~ On
Track to Begin Pivotal Study of AMT-061 in Hemophilia B in Q3
2018
~ Submitted IND Amendment for
AMT-061 Dose Confirmation Study, with Topline Data Expected by End
of 2018
~ Expects IND Submission for
AMT-130 in Huntington's Disease in the Second Half of 2018
~ $159 million of cash and cash
equivalents as of December 31, 2017
LEXINGTON, Mass. and AMSTERDAM,
the Netherlands, March 14, 2018 (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 2017 and
highlighted recent progress across its business.
"We ended 2017 with significant
momentum across all of our gene therapy programs, and a strong cash
position to fund our business into 2020," stated Matthew Kapusta,
chief executive officer of uniQure. "We remain highly focused
on initiating our pivotal study for AMT-061 in hemophilia B and
advancing AMT-130 into a Phase I/II study in Huntington's
disease. We look forward to providing updates on our progress
throughout the year, including the presentation of top-line
clinical data on AMT-061 by year-end."
Recent
Company Progress:
- Advancing
AMT-061 for the treatment of hemophilia B into a pivotal
trial
- In the first quarter of 2018, the Company
submitted an Investigational New Drug (IND) amendment to the U.S.
Food and Drug Administration (FDA) supporting its planned AMT-061
dose-confirmation study. Extensive data including
comparability, manufacturing capability and non-clinical safety and
bioequivalence were included in the submission. The Company
anticipates initiating the treatment of patients early in the third
quarter of 2018 at a single dose of 2 x 1013 gc/kg.
Top line data from these patients are expected to be presented by
the end of the year.
- The process of site selection for the pivotal
phase III study has been initiated and reviews by institutional
review boards (IRB) are underway. The Company expects to begin
patient enrollment in the lead-in phase of the pivotal study in the
third quarter of 2018.
- In the first quarter of 2018, the Company
completed the full comparability analysis for AMT-061, which the
Company believes continues to support the comparability between
AMT-060 and AMT-061.
- The Company has completed the manufacturing and
full quality release of product for use in the dose confirmation
study. The product was produced in the Company's state-of-the-art
manufacturing facility in Lexington, Massachusetts.
- The European Medicines Agency (EMA) issued a
positive opinion on Orphan Drug Designation for AMT-061 in
hemophilia B.
- Advancing
AMT-130 for the treatment of Huntington's disease into a Phase I/II
study
- Preclinical data presented at the 13th Annual
Huntington's Disease Therapeutics Conference in Palm Springs, CA
demonstrated that knocking down human mutant huntingtin (mHTT) by
AMT-130 was safe and well-tolerated in both the deep structure of
the striatum and the cortex when evaluated in a humanized mouse
model of Huntington's disease. A single administration of AMT-130
resulted in sustained dose-dependent distribution of AAV5,
consistent with previous experience, strongly corresponding with
human mHTT lowering in this model. Expression of total mHTT
was significantly reduced by up to 87% in the striatum and up to
62% in the cortex at seven months post treatment, resulting in
improvements of select behavioral and neuropathological
Huntington's disease phenotypes. Treatment was well tolerated with
no adverse events, including no psychiatric or cognitive
defects.
- A GLP-safety and toxicology study in non-human
primates is ongoing and expected to conclude mid-year. Data
from this study will be submitted in support of the IND,
which is expected to be submitted to the FDA in the second half of
2018.
- In January, the Company announced that it had
received Orphan Medicinal Product Designation (OMDP) for AMT-130 in
Huntingtin's disease by the EMA. OMPD offers product market
exclusivity for ten years in the European Union following
regulatory approval, along with tax and financial incentives for
companies developing medicines for such orphan
indications.
- Continuing research
collaboration with Bristol Myers-Squibb, including AMT-126 for the
treatment of congestive heart failure
- Under the collaboration with Bristol-Myers
Squibb, a preclinical study of AMT-126 to assess heart function in
a diseased pig model was initiated in January 2018. Pending data
from this study, which is expected later this year, the Company and
Bristol-Myers Squibb plan to initiate a safety and toxicology study
to enable an IND submission.
- Adding talent to leadership
team
- On March 12, the Company appointed David Cerveny,
J.D., as Chief Legal Officer. Prior to joining uniQure, Mr. Cerveny
was Chief Legal Officer, General Counsel and Secretary at
ConforMIS, Inc., a publicly traded medical technology company.
Prior to this, he was Chief Intellectual Property Counsel at
Palomar Medical Technologies, Inc., which was subsequently acquired
by Cynosure. Earlier in his career, Mr. Cerveny was a partner at
Hale & Dorr LLP (now WilmerHale). Mr. Cerveny earned a
Bachelor of Science degree in biomedical engineering from Marquette
University and a Juris Doctor degree from Boston College Law
School.
Upcoming
Anticipated Milestones
- Initiate dosing of patients in the dose
confirmation study of AMT-061, data from which will be available by
the end of 2018
- Initiate patient enrollment in the lead-in phase
of the pivotal study of AMT-061 in the third quarter of 2018
- Completion of the GLP-safety and toxicology study
of AMT-130 and submission of the IND in Huntington's disease in the
second half of 2018
- Completion of a preclinical therapeutic heart
function study of AMT-126 in congestive heart failure in the second
half of 2018
- Expansion of the Company's early-stage research
pipeline
Financial
Highlights
Cash
Position: As of December 31, 2017, the Company held
cash and cash equivalents of $159.4 million, compared to $132.5
million as of December 31, 2016. In October 2017, the Company
raised net proceeds of $85.3 million in an underwritten public
offering of ordinary shares. The Company currently expects cash and
cash equivalents will be sufficient to fund operations into early
2020.
Revenues: Revenue for the year ended December
31, 2017 was $13.1 million, compared to $25.1 million for the year
ended December 31, 2016. Collaboration revenue for the year ended
December 31, 2017 was $9.0 million, compared to $20.2 million for
the year ended December 31, 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 were
$72.2 million for the year ended December 31, 2017, compared to
$72.5 million for the year ended December 31, 2016. During the year
ended December 31, 2017, we recorded expenses related to an
increase in the fair market value of the contingent consideration
owed to the sellers of the InoCard business, which was offset by
cost savings associated with the consolidation of manufacturing
activities into our Lexington, Massachusetts facility.
SG&A
Expenses: Selling, general and administrative
expenses were $24.6 million for the year ended December 31, 2017,
compared to $26.0 million for the year ended December 31, 2016. The
decrease in SG&A expenses was primarily due to certain one-time
costs incurred in 2016 related to the settlement of our arbitration
proceedings with Extera and the conversion of our financial
reporting to U.S. GAAP. This reduction was offset, in part,
by an increase in share-based compensation costs during the year
ended December 31, 2017 compared to the prior year.
Other
income: Other income was $15.4 million for the year
ended December 31, 2017, compared to $1.5 million for the year
ended December 31, 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 was $79.3 million, or $2.94 per
share, for the year ended December 31, compared to $73.4 million,
or $2.93 per share, for the year ended December 31, 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,our upcoming anticipated milestones, 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. 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
Annual Report on Form 10-K filed on March 14, 2018. 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:
FOR MEDIA:
Maria E.
Cantor
Eva
M.
Mulder
Tom Malone
Direct:
339-970-7536 Direct:
+31 20 240 6103
Direct: 339-970-7558
Mobile:
617-680-9452 Mobile:
+31 6 52 33 15 79
Mobile: 339-223-8541
m.cantor@uniQure.com e.mulder@uniQure.com
t.malone@uniQure.com
uniQure
N.V.
UNAUDITED
CONSOLIDATED BALANCE SHEETS
|
|
|
|
|
December
31, |
|
December
31, |
|
2017 |
|
2016 |
|
in
thousands, except share and per share amounts |
Current assets |
|
|
|
Cash
and cash equivalents |
$ |
159,371 |
|
$ |
132,496 |
Accounts receivables and accrued income |
1,586 |
|
9,180 |
Prepaid assets and other current assets |
1,826 |
|
2,270 |
Total current assets |
162,783 |
|
143,946 |
Non-current assets |
|
|
|
Property, plant and equipment, net |
34,281 |
|
35,702 |
Intangible assets and goodwill |
10,100 |
|
8,789 |
Other
non-current assets |
2,480 |
|
1,828 |
Total non-current assets |
46,861 |
|
46,319 |
Total assets |
$ |
209,644 |
|
$ |
190,265 |
Current liabilities |
|
|
|
Accounts payable |
$ |
2,908 |
|
$ |
5,524 |
Accrued expenses and other current liabilities |
8,838 |
|
9,766 |
Current portion of long-term debt |
1,050 |
|
605 |
Current portion of deferred rent |
737 |
|
684 |
Current portion of deferred revenue |
4,613 |
|
6,142 |
Current portion of contingent consideration |
1,084 |
|
- |
Total current liabilities |
19,230 |
|
22,721 |
Non-current liabilities |
|
|
|
Long-term debt, net of current portion |
19,741 |
|
19,631 |
Deferred rent, net of current portion |
9,114 |
|
6,781 |
Deferred revenue, net of current portion |
67,408 |
|
75,612 |
Contingent consideration, net of current portion |
2,880 |
|
1,838 |
Derivative financial instruments related party |
1,298 |
|
51 |
Other
non-current liabilities |
614 |
|
- |
Total non-current liabilities |
101,055 |
|
103,913 |
Total liabilities |
120,285 |
|
126,634 |
Total shareholders' equity |
89,359 |
|
63,631 |
Total liabilities and shareholders'
equity |
$ |
209,644 |
|
$ |
190,265 |
uniQure
N.V.
UNAUDITED
CONSOLIDATED STATEMENTS OF OPERATIONS
|
|
|
Years ended
December 31,
|
|
2017 |
|
|
2016 |
|
|
2015 |
|
|
in
thousands, except share and per share amounts |
|
|
|
Total revenues |
$ |
13,107 |
|
|
$ |
25,098 |
|
|
$ |
10,578 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
Research and development expenses |
(72,172 |
) |
|
(72,510 |
) |
|
(59,125 |
) |
Selling, general and administrative expenses |
(24,635 |
) |
|
(25,999 |
) |
|
(23,383 |
) |
Total operating expenses |
(96,807 |
) |
|
(98,509 |
) |
|
(82,508 |
) |
Other
income |
15,430 |
|
|
1,465 |
|
|
779 |
|
Other
expense |
(3,073 |
) |
|
- |
|
|
- |
|
Loss from operations |
(71,343 |
) |
|
(71,946 |
) |
|
(71,151 |
) |
Non
operating items, net |
(8,116 |
) |
|
(283 |
) |
|
(12,111 |
) |
Loss before income tax expense |
(79,459 |
) |
|
(72,229 |
) |
|
(83,262 |
) |
Income
tax benefit / (expense) |
199 |
|
|
(1,145 |
) |
|
1,179 |
|
Net loss |
$ |
(79,260 |
) |
|
$ |
(73,374 |
) |
|
$ |
(82,083 |
) |
|
|
|
|
|
|
|
|
|
Basic
and diluted net loss per common share |
$ |
(2.94 |
) |
|
$ |
(2.93 |
) |
|
$ |
(3.72 |
) |
Weighted average shares used in computing basic and diluted net
loss per common share |
26,984,183 |
|
|
25,036,465 |
|
|
22,082,345 |
|
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
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