uniQure Announces 2016 Financial Results and Provides Update on Company Progress
(Thomson Reuters ONE) -
LEXINGTON, Mass. and AMSTERDAM, the Netherlands, March 15, 2017 (GLOBE NEWSWIRE)
-- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company developing
transformative therapies for patients with severe medical needs, today announces
its financial results for the year 2016 and provides an update on company
progress.
"In 2016, we made significant progress across all of our core programs and
implemented strategic changes that have strengthened our organization and
enhanced focus on our key priorities," stated Matthew Kapusta, chief executive
officer of uniQure. "We enter 2017 with a solid financial position and strong
clinical data to advance our hemophilia B program into a Phase III study, as
well as to progress our gene therapy candidates in Huntington's disease and
congestive heart failure towards IND filings. We believe the achievement of
these objectives will deliver meaningful value to shareholders."
Recent Highlights from 2016
* Promising Updated Phase I/II Data Presented on AMT-060 at ASH
- Updated results on AMT-060, including up to 52 weeks of follow-up on the
first patient cohort receiving 5x10(12) gc/kg and up to 26 weeks of follow-
up on the second patient cohort receiving a higher dose of 2x10(13 )gc/kg,
were presented at the 58(th) American Society of Hemophilia Annual Meeting.
- Patients in the first dose cohort that discontinued prophylactic FIX
infusions experienced an 85 percent reduction in total FIX usage and a
marked reduction in frequency of spontaneous bleeding after treatment with
AMT-060, including a complete cessation of spontaneous bleeding during the
last 14 weeks of observation. The five patients in the second dose cohort
experienced a near cessation of spontaneous bleeds with only one spontaneous
bleed reported in 96 weeks of cumulative observation.
- At both doses, AMT-060 appears to be safe and well-tolerated with no loss
of FIX activity, no activation of T-cell response and no development of
inhibitors for any of the 10 patients in the study. None of the 10 patients
in the study tested positive for anti-AAV5 antibodies.
* FDA Breakthrough Therapy Designation Granted for AMT-060 in Hemophilia B
- In January 2017, the U.S. Food and Drug Administration (FDA), granted
Breakthrough Therapy Designation for AMT-060 in Hemophilia B patients. The
designation is based on results from the ongoing Phase I/II study in
patients with severe disease at up to 12 months follow-up. According to FDA
data for its fiscal year 2016, the Center for Biologics Research and Review
received a total of 23 requests for Breakthrough Therapy designation with
only four designations granted, or 17% of all requests.
* Initiated Regulatory Discussions for Phase III Program in Hemophilia B
- Regulatory discussions have now begun as the Company conducted its end-of-
Phase II meeting with the FDA earlier this quarter. The meeting was
positive, with the FDA acknowledging the clinical benefit of AMT-060 and
expressing no safety concerns based on the Phase I/II data. The Company
anticipates initiating discussions with the European Medicines Association
later this year. Plans to advance the hemophilia B program into late-stage
clinical development are ongoing, with a pivotal trial expected to begin in
2018.
* Preclinical Data Published on AMT-130 in Huntington's Disease
- uniQure is conducting ongoing preclinical studies of AMT-130, its wholly-
owned AAV5-based gene therapy product candidate for Huntington's disease.
Preclinical data published in a peer-reviewed journal showed sustained and
strong wild-type HTT protein silencing in humanized control mice, including
knock-down efficiency up to 80% using optimized miHTT scaffolds. Data from
additional preclinical studies will be presented during the year at various
academic meetings. uniQure continues to advance AMT-130 towards filing an
Investigational New Drug (IND) application to begin clinical studies in
2018.
* Research Collaboration in Congestive Heart Failure Ongoing with Multiple
Preclinical Studies
- Steady progress has been made with collaborator Bristol-Myers Squibb (BMS)
in transferring S100A to an insect-cell preparation, manufacturing material
for nonclinical and preclinical studies and conducting dose-ranging analyses
and comparability studies in healthy and diseased pigs. This ongoing work is
in support of an IND application expected to be filed by BMS in 2018.
* Completion of a Company-wide Strategic Review
- uniQure announced the completion of a company-wide strategic review
prioritizing programs in hemophilia B, Huntington's disease and
cardiovascular disease, consolidating its GMP-manufacturing operations into
its Lexington, MA location, streamlining its organization and strengthening
its financial position.
* Key Management and Board Appointments
- uniQure strengthened its senior management team with the appointment of
Paul Firuta as Chief Commercial Officer, Alex Kuta as Senior Vice President
of Regulatory Affairs, Maria Cantor as Senior Vice President, Investor
Relations & Communications, Jonathan Garen as Chief Business Officer and
Maiken Keson-Brookes as Senior Vice President and General Counsel, all of
whom are based in the U.S.
- Following its Annual General Meeting, the Company completed corporate
governance changes in transitioning from a two-tier Supervisory Board and
Management Board to a single Board of Directors structure with executive and
non-executive members. In addition, the Company appointed Jack Kaye, a
seasoned financial executive with more than 40 years of diversified
experience, to its Board of Directors.
Matthew Kapusta was appointed by the Board of Directors as Chief Executive
Officer (CEO). Mr. Kapusta had previously served as interim CEO and as Chief
Financial Officer of the Company. Mr. Kapusta will continue to serve as an
executive member of the uniQure Board of Directors.
Financial Highlights
Cash Position: As of December 31, 2016, the Company held cash and cash
equivalents of $132.5 million, compared with $221.6 million as of December
31, 2015. 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 state-of-the-art manufacturing facility in
Lexington, Massachusetts and the build-out of its new research facility in
Amsterdam, the Netherlands. The Company intends to significantly reduce capital
expenditures in 2017 and 2018 and realize operational cost savings from the
strategic restructuring initiated in November 2016. As a result of these
initiatives, the Company expects its cash on hand will be sufficient to fund
operations into 2019.
Revenues: Revenue for 2016 was $25.1 million, compared with $10.6 million in
2015 and $6.1 million in 2014. The increases are driven by research activity
associated with S100A1 for heart failure, which are fully reimbursed by BMS in
accordance with the Company's collaboration agreement.
R&D Expenses: Research and development expenses were $72.5 million in 2016,
compared with $59.1 million in 2015 and $43.8 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 intensification of the Company's activities to support the
research of S100A1, the continued progression of uniQure's preclinical candidate
for Huntington's disease, increased activity in the Company's U.S. facility and
ongoing studies associated with the Company's collaboration with 4D Molecular
Therapeutics to develop next-generation vector serotypes.
SG&A Expenses: Selling, general and administrative expenses were $26.0 million
in 2016, compared with $23.4 million in 2015 and $17.1 million in 2014.
Other income, generated from research and development subsidies, was $1.5
million in 2016 compared with $0.8 million in 2015 and $1.0 million in 2014.
Net Loss: The net loss for the fourth quarter of 2016 was $14.7 million, or
$0.58 per share, compared with $16.9 million, or $0.69 per share, for the fourth
quarter of 2015. The net loss for the full years 2016, 2015 and 2014 was $73.4
million, or $2.93 per share, $82.1 million, or $3.72 per share, and $49.8
million, or $2.91 per share, respectively.
Form S-3 registration: In March 2015, the Company filed a "shelf registration
statement" on Form F-3 with the Securities and Exchange Commission. Now that the
Company reports as a domestic issuer, the F-3 registration statement is no
longer effective, and therefore the Company today will file a new shelf
registration statement on Form S-3. This will provide the Company with
important financial flexibility for advancing its programs in the future. The
Company has no immediate plans for any financing transaction.
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 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,
statements regarding the implementation and effects of the Company's new
strategic and organizational changes, the development of our gene therapy
product candidates, 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. Our actual results could differ
materially from those anticipated in these forward-looking statements for many
reasons, including, without limitation, risks associated with corporate
reorganizations and strategic shifts, 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 2015 Annual Report on Form 20-F filed with the Securities and Exchange
Commission on April 4, 2016 and its 2016 Annual Report on Form 10-K filed 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.
UNAUDITED CONSOLIDATED BALANCE SHEETS
December 31, December 31,
2016 2015
--------------- ----------------------
in thousands, except share and per
share amounts
Current assets
Cash and cash equivalents $ 132,496 $ 221,626
Accounts receivable and accrued
income 3,680 -
Accounts receivable and accrued
income from related parties 5,500 4,129
Inventories - 474
Prepaid expenses 996 690
Other current assets 1,274 1,194
--------------- ----------------------
Total current assets 143,946 228,113
Non-current assets
Property, plant and equipment, net 35,702 26,011
Intangible assets, net 8,324 6,815
Goodwill 465 481
Other non-current assets 1,828 1,243
--------------- ----------------------
Total non-current assets 46,319 34,550
--------------- ----------------------
Total assets $ 190,265 $ 262,663
--------------- ----------------------
Current liabilities
Accounts payable $ 5,524 $ 4,059
Accrued expenses and other current
liabilities 9,766 9,863
Current portion of long-term debt 605 5,579
Current portion of deferred rent 684 630
Current portion of deferred revenue 6,142 6,778
--------------- ----------------------
Total current liabilities 22,721 26,909
Non-current liabilities
Long-term debt, net of current
portion 19,631 14,631
Deferred rent, net of current portion 6,781 6,247
Deferred revenue, net of current
portion 75,612 83,445
Contingent consideration 1,838 2,926
Other non-current liabilities 51 578
--------------- ----------------------
Total non-current liabilities 103,913 107,827
--------------- ----------------------
Total liabilities 126,634 134,736
Commitments and contingencies
Shareholders' equity
Ordinary shares, ?0.05 par value:
60,000,000 shares authorized at
December 31, 2016 and 2015 and
25,257,420 and 24,327,944 shares
issued and outstanding at December
31, 2016 and 2015, respectively. 1,593 1,542
Additional paid-in-capital 464,653 455,897
Accumulated other comprehensive loss (6,557 ) (6,828 )
Accumulated deficit (396,058 ) (322,684 )
--------------- ----------------------
Total shareholders' equity 63,631 127,927
--------------- ----------------------
Total liabilities and shareholders'
equity $ 190,265 $ 262,663
--------------- ----------------------
uniQure N.V.
UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
Years ended December 31,
--------------------------------------------------
2016 2015 2014
---------------- ---------------- ----------------
in thousands, except share and per share amounts
Total revenues $ 25,098 10,578 6,141
Operating expenses:
Research and development
expenses (72,510 ) (59,125 ) (43,772 )
Selling, general and
administrative expenses (25,999 ) (23,383 ) (17,073 )
---------------- ---------------- ----------------
Total operating expenses (98,509 ) (82,508 ) (60,845 )
Other income 1,465 779 1,022
---------------- ---------------- ----------------
Loss from operations (71,946 ) (71,151 ) (53,682 )
Non operating items, net (283 ) (12,111 ) 3,370
Loss before income tax
benefit / (expense) (72,229 ) (83,262 ) (50,312 )
Income tax benefit /
(expense) (1,145 ) 1,179 535
---------------- ---------------- ----------------
Net loss $ (73,374 ) $ (82,083 ) $ (49,777 )
---------------- ---------------- ----------------
Basic and diluted net
loss per common share $ (2.93 ) $ (3.72 ) $ (2.91 )
Weighted average shares
used in computing basic
and diluted net loss per
common share 25,036,465 22,082,345 17,121,328
uniQure Contacts:
Maria E. Cantor
Direct: 339-970-7536
Mobile: 617-680-9452
m.cantor(at)uniQure.com
Tom Malone
Direct: 339-970-7558
Mobile: 339-223-8541
t.malone(at)uniQure.com
Eva M. Mulder
Direct: +31 20 240 6103
Mobile: +31 6 52 33 15 79
e.mulder(at)uniQure.com
This announcement is distributed by Nasdaq Corporate Solutions on behalf of Nasdaq Corporate Solutions clients.
The issuer of this announcement warrants that they are solely responsible for the content, accuracy and originality of the information contained therein.
Source: uniQure N.V. via GlobeNewswire
Datum: 15.03.2017 - 12:00 Uhr
Sprache: Deutsch
News-ID 530403
Anzahl Zeichen: 20185
contact information:
Town:
Amsterdam
Kategorie:
Business News
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