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 second quarter
of 2022 and highlighted recent progress across its business.
“We continued to make strong progress across all
of our programs highlighted by encouraging data from the lower-dose
cohort in our Phase I/II study of AMT-130 and the advancement of
the U.S. and European regulatory reviews for the marketing
applications for etranacogene dezaparvovec in hemophilia B,” stated
Matt Kapusta, chief executive officer at uniQure. “Our
manufacturing, CMC and Quality teams are working tirelessly to
produce commercial launch supplies and also to ensure successful
pre-approval site inspections, which are expected to be completed
in the third quarter of this year. We also are very pleased to have
recently received notification that GMP certification will be
provided to our Lexington manufacturing facility by the European
authorities.”
“In July, we reported to the health authorities
suspected unexpected severe adverse reactions in three of the 14
patients treated with the higher dose of AMT-130,” he continued.
“While these patients have fully or substantially recovered, and no
clear root cause has yet been identified, we believe it’s prudent
to temporarily delay additional higher-dose procedures until we put
additional monitoring and treatment plans in place and complete our
safety review early in the fourth quarter of 2022. The delay is not
expected to impact any future lower-dose procedures, as no serious
adverse events related to AMT-130 have been reported in any of the
additional 12 patients treated at the lower dose, and no impact is
expected on the timing of previously guided data read-outs in 2023,
including the presentation of one to two-year follow-up on both low
and high-dose cohorts in the second quarter of 2023.”
Recent Updates
- AMT-061 for the treatment of
hemophilia B
- In May 2022, the Company’s global
commercialization partner, CSL Behring, announced that the BLA for
etranacogene dezaparvovec was accepted by the Food and Drug
Administration (FDA) for priority review. This follows the MAA
submission by CSL Behring which was validated by the EMA in March
2022.
- In July 2022, CSL Behring was
notified by the Committee for Advanced Therapies (CAT) in Europe
that they will be unable to complete their review in accordance
with the accelerated assessment timetable and will now switch to a
standard review procedure. In the United States, the BLA remains
under Priority Review at this time. In accordance with the
Company’s commercialization and license agreement, CSL Behring is
solely responsible for all regulatory activities, including filings
and agency interactions associated with etranacogene
dezaparvovec.
- In July 2022, following a
comprehensive multi-day facility inspection, the European Medicines
Agency notified the Company that Good Manufacturing Practice (GMP)
certification can be issued for the Company’s Lexington
manufacturing site to produce commercial supply of etranacogene
dezaparvovec.
- AMT-130 for the treatment of
Huntington’s disease
- In June 2022,
initial 12-month observations were announced on the lower-dose
cohort of the double-blinded and randomized U.S. Phase I/II study
of AMT-130 for the treatment of early-stage Huntington’s disease.
- At 12 months of
follow-up on these patients, the lower-dose was generally
well-tolerated with no serious adverse events related to treatment.
In the four treated patients with evaluable data from this cohort,
mean levels of mutant Huntingtin protein (mHTT) in the cerebral
spinal fluid (CSF) declined at all timepoints compared to baseline
and decreased by 53.8% at 12 months of follow-up. In the three
control patients with evaluable data, mean levels of mHTT showed an
increase compared to baseline at one, three, six and nine months of
follow-up, and decreased by 16.8% at 12 months of follow-up.
- In the six
treated patients in the lower-dose cohort, measurements of
neurofilament light chain (NfL) in the CSF, a biomarker of neuronal
damage, initially increased as expected following the AMT-130
surgical procedure and declined thereafter, nearing baseline at 12
months of follow-up. At 12 months, mean NfL showed an 8% increase
compared to baseline. Two of the six treated patients were at or
below baseline at 12 months of follow-up, with an additional
patient below baseline at 15 and 18 months of follow-up.
- In mid-July,
the Company reported to the appropriate regulatory agencies
suspected unexpected severe adverse reactions (SUSARs) in two
patients that were treated with the higher-dose of AMT-130 at a
single clinical site in the European Phase Ib/II study. Both
patients presented with localized inflammatory responses and other
related symptoms approximately one to two weeks after their
procedures. A third patient, who had previously been treated with
the higher-dose of AMT-130 in the U.S. during March 2022,
experienced severe headache and other related symptoms soon after
AMT-130 administration that was initially deemed by the
investigator as not related to AMT-130 but related to the
procedure. Upon further review and discussion with the clinical
trial’s independent Data Safety Monitoring Board (DSMB) following
the events observed in the European study, the Company has
reclassified and reported the patient reaction in the U.S. trial as
a SUSAR. All three patients have fully or substantially recovered
after treatment and have been released from the hospital.
- At this time,
the DSMB does not view these findings as a dose-limiting toxicity.
The DSMB has recommended temporarily delaying higher-dose
enrollment pending a safety review, which is expected to take place
early in the fourth quarter of 2022. The DSMB is permitting
continued enrollment at the lower-dose of AMT-130, and the Company
expects to begin crossing over control patients from the U.S. study
at this dose in the third quarter of 2022.
- No impact is
expected on the timing of previously guided data read-outs in 2023.
In the U.S. Phase I/II study, all 26 patients in the first two dose
cohorts have been enrolled, and the Company continues to expect to
present one to two-year follow up data in the second quarter of
2023. In the European Phase Ib/II study, the six-patient lower-dose
cohort is fully enrolled and the Company continues to expect to
present one-year follow-up data in 2023. Four of nine patients in
the European study have been enrolled in the higher-dose cohort. To
date, a total of 36 patients have been enrolled across the two
clinical trials including 10 control patients and 26 patients
treated with AMT-130, of which 14 patients received the higher-dose
and 12 patients received the lower-dose.
- Progress towards 2023 INDs
- AMT-260 for the treatment of
refractory temporal lobe epilepsy (rTLE) – In July 2022, the
Company initiated an IND-enabling GLP toxicology study in non-human
primates to support an IND submission expected in 2023.
- AMT-191 for the treatment of Fabry
disease – The Company expects to initiate a GLP toxicology study of
AMT-191 in non-human primates in the second half of 2022, which is
expected to support an IND submission in 2023.
- Strong cash position to advance the
Company’s programs
- As of June 30, 2022, the Company
had cash and cash equivalents of $500.5 million. The Company
expects that its cash and cash equivalents will fund operations
into the first half of 2025 assuming the achievement of the first
commercial sales milestones under the CSL Behring Agreement.
Upcoming Investor Events
- Citi’s 17th Annual BioPharma
Conference, September 7 – 8, 2022
- Wells Fargo 2022 Healthcare
Conference, September 7 – 9, 2022
- Wainwright 24th Global Investment
Conference, September 12 – 14th, 2022 (virtual attendance)
- Cantor Fitzgerald Cell & Gene
Therapy Conference, September 15, 2022
- Jefferies Cell and Genetic
Medicines Summit, September 29 - 30, 2022
- Chardan 6th Annual Genetic
Medicines Conference, October 3 - 4, 2022
Financial Highlights
Cash position: As of June 30,
2022, the Company held cash and cash equivalents of $500.5 million,
compared to $556.3 million as of December 31, 2021.
Revenues: Collaboration revenue
for the three months ended June 30, 2022, was $0.5 million,
compared to collaboration revenue of $1.5 million for the same
period in 2021. Revenue for 2021 during this period also included
license revenue of $462.4 million from selling the exclusive global
rights to etranacogene dezaparvovec to CSL Behring and nil in the
same period 2022.
Cost of contract
revenues: Cost of contract revenues for the three
months ended June 30, 2022 was nil compared to $23.2 million for
the same period in 2021. Costs incurred in 2021 are associated with
recognized license revenue.
Cost of contract manufacturing:
Cost of contract manufacturing for the three months ended June 30,
2022, was $0.8 million compared to nil for the same period in 2021.
Costs incurred in 2022 related to the manufacture of etranacogene
dezaparvovec for CSL Behring.
R&D expenses: Research and
development expenses were $46.2 million for the three months ended
June 30, 2022, compared to $32.8 million during the same period in
2021. The increase was related to the preclinical development of
temporal lobe epilepsy (AMT-260) advancing the clinical development
of the Company’s Huntington’s disease gene therapy program, and
recruitment of personnel to support the development of product
candidates.
SG&A expenses: Selling,
general and administrative expenses were $12.5 million for the
three months ended June 30, 2022, compared to $17.3 million during
the same period in 2021. The reduction was primarily related to
incurring financial advisory fees to close the CSL Behring
transaction in 2021 with no such fees in 2022.
Other non-operating Items, net:
Other non-operating income, net was income of $16.7 million for the
three months ended June 30, 2022, compared to other non-operating
income, net of $4.7 million for the same period in 2021. The
increase in other non-operating income, net was primarily related
to an increase in net foreign currency gains of $12.8 million
($19.4 million recorded in the current period compared to $6.6
million for the same period in 2021).
Net (loss) / income: The net
loss for the three months ended June 30, 2022, was $39.1 million,
or $0.84 basic and diluted loss per ordinary share, compared to
$399.5 million net income for the same period in 2021, or $8.68
basic net income per ordinary share and $8.51 diluted net income
per ordinary share.
Investor Conference Call and Webcast
Information
uniQure management will host an investor
conference call and webcast today, Monday, August 8, 2022, at 8:30
a.m. ET. The conference call may be accessed via pre-registering
here. Participants will then receive a dial-in number and personal
PIN. Research analysts who wish to ask a question should do so
through the pre-registered conference call number. If you are
joining the conference call, please dial-in 15 minutes before the
start time. The webcast of the conference call also may be accessed
here, or through the Investors & Newsroom section of the
uniQure website. Following the live webcast, a replay of the call
will be available on our website for several weeks.
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, refractory
temporal lobe epilepsy, Fabry disease, 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, whether our announced
delay of the AMT-130 high-dose procedures will be temporary,
whether the delay will impact any future low-dose procedures,
whether any control patients in the low-dose cohort of the AMT-130
clinical trial study will receive treatment in the third quarter of
2022 or ever, whether we will announce any data readouts for our
AMT-130 clinical trial in 2023, whether our pre-approval site
inspections for AMT-061 will be completed in the third quarter of
this year or will be successful, whether we will ultimately receive
GMP certification for our Lexington manufacturing facility by the
European authorities or other regulatory authorities, whether we
will submit an IND in for AMT-260 for the treatment of refractory
temporal lobe epilepsy (rTLE) in 2023 or ever, whether we will
initiate a GLP toxicology study of AMT-191 for Fabry disease in
non-human primates in the second half of 2022 or submit an IND in
2023, and whether our cash and cash equivalents will fund
operations into the first half of 2025. 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 postponement in
our clinical trial for Huntington’s disease and the pending safety
review, 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 February
25, 2022. 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. CantorDirect: 339-970-7536Mobile:
617-680-9452m.cantor@uniQure.com |
|
Chiara RussoDirect: 617-306-9137Mobile:
617-306-9137c.russo@uniQure.com |
|
Tom MaloneDirect:
339-970-7558Mobile:339-223-8541t.malone@uniQure.com |
uniQure
N.V. |
|
UNAUDITED
CONSOLIDATED BALANCE SHEETS |
|
|
June
30, |
|
December
31, |
|
2022 |
|
2021 |
|
|
(in
thousands, except share and per share amounts) |
|
Current assets |
|
|
|
|
Cash and cash equivalents |
$ |
500,524 |
|
$ |
556,256 |
|
Accounts
receivable and contract asset |
|
3,119 |
|
|
58,768 |
|
Inventories |
|
2,949 |
|
|
- |
|
Prepaid
expenses |
|
15,126 |
|
|
10,540 |
|
Other
current assets and receivables |
|
1,581 |
|
|
2,675 |
|
Total current assets |
|
523,299 |
|
|
628,239 |
|
Non-current assets |
|
|
|
|
Property,
plant and equipment, net |
|
45,984 |
|
|
43,505 |
|
Operating
lease right-of-use assets |
|
28,482 |
|
|
25,573 |
|
Intangible
assets, net |
|
57,450 |
|
|
62,686 |
|
Goodwill |
|
24,976 |
|
|
27,633 |
|
Deferred tax
assets, net |
|
15,046 |
|
|
15,647 |
|
Other
non-current assets |
|
5,974 |
|
|
5,897 |
|
Total non-current assets |
|
177,912 |
|
|
180,941 |
|
Total assets |
$ |
701,211 |
|
$ |
809,180 |
|
Current liabilities |
|
|
|
|
Accounts
payable |
$ |
10,028 |
|
$ |
2,502 |
|
Accrued
expenses and other current liabilities |
|
23,047 |
|
|
28,487 |
|
Current
portion of contingent consideration |
|
8,681 |
|
|
- |
|
Current
portion of operating lease liabilities |
|
6,505 |
|
|
5,774 |
|
Total current liabilities |
|
48,261 |
|
|
36,763 |
|
Non-current liabilities |
|
|
|
|
Long-term
debt |
|
101,890 |
|
|
100,963 |
|
Contingent
consideration, net of current portion |
|
20,405 |
|
|
29,542 |
|
Operating
lease liabilities, net of current portion |
|
30,721 |
|
|
28,987 |
|
Deferred tax
liability, net |
|
9,953 |
|
|
12,913 |
|
Other
non-current liabilities |
|
3,493 |
|
|
4,236 |
|
Total non-current liabilities |
|
166,462 |
|
|
176,641 |
|
Total liabilities |
|
214,723 |
|
|
213,404 |
|
Shareholders' equity |
|
|
|
|
Total shareholders' equity |
|
486,488 |
|
|
595,776 |
|
Total liabilities and shareholders' equity |
$ |
701,211 |
|
$ |
809,180 |
|
|
|
|
|
|
uniQure
N.V. |
|
UNAUDITED
CONSOLIDATED STATEMENTS OF OPERATIONS |
|
|
Three months ended June 30, |
|
2022 |
|
2021 |
|
(in
thousands, except share and per share amounts) |
Total revenues |
$ |
497 |
|
|
$ |
463,868 |
|
Operating expenses: |
|
|
|
Cost of
contract revenues |
|
- |
|
|
|
(23,178) |
|
Cost of
contract manufacturing |
|
(832) |
|
|
|
- |
|
Research and
development expenses |
|
(46,192) |
|
|
|
(32,747) |
|
Selling,
general and administrative expenses |
|
(12,491) |
|
|
|
(17,299) |
|
Total operating expenses |
|
(59,515) |
|
|
|
(73,224) |
|
Other
income |
|
3,186 |
|
|
|
7,590 |
|
Other
expense |
|
(229) |
|
|
|
(226) |
|
(Loss) / income from operations |
|
(56,061) |
|
|
|
398,008 |
|
Non-operating items, net |
|
16,682 |
|
|
|
4,718 |
|
(Loss) / income before income tax benefit /
(expense) |
$ |
(39,379) |
|
|
$ |
402,726 |
|
Income tax
benefit / (expense) |
|
318 |
|
|
|
(3,258) |
|
Net
(loss) / income |
$ |
(39,061) |
|
|
$ |
399,468 |
|
Earnings per ordinary share - basic |
|
|
|
Basic net
(loss) / income per ordinary share |
$ |
(0.84) |
|
|
$ |
8.68 |
|
Earnings per ordinary share - diluted |
|
|
|
Diluted net
(loss) / income per ordinary share |
$ |
(0.84) |
|
|
$ |
8.51 |
|
Weighted
average shares - basic |
|
46,668,554 |
|
|
|
46,037,900 |
|
Weighted
average shares - diluted |
|
46,668,554 |
|
|
|
46,929,870 |
|
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