uniQure Announces Third Quarter 2019 Results and Highlights Recent Company Progress
October 28 2019 - 07:00AM
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 2019 and highlighted recent progress across its business.
“We achieved a major company milestone for 2019
with the earlier than expected completion of full patient
enrollment in the HOPE-B pivotal study of etranacogene dezaparvovec
in hemophilia B, as well as the initiation of clinical sites and
patient screening for the Phase I/II clinical study of AMT-130 in
Huntington’s disease,” stated Matt Kapusta, chief executive officer
of uniQure. “We also strengthened our balance sheet with new
capital that we expect will fund operations into mid-2022 and
allows us to deliver on key corporate goals across the
business. We look forward to continuing our momentum through
the remainder of the year and sharing our progress during what we
believe will be a very productive 2020.”
Recent Company Progress
- Advancing late-stage development of etranacogene dezaparvovec
(AMT-061 )for the treatment of hemophilia B
- In early September 2019, the Company announced the achievement
of patient enrollment in its HOPE-B pivotal study of etranacogene
dezaparvovec for the treatment of patients with severe and
moderately severe hemophilia B. Due to the high level of interest
in the study from both patients and study investigators, the study
was over-enrolled with 6 patients, providing a cumulative
enrollment of 62 patients across 39 sites in the United States and
Europe.
- With the achieved enrollment of the HOPE-B pivotal study, the
Company expects to provide top-line Factor IX (FIX) activity data
on all patients before the end of 2020, and to submit a Biologics
License Application (BLA) for marketing authorization of
etranacogene dezaparvovec in 2021.
- Advancing AMT-130 into clinical development for the treatment
of Huntington’s disease
- The Company continues to make important progress with its
dose-escalating, randomized and controlled Phase I/II clinical
study to assess the safety, tolerability and efficacy of a one-time
treatment of AMT-130 in patients with Huntington’s disease. Several
clinical sites are now open for enrollment and patient screening is
underway. The Company expects to enroll the first sentinel patient
later this year or in early 2020.
- Advancing research pipeline of gene therapy candidates towards
the clinic
- Numerous preclinical and nonclinical studies of AMT-180 for the
treatment of hemophilia A are underway to support the submission of
a clinical trial application next year. AMT-180 incorporates a
proprietary, exclusively licensed, modified Factor IX gene that has
been demonstrated in preclinical studies to convey Factor
VIII-independent activity and circumvent inhibitors to Factor
VIII.
- The Company continues to progress preclinical studies of
AMT-150 for the treatment of spinocerebellar ataxia type 3. uniQure
recently selected its lead AMT-150 candidate and expects to
initiate IND-enabling studies in the first half of 2020. In May
2019, the Company presented data from a preclinical study
demonstrating that a single administration of AMT-150 in the
cerebrospinal fluid resulted in strong AAV transduction and
significant mutant ataxin-3 lowering of up to 65%.
- The Company is currently conducting preclinical studies of
AMT-190 for the treatment of Fabry disease and expects to select a
lead candidate in 2020. AMT-190 incorporates a proprietary,
exclusively licensed, modified NAGA (ModNAGA) variant that may have
several advantages over other therapies for Fabry disease,
including higher stability in blood, circumvention of inhibitors,
better biodistribution in the target organs, secondary toxic
metabolite reduction and improved cross-correction of neighboring
cells.
- Successful follow-on offering funding operations into mid-2022
- In September 2019, the Company successfully completed the
public follow-on offering of 5.6 million ordinary shares at a
public offering price of $46.00 per ordinary share, raising
approximately $242.8 million in net proceeds.
- As of September 30, 2019, the Company held $403.2 million in
cash and cash equivalents, positioning the Company to complete the
clinical development of etranacogene dezaparvovec in hemophilia B,
begin preparations for commercial launch, to fund the Phase I/II
clinical study of AMT-130 in Huntington’s disease as well as the
further development of our other preclinical product
candidates.
Upcoming Investor and Industry
Events
- World Orphan Drug Congress, November 11 – 14, 2019, Washington,
D.C.
- Credit Suisse Healthcare Conference, November 12 – 13, 2019,
Phoenix, AZ.
- Barclays Gene Therapy Summit, November 13, 2019, New York City,
NY.
- 2019 International Ataxia Research Conference, November 13 –
16, 2019, Washington, D.C.
- Stifel Global Healthcare Conference, November 19 – 20, 2019,
New York City, NY.
- 29th Annual NECTAR Meeting, November 28 -29, 2019, Cardiff, UK.
- Piper Jaffray, 31st Annual Healthcare Conference, December 3,
2019, New York City, NY.
- Evercore ISI 2nd Annual HealthCONx Conference, December 4,
2019, Boston, MA.
Financial Highlights
Cash Position: As of September
30, 2019, the Company held cash and cash equivalents of $403.2
million, compared to $184.1 million as of June 30, 2019. The
Company currently expects cash and cash equivalents will be
sufficient to fund operations into 2022.
Revenues: Revenue for the three
months ended September 30, 2019 was $1.0 million, compared to $3.1
million during the same period 2018. The decrease reflects the
October 2018 termination of the development of S100A1 under our
collaboration with Bristol-Myers Squibb.
R&D Expenses: Research and
development expenses were $23.6 million for the three months ended
September 30, 2019, compared to $20.5 million during the same
period 2018. The change was primarily related to increased
activities associated with our ongoing clinical studies of
etranacogene dezaparvovec, the planned Phase I/II study of AMT-130,
increased share-based compensation and the hiring of additional
clinical and operations staff at our Lexington site.
SG&A Expenses: Selling,
general and administrative expenses were $8.9 million for three
months ended September 30, 2019, compared to $5.9 million during
the same period 2018. The change was primarily related to increases
in personnel and consulting expenses, professional fees and
share-based compensation expenses.
Net Loss: The net loss for the
three months ended September 30, 2019 was $23.6 million, or $0.58
per share, compared to $22.0 million, or $0.59 per share during the
same period 2018.
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 gene therapies to treat
patients with hemophilia B, hemophilia A, Huntington's disease,
Fabry disease, spinocerebellar ataxia Type 3 and other
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 ability to provide
further clinical updates and data with respect to our pivotal
Phase III study of etranacogene dezaparvovec before the end of 2020
or ever, the ability to submit a BLA for marketing authorization of
etranacogene dezaparvovec in 2021, or ever, the risk of cessation,
delay or lack of success of any of our ongoing or planned clinical
studies, our ability to enroll the first sentinel patient in our
AMT-130 Phase I/II clinical study in early 2020 or ever, and our
ability to fund our operations into mid-2020. Our actual results
could differ materially from those anticipated in these
forward-looking statements for many reasons, including, without
limitation, risks associated with our clinical development
activities, clinical results, collaboration arrangements,
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 October 28, 2019. 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 Direct:
339-970-7536 Mobile: 617-680-9452
m.canto@uniQure.com |
Eva M. Mulder Direct: +31 20 240 6103
Mobile: +31 6 52 33 15 79e.mulder@uniQure.com |
Tom MaloneDirect:
339-970-7558Mobile:339-223-8541 t.malone@uniQure.com |
uniQure N.V.
UNAUDITED CONSOLIDATED BALANCE
SHEETS
|
September 30, |
|
December 31, |
|
2019 |
|
2018 |
|
|
|
|
|
|
(in thousands, except share and per share
amounts) |
|
Current assets |
|
|
|
|
Cash and cash equivalents |
$ |
403,163 |
|
$ |
234,898 |
|
Accounts receivable and accrued income from related party |
|
101 |
|
|
233 |
|
Prepaid expenses |
|
3,820 |
|
|
1,116 |
|
Other current assets |
|
558 |
|
|
329 |
|
Total current assets |
|
407,642 |
|
|
236,576 |
|
Non-current assets |
|
|
|
|
Property, plant and equipment, net of accumulated depreciation of
$26.9 million as of September 30, 2019, and $22.9 million as of
December 31, 2018, respectively. |
|
28,278 |
|
|
29,179 |
|
Operating lease right-of-use assets |
|
26,852 |
|
|
- |
|
Intangible assets, net |
|
5,424 |
|
|
5,201 |
|
Goodwill |
|
482 |
|
|
506 |
|
Restricted cash |
|
2,914 |
|
|
2,444 |
|
Total non-current assets |
|
63,950 |
|
|
37,330 |
|
Total assets |
$ |
471,592 |
|
$ |
273,906 |
|
Current liabilities |
|
|
|
|
Accounts payable |
$ |
3,782 |
|
$ |
3,792 |
|
Accrued expenses and other current liabilities |
|
12,702 |
|
|
8,232 |
|
Current portion of operating lease liabilities |
|
4,436 |
|
|
- |
|
Current portion of deferred rent |
|
- |
|
|
311 |
|
Current portion of deferred revenue |
|
7,527 |
|
|
7,634 |
|
Total current liabilities |
|
28,447 |
|
|
19,969 |
|
Non-current liabilities |
|
|
|
|
Long-term debt |
|
35,928 |
|
|
35,471 |
|
Operating lease liabilities, net of current portion |
|
31,242 |
|
|
- |
|
Deferred rent, net of current portion |
|
- |
|
|
8,761 |
|
Deferred revenue, net of current portion |
|
23,882 |
|
|
28,861 |
|
Derivative financial instruments related party |
|
1,354 |
|
|
803 |
|
Other non-current liabilities |
|
507 |
|
|
435 |
|
Total non-current liabilities |
|
92,913 |
|
|
74,331 |
|
Total liabilities |
|
121,360 |
|
$ |
94,300 |
|
Total shareholders' equity |
|
350,232 |
|
|
179,606 |
|
Total liabilities and shareholders' equity |
$ |
471,592 |
|
$ |
273,906 |
|
|
|
uniQure N.V.
UNAUDITED CONSOLIDATED STATEMENTS OF
OPERATIONS
|
Three months ended September 30, |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
2019 |
|
|
|
2018 |
|
|
|
|
in thousands, except share and per share
amounts |
Total revenues |
$ |
1,046 |
|
|
$ |
3,148 |
|
Operating expenses: |
|
|
|
Research and development expenses |
|
(23,554 |
) |
|
|
(20,541 |
) |
Selling, general and administrative expenses |
|
(8,929 |
) |
|
|
(5,898 |
) |
Total operating expenses |
|
(32,483 |
) |
|
|
(26,439 |
) |
Other income |
|
453 |
|
|
|
557 |
|
Other expense |
|
(342 |
) |
|
|
(490 |
) |
Loss from operations |
|
(31,326 |
) |
|
|
(23,224 |
) |
Non operating items, net |
|
7,722 |
|
|
|
1,157 |
|
Loss before income tax expense |
|
(23,604 |
) |
|
|
(22,067 |
) |
Income tax expense |
|
- |
|
|
|
32 |
|
Net loss |
$ |
(23,604 |
) |
|
$ |
(22,035 |
) |
|
|
|
|
Basic and diluted net loss per ordinary share |
$ |
(0.58 |
) |
|
$ |
(0.59 |
) |
Weighted average shares used in computing basic and diluted net
loss per ordinary share |
|
40,738,938 |
|
|
|
37,247,193 |
|
|
|
|
|
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