uniQure Announces Third Quarter 2021 Financial Results and
Highlights Recent Company Progress
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 2021 and highlighted recent progress across its business.
“We continue to make strong progress across our
clinical trials and research programs, and CSL Behring is making
significant strides towards a BLA submission for etranacogene
dezaparvovec in hemophilia B, which is expected in the first half
of 2022,” stated Matt Kapusta, chief executive officer at uniQure.
“Before the end of this year, we look forward to announcing
clinical data from our Huntington’s disease gene therapy program,
including preliminary data on the first four patients in our
ongoing U.S. Phase I/II study of AMT-130. Additionally, top-line
78-week follow-up data on all HOPE-B pivotal study patients is
expected to be announced by CSL Behring in late 4Q2021.”
- Advancing late-stage development of
etranacogene dezaparvovec (AMT-061) for the treatment of hemophilia
B – licensed to CSL Behring
- In September 2021, all patients in
the HOPE-B pivotal trial of etranacogene dezaparvovec in severe to
moderately severe hemophilia B completed their 78-week follow-up
visits. Along with our global partner, CSL Behring, the
Company expects to announce top-line data from the HOPE-B trial in
the late fourth quarter of 2021.The Company announced initial
52-week follow-up data from the HOPE-B pivotal trial in June 2021.
Data showed that after a single dose of etranacogene dezaparvovec,
patients continued to demonstrate durable, sustained increases in
FIX activity with a mean FIX activity of 41.5% of normal at 52
weeks of follow-up, compared to a mean of 39.0% of normal at
26-weeks of follow-up. During the 52-week period, patients
reported an 80% reduction in total bleeding events requiring
treatment, an 85% reduction in spontaneous bleeding events
requiring treatment, and a 96% reduction in annualized usage of FIX
replacement therapy.
- The Company and CSL Behring
continue to make significant progress on the preparation of a
biologics license application (BLA) for etranacogene dezaparvovec,
which is expected to be submitted by CSL Behring to the U.S. Food
and Drug Administration (FDA) in the first half of 2022. As part of
these efforts, the Company anticipates completing the validation of
its commercial manufacturing process before year-end 2021.
- Advancing the clinical development
of AMT-130 for the treatment of Huntington’s disease
- In August 2021, two additional
patient procedures were completed in the second dose cohort of the
ongoing Phase I/II clinical trial of AMT-130. These procedures
followed a positive recommendation from the study’s Data Safety
Monitoring Board (DSMB) after reviewing available follow-up data
from all participants in the trial. The DSMB is expected to meet
again in the fourth quarter of 2021, and assuming a positive
recommendation, the Company expects to enroll additional patients
before the end of 2021. Full study enrollment of all 26 patients is
expected to be completed by mid-2022.
- A total of 14 patients have been
enrolled in the Phase I/II study of AMT-130, including all 10
patients in the first dose cohort and four patients in the second,
higher-dose cohort. Of the patients enrolled to date, eight
patients have been treated with AMT-130 and six patients have
received imitation surgery.Initial data from the first four
enrolled patients in this first-in-human AAV-gene therapy trial are
anticipated by year-end 2021. These initial data will focus
primarily on safety up to 12 months following administration of
AMT-130, as well as early imaging and biomarker data at one year.
Additional clinical safety and efficacy data on longer patient
follow-up in this clinical trial are expected throughout 2022.
- The Company also remains on track
to initiate a second Phase I/II clinical study of AMT-130 in
Europe, with enrollment beginning before year-end 2021. The
open-label study will enroll 15 patients with early manifest
Huntington’s disease across two dose cohorts. Together with the
U.S. study, the European study is intended to establish safety,
proof of concept, and the optimal dose of AMT-130 to take forward
into Phase III development or into a confirmatory study should an
accelerated registration pathway be feasible.
- Expanding the pipeline and
strengthening the platform
- In July 2021, the Company closed
the acquisition of Corlieve Therapeutics, a pre-clinical gene
therapy company based in France. Corlieve’s lead program, now
designated as AMT-260, employs miRNA silencing technology to
suppress aberrantly expressed kainite receptors in the hippocampus
of patients with temporal lobe epilepsy (TLE). TLE represents a
large unmet clinical need affecting approximately 1.3 million
people in the U.S. and Europe alone, of which approximately 800,000
patients are unable to adequately control acute seizures with
currently approved anti-epileptic therapies. The acquisition was
announced in June 2021 during the Company’s virtual Research &
Development Day.
- In October 2021, the Company had a
strong presence at the European Society of Gene and Cell Therapy
(ESGCT) 28th Annual Meeting with four data presentations, each
described below.
- Preclinical data of
AMT-191, a gene-therapy candidate for the treatment of Fabry
disease, confirming high efficiency and cross correction in a Fabry
mouse model, with increased GLA-activity in the liver, kidney,
heart, and brain and normalized (lyso-)Gb3 levels in main target
organs.
- Preclinical data
from AMT-230, an AAV gene therapy candidate for Parkinson’s
Disease, reduces alpha synuclein in dopaminergic neurons and
rescues motor phenotypes in a genetic model of Parkinson’s
Disease.
- Preclinical data
from AMT-161, an AAV gene-therapy for the treatment of ALS, reduces
the expression of c9ORF72 in human iPSC derived neurons, reduces
C9ORF72-containing RNA foci in a model of the disease and is
distributed to relevant regions of the spinal cord and brain when
expressed in non-human primates.
- Preclinical data
from AMT-260, a gene therapy candidate for temporal lobe epilepsy,
efficiently reduces the expression of GluK2 in cortical neurons,
reduces epileptiform activity and hyperlocomotion in a preclinical
model of epilepsy and blocks epileptiform discharges in organotypic
slices from patients with TLE.
- Appointment of experienced
biopharma executive to the Board of Directors
- Rachelle Jacques was appointed to
the Company’s Board of Directors in October 2021 and will serve on
the Company’s Audit Committee. Ms. Jacques has more than 25 years
of industry experience. She has led successful launches of several
rare disease therapies including the recent approval of a
Regenerative Medicine Advanced Therapy (RMAT)-designated product,
one of only three since the program inception in 2016. Since
February 2019, Ms. Jacques has served as the Chief Executive
Officer of Enzyvant Therapeutics Inc, focusing on the development
of transformative regenerative therapies for rare diseases.
- Strong cash position to advance the
Company’s programs
- As of September 30, 2021, the
Company had cash and cash equivalents of $578.5 million. The
Company expects that its cash and cash equivalents will be
sufficient to fund operations into the first half of 2024. In
addition, under the CSL Behring Agreement, the Company is eligible
to receive more than $300 million in regulatory, development, and
first commercial sale milestones, $1.3 billion in additional
commercial milestones, and tiered double-digit royalties of up to a
low-twenties percentage of net product sales arising from the
collaboration. The receipt of approximately $300 million in
regulatory, development, and first commercial sale milestones would
be expected to extend the cash runway by approximately 12-18
months.
Upcoming Investor Events (each to be
conducted virtually)
- Jefferies Gene Therapy/Editing
Summit, October 27-28, 2021
- Stifel 2021 Virtual Healthcare
Conference, November 15 -17, 2021
Financial Highlights
Cash Position: As of September
30, 2021, the Company held cash and cash equivalents of $578.5
million, compared to $244.9 million as of December 31, 2020. Upon
the CSL Behring Agreement becoming fully effective on May 6, 2021,
the Company received $462.4 million of payments. In January 2021,
the Company and Hercules entered into an amended debt facility
agreement, under which the Company drew down an additional $35.0
million for a total of $70.0 million outstanding under the facility
as of September 30, 2021. In March and April 2021, the Company sold
921,730 ordinary shares for gross proceeds of approximately $29.6
million under an Open Market Sale Agreement with SVB Leerink LLC.
In July 2021, the Company paid a net EUR 42.1 million ($49.6
million) related to its acquisition of Corlieve.
Revenues: Revenue for the three
months ended September 30, 2021 was $2.0 million, compared to $1.8
million during the same period in 2020.
R&D Expenses: Research and
development expenses were $36.4 million for the three months ended
September 30, 2021, compared to $36.3 million during the same
period in 2020. The change was primarily related to recruitment of
personnel to support the development of product candidates,
advancing the clinical development of the Company’s Huntington’s
disease gene therapy program, and increased activities associated
with preclinical product candidates.
SG&A Expenses: Selling,
general and administrative expenses were $12.0 million for the
three months ended September 30, 2021, compared to $10.8 million
during the same period in 2020. The change was primarily related to
recruitment of personnel and increased share-based compensation,
offset partially by lower professional fees.
Other Income, net: Other
income, net was $1.5 million for the three months ended September
30, 2021, compared to other income, net of $0.8 million during the
same period in 2020. The increase in other income, net was
primarily related to the receipt of employee retention credit under
the U.S. CARES Act.
Other Non-operating Items, net:
Other non-operating income, net was income of $8.6 million for the
three months ended September 30, 2021, compared to other
non-operating loss of $9.3 million for the same period in 2020. The
increase in other non-operating income was primarily related to
foreign currency gains in the current period compared to a net
foreign currency loss in the same period in 2020.
Net Loss: The net loss for the
three months ended September 30, 2021, was $36.5 million, or $0.79
basic and diluted net loss per share, compared to a loss of $53.8
million, or $1.21 basic and diluted loss per share during the same
period in 2020.
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, 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, the Company’s
expectations to announce clinical data from its gene therapy
programs in hemophilia B and Huntington’s disease , patient
enrollment in the second cohort of AMT-130, the initiation of an
open-label European study of AMT-130, the announcement of data on
the first four patients in the Phase I/II study of AMT-130, the
submission of the regulatory filing for marketing approval of
etranacogene dezaparvovec, the Company’s cash runway, or other
updates on the Company’s research pipeline, and its plans to
advance or expand its pipeline, accelerate research, identify
business development opportunities, invest in technology, or scale
its manufacturing capabilities. The Company’s actual results could
differ materially from those anticipated in these forward-looking
statements for many reasons, including, without limitation, risks
associated with the impact of the ongoing COVID-19 pandemic on our
Company and the wider economy and health care system, our
Commercialization and License Agreement with CSL Behring, 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 the
Company’s periodic securities filings, including its Annual Report
on Form 10-K filed March 1, 2021. Given these risks, uncertainties
and other factors, you should not place undue reliance on these
forward-looking statements, and the Company assumes 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 |
Chiara Russo |
Tom Malone |
Direct: 339-970-7536 |
Direct: 617-306-9137 |
Direct: 339-970-7558 |
Mobile: 617-680-9452 |
Mobile: 617-306-9137 |
Mobile: 339-223-8541 |
m.cantor@uniQure.com |
c.russo@uniQure.com |
t.malone@uniQure.com |
uniQure N.V.
UNAUDITED CONSOLIDATED BALANCE
SHEETS
|
|
|
|
|
September
30, |
|
December
31, |
2021 |
|
2020 |
|
(in
thousands, except share and per share amounts) |
Current assets |
|
|
|
Cash and cash equivalents |
$ |
578,464 |
|
$ |
244,932 |
Accounts
receivable |
|
3,771 |
|
|
6,618 |
Prepaid
expenses |
|
10,345 |
|
|
4,337 |
Other
current assets and receivables |
|
1,184 |
|
|
3,024 |
Total current assets |
|
593,764 |
|
|
258,911 |
Non-current assets |
|
|
|
Property,
plant and equipment, net |
|
41,215 |
|
|
32,328 |
Operating
lease right-of-use assets |
|
26,194 |
|
|
26,086 |
Intangible
assets, net |
|
64,005 |
|
|
3,361 |
Goodwill |
|
28,060 |
|
|
542 |
Deferred tax
assets |
|
15,719 |
|
|
16,419 |
Other
non-current assets |
|
5,668 |
|
|
2,748 |
Total non-current assets |
|
180,861 |
|
|
81,484 |
Total assets |
$ |
774,625 |
|
$ |
340,395 |
Current liabilities |
|
|
|
Accounts
payable |
$ |
5,373 |
|
$ |
3,772 |
Accrued
expenses and other current liabilities |
|
30,746 |
|
|
18,038 |
Current
portion of operating lease liabilities |
|
5,798 |
|
|
5,524 |
Total current liabilities |
|
41,917 |
|
|
27,334 |
Non-current liabilities |
|
|
|
Long-term
debt |
|
71,490 |
|
|
35,617 |
Operating
lease liabilities, net of current portion |
|
29,733 |
|
|
30,403 |
Contingent
consideration |
|
23,781 |
|
|
- |
Deferred tax
liabilities, net |
|
13,141 |
|
|
- |
Other
non-current liabilities |
|
3,808 |
|
|
3,136 |
Total non-current liabilities |
|
141,953 |
|
|
69,156 |
Total liabilities |
$ |
183,870 |
|
$ |
96,490 |
Shareholders' equity |
|
|
|
Total shareholders' equity |
|
590,755 |
|
|
243,905 |
Total liabilities and shareholders' equity |
$ |
774,625 |
|
$ |
340,395 |
uniQure N.V.
UNAUDITED CONSOLIDATED STATEMENTS OF
OPERATIONS
|
|
|
|
|
Three months ended September 30, |
|
2021 |
|
2020 |
|
(in
thousands, except share and per share amounts) |
Total revenues |
$ |
1,989 |
|
|
$ |
1,789 |
|
Operating expenses: |
|
|
|
Research and
development expenses |
|
(36,432 |
) |
|
|
(36,302 |
) |
Selling,
general and administrative expenses |
|
(12,023 |
) |
|
|
(10,789 |
) |
Total operating expenses |
|
(48,455 |
) |
|
|
(47,091 |
) |
Other
income |
|
1,680 |
|
|
|
1,032 |
|
Other
expense |
|
(214 |
) |
|
|
(220 |
) |
Loss
from operations |
|
(45,000 |
) |
|
|
(44,490 |
) |
Non-operating items, net |
|
8,558 |
|
|
|
(9,285 |
) |
Loss
before income tax expense |
|
(36,442 |
) |
|
|
(53,775 |
) |
Income tax
expense |
|
(89 |
) |
|
|
- |
|
Net
loss |
$ |
(36,531 |
) |
|
$ |
(53,775 |
) |
Earnings per ordinary share - basic |
|
|
|
Basic and diluted net loss per ordinary share |
$ |
(0.79 |
) |
|
$ |
(1.21 |
) |
Weighted
average shares - basic and diluted |
|
46,152,404 |
|
|
|
44,471,844 |
|
Uniqure NV (TG:UQ1)
Historical Stock Chart
From Jun 2024 to Jul 2024
Uniqure NV (TG:UQ1)
Historical Stock Chart
From Jul 2023 to Jul 2024