uniQure Announces Third Quarter 2017 Financial Results and Recent Company Progress
(Thomson Reuters ONE) -
LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Nov. 01, 2017 (GLOBE NEWSWIRE)
-- 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) 25(th)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 59(th) 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 59(th) 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 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, 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. Cantor
Direct: 339-970-7536
Mobile: 617-680-9452
m.cantor(at)uniQure.com
Eva M. Mulder
Direct: +31 20 240 6103
Mobile: +31 6 52 33 15 79
e.mulder(at)uniQure.com
FOR MEDIA:
Tom Malone
Direct: 339-970-7558
Mobile: 339-223-8541
t.malone(at)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
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: 01.11.2017 - 12:00 Uhr
Sprache: Deutsch
News-ID 566200
Anzahl Zeichen: 19242
contact information:
Town:
Amsterdam
Kategorie:
Business News
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