease
liabilities |
16,26 |
134 |
-- |
Deferred rent |
26 |
5,658 |
5,711 |
Deferred revenue |
17 |
15,387 |
77,043 |
Deferred tax liabilities |
9 |
1,379 |
1,349 |
Contingent considerations |
9 |
1,454 |
2,061 |
Total non-current
liabilities |
|
40,430 |
100,217 |
Current liabilities |
|
|
|
Trade and other payables |
15 |
9,617 |
12,090 |
Derivative financial
instruments- related parties |
16 |
645 |
1,268 |
Borrowings |
16 |
-- |
4,636 |
Borrowings - derivative |
16 |
207 |
357 |
Deferred rent |
26 |
475 |
550 |
Deferred revenue |
17 |
1,328 |
5,079 |
Total current
liabilities |
|
12,272 |
23,980 |
Total liabilities |
|
52,702 |
124,197 |
Total equity and
liabilities |
|
95,786 |
254,652 |
|
UNIQURE
N.V. |
Unaudited Condensed
Consolidated Statements of Comprehensive Loss |
(€ in thousands, except share
and per share data) |
|
|
|
|
|
|
|
|
THREE MONTHS
ENDED |
NINE MONTHS
ENDED |
|
|
SEPTEMBER
30, |
SEPTEMBER
30, |
|
|
|
|
|
|
|
NOTE |
2014 |
2015 |
2014 |
2015 |
|
|
(€
in thousands) |
License revenues |
17 |
221 |
1,041 |
662 |
1,754 |
Collaboration revenues |
17 |
780 |
1,809 |
2,551 |
3,818 |
Product sales |
17 |
-- |
300 |
-- |
300 |
Total revenues |
|
1,001 |
3,150 |
3,213 |
5,872 |
Cost of goods sold |
|
-- |
(428) |
-- |
(428) |
Other income |
|
208 |
186 |
598 |
532 |
Research and development expenses |
18 |
(9,514) |
(11,933) |
(23,740) |
(32,652) |
Selling, general and administrative
expenses |
19 |
(3,218) |
(4,835) |
(8,035) |
(13,503) |
Impairment of intangible assets |
8 |
-- |
(11,640) |
-- |
(11,640) |
Other gains / losses, net |
20 |
3,630 |
(1,552) |
3,694 |
(2,548) |
Total operating costs |
|
(8,894) |
(30,202) |
(27,483) |
(60,239) |
Operating result |
|
(7,893) |
(27,052) |
(24,270) |
(54,367) |
Finance income |
|
54 |
5 |
125 |
70 |
Finance expense |
22 |
(1,220) |
1,173 |
(1,734) |
(3,102) |
Finance
income/(expense)--net |
|
(1,166) |
1,178 |
(1,609) |
(3,032) |
Result before corporate income
tax |
|
(9,059) |
(25,874) |
(25,879) |
(57,399) |
Corporate income taxes |
|
-- |
30 |
-- |
30 |
Net loss |
|
(9,059) |
(25,844) |
(25,879) |
(57,369) |
Items that may be subsequently reclassified
to profit or loss |
|
|
|
|
|
Currency translation differences on foreign
operations |
|
703 |
(159) |
693 |
927 |
Other comprehensive income/(loss) |
21 |
703 |
(159) |
693 |
927 |
Total comprehensive
loss |
|
(8,356) |
(26,003) |
(25,186) |
(56,442) |
Loss per share attributable to the equity
holders of the Company during the year: |
|
|
|
|
|
Basic and diluted loss per
share |
24 |
(0.51) |
(1.08) |
(1.54) |
(2.69) |
|
UNIQURE
N.V. |
Unaudited Condensed
Consolidated Statement of Changes in Equity/Deficit |
(€ in thousands) |
|
|
|
|
|
|
|
|
|
Total |
|
|
|
|
|
|
Share |
Share |
Other |
Accumulated |
Total |
|
Note |
Capital |
Premium |
Reserves |
Deficit |
Equity/Deficit |
Balance at January 1,
2014 |
|
610 |
142,459 |
6,536 |
(144,041) |
5,564 |
Result for the period |
|
-- |
-- |
-- |
(25,868) |
(25,868) |
Other comprehensive income/(loss) |
|
-- |
-- |
693 |
(10) |
683 |
Total comprehensive
loss |
|
-- |
-- |
693 |
(25,878) |
(25,185) |
Capital contributions |
|
282 |
64,020 |
-- |
-- |
64,302 |
Share issuance costs |
|
-- |
(668) |
-- |
-- |
(668) |
Share based payment/expense |
|
-- |
-- |
7,193 |
-- |
7,193 |
Balance at September 30,
2014 |
|
892 |
205,811 |
14,422 |
(169,920) |
51,205 |
Result for the period |
|
-- |
-- |
-- |
(11,170) |
(11,170) |
Other comprehensive income |
|
-- |
-- |
456 |
9 |
465 |
Total comprehensive
loss |
|
-- |
-- |
456 |
(11,161) |
(10,705) |
Capital contributions |
|
13 |
300 |
-- |
-- |
313 |
Share based payment/expense |
|
-- |
-- |
2,271 |
-- |
2,271 |
Balance at December 31,
2014 |
14 |
905 |
206,111 |
17,149 |
(181,081) |
43,084 |
Result for the period |
|
-- |
-- |
-- |
(57,369) |
(57,369) |
Other comprehensive income |
|
-- |
-- |
927 |
-- |
927 |
Total comprehensive
loss |
|
-- |
-- |
927 |
(57,369) |
(56,442) |
Capital contributions |
|
306 |
138,877 |
-- |
-- |
139,183 |
Share issuance costs |
|
-- |
(612) |
-- |
-- |
(612) |
Share based payment/expense |
|
-- |
-- |
5,242 |
-- |
5,242 |
Balance at September 30,
2015 |
14 |
1,211 |
344,376 |
23,318 |
(238,450) |
130,455 |
|
UNIQURE
N.V. |
Unaudited Condensed
Consolidated Statement of Cash Flows |
(€ in thousands) |
|
|
|
|
|
|
NINE MONTHS
ENDED |
|
|
September
30, |
|
|
|
|
|
NOTE |
2014 |
2015 |
Cash flow from operating
activities |
|
|
|
Net loss |
|
(25,879) |
(57,369) |
Adjustments for: |
|
|
|
Depreciation |
7 |
859 |
2,872 |
Amortization on intangibles
assets |
7 |
-- |
274 |
Impairment of intangible
assets |
8 |
-- |
11,640 |
Lease incentive |
26 |
4,854 |
129 |
Loss/(gain) on derivatives |
16 |
(574) |
164 |
Loss/(gain) on foreign
exchanges |
|
(2,545) |
3,160 |
Other non-cash items |
|
(1) |
606 |
Share-based expenses |
23 |
7,193 |
5,242 |
Changes in trade and other
receivables |
|
(142) |
(3,339) |
Movement in inventories |
12 |
326 |
(210) |
Changes in trade and other
payables |
15 |
(1,812) |
(5,234) |
Changes in deferred revenue and
provisions |
|
(635) |
65,407 |
Initial recognition of
warrants |
3 |
-- |
2,622 |
Movement in other
liabilities |
|
1,209 |
2,435 |
Interest (income) /
expense |
|
1,035 |
1,504 |
Cash (used in) / generated by
operations |
|
(16,112) |
29,903 |
Interest paid |
|
(807) |
(1,404) |
Net cash (used in) / generated by
operating activities |
|
(16,919) |
28,499 |
Cash flow from investing
activities |
|
|
|
Purchases of property, plant
and equipment |
7 |
(13,365) |
(3,289) |
Purchases of intangible
assets |
8 |
(2,129) |
(2,908) |
Interest received |
|
95 |
70 |
Acquisition of businesses |
|
(1,463) |
-- |
Net cash used in investing
activities |
|
(16,862) |
(6,127) |
Cash flow from financing
activities |
|
|
|
Proceeds from shares
issued |
14 |
62,786 |
137,909 |
Share issuance cost |
14 |
(668) |
(612) |
Proceeds from Borrowings |
16 |
7,184 |
-- |
Payments of finance lease |
16 |
(116) |
(125) |
Net cash generated from financing
activities |
|
69,186 |
137,172 |
Net increase in cash, cash
equivalents and bank overdrafts |
|
35,405 |
159,544 |
Currency effect cash and cash
equivalents |
|
3,567 |
1,731 |
Cash, cash equivalents and bank overdrafts at
beginning of the period |
|
23,810 |
53,219 |
Cash, cash equivalents and bank
overdrafts at end of the period |
|
62,782 |
214,494 |
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