uniQure N.V. (NASDAQ: QURE), a leading gene therapy company
advancing transformative therapies for patients with severe medical
needs, announced that its partner, global biotechnology leader CSL
(ASX: CSL), has received a positive opinion recommending
conditional marketing authorization (CMA) for etranacogene
dezaparvovec from the Committee for Medicinal Products for Human
Use (CHMP) of the European Medicines Agency (EMA). Etranacogene
dezaparvovec is a one-time gene therapy for the treatment of
appropriate adults with hemophilia B that reduces the rate of
annual bleeds after a single infusion.1,2 uniQure conducted the
multi-year clinical development program for etranacogene
dezaparvovec and will leverage its leading gene therapy
manufacturing capabilities to supply CSL for commercialization.
“We are very pleased with this positive opinion
recommending conditional marketing authorization for etranacogene
dezaparvovec,” said Matt Kapusta, chief executive officer of
uniQure. “The program’s multi-year clinical journey began in Europe
with our first-in-human Phase I/II study, and the favorable CHMP
opinion moves etranacogene dezaparvovec one step closer to the
European hemophilia B community. We look forward to continuing our
partnership with CSL to help bring this potentially life-changing
treatment option to people with hemophilia B.”
Etranacogene dezaparvovec was recently approved
by the U.S. Food and Drug Administration. CSL licensed the
exclusive global rights to etranacogene dezaparvovec from uniQure
in May 2021, and is now solely responsible for the further
development, registration, and commercialization of the therapy.
uniQure is responsible for the global commercial supply of
etranacogene dezaparvovec.
The positive CHMP opinion is based on findings
from the pivotal HOPE-B trial, the largest gene therapy trial in
hemophilia B to date.3,4 These findings showed that hemophilia B
patients treated with etranacogene dezaparvovec demonstrated stable
and durable increases in mean Factor IX (FIX) activity levels (with
a mean FIX activity of 36.9%) which led to an adjusted annualized
bleed rate (ABR) reduction of 64%.1 Following infusion of
etranacogene dezaparvovec, 96% of patients discontinued routine FIX
prophylaxis.1
The HOPE-B study 24 months analysis showed a
sustained and durable effect of etranacogene dezaparvovec.5,6 In a
clinical setting, etranacogene dezaparvovec continued to be
generally well-tolerated with no serious treatment-related adverse
events.5,6
The positive opinion from the CHMP will now be
reviewed by the European Commission, which has the authority to
approve medicines for the European Union member states.
About Hemophilia
B Hemophilia B is a life-threatening rare disease.
People with the condition are particularly vulnerable to bleeds in
their joints, muscles, and internal organs, leading to pain,
swelling, and joint damage. Current treatments for moderate to
severe hemophilia B include life-long prophylactic infusions of
factor IX to temporarily replace or supplement low levels of the
blood-clotting factor.
About Etranacogene
DezaparvovecEtranacogene dezaparvovec is an
adeno-associated virus five (AAV5)-based gene therapy given as a
one-time treatment for moderately severe to severe hemophilia B
patients. Etranacogene dezaparvovec (also known as CSL222,
previously known as AMT-061) uses a specific type of AAV, called
AAV5, as its vector. The AAV5 vector carries the Padua gene variant
of Factor IX (FIX-Padua), which generates FIX proteins that are
5x-8x more active than normal.
About the Pivotal HOPE-B
TrialThe pivotal Phase III HOPE-B trial is an ongoing,
multinational, open-label, single-arm study to evaluate the safety
and efficacy of etranacogene dezaparvovec. Fifty-four adult
hemophilia B patients classified as having a diagnosis of
moderately severe or severe hemophilia B and requiring prophylactic
FIX replacement therapy were enrolled in a prospective, six-month
observational period during which time they continued to use their
current standard of care therapy to establish a baseline Annual
Bleeding Rate (ABR). After the six-month lead-in period, patients
received a single intravenous administration of etranacogene
dezaparvovec at the 2x10^13 gc/kg dose. Patients were not excluded
from the trial based on pre-existing neutralizing antibodies (NAbs)
to AAV5. A total of 54 patients received a single dose of
etranacogene dezaparvovec in the pivotal trial, with 53 patients
completing at least 18 months of follow-up. The primary endpoint in
the pivotal HOPE-B study was 52-week ABR after achievement of
stable FIX expression compared with the six-month lead-in period.
For this endpoint, ABR was measured from month seven to month 18
after infusion, ensuring the observation period represented a
steady-state FIX transgene expression.
Results from the pivotal HOPE-B study
demonstrated that etranacogene dezaparvovec produced mean FIX
activity of 36.9 IU/dL at 18 months post infusion. At 24 months
follow-up, FIX activity remained stable at 36.7 IU/DL. After the
six-month lead-in period post-infusion, the adjusted annualized
bleeding rate (ABR) (1.51) for all bleeds was reduced by 64 percent
(p=0.0002) and all FIX-treated bleeds was reduced by 77 percent
(3.65 to 0.83; p<0.0001) over months seven to 18. 96 percent of
subjects treated with a full dose of etranacogene dezaparvovec
discontinued use of routine prophylaxis, with an overall 97 percent
reduction in mean unadjusted annualized FIX consumption
of 257338.8 IU/yr/participant to 8486.6
IU/yr/participant (from lead-in period to months 13-18).
Further analyses showed that there was no
clinically meaningful correlation between patient AAV5 NAb levels
at baseline and FIX activity.
No serious adverse reactions were reported. One
death resulting from urosepsis and cardiogenic shock in a
77-year-old patient at 65 weeks following dosing was considered
unrelated to treatment by investigators and the company sponsor. A
serious adverse event of hepatocellular carcinoma was determined to
be unrelated to treatment with etranacogene dezaparvovec by
independent molecular tumor characterization and vector integration
analysis. No inhibitors to FIX were reported.
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
StatementsThis 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," “establish,” "estimate,"
"expect," "goal," "intend," "look forward to", "may," "plan,"
"potential," "predict," "project," “seek,” "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,
statements about whether etranacogene dezaparvovec will be approved
in Europe, whether we are able to bring etranacogene dezaparvovec
to people living with hemophilia B, and whether the treatment will
be life-changing. 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, the impact of financial and
geopolitical events 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: |
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FOR MEDIA: |
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Maria E. Cantor Direct:
339-970-7536 Mobile:
617-680-9452 m.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 |
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1 Miesbach W et al. Oral presentation at 15th EAHAD meeting,
February 20222 Summary of Product Characteristics: etranacogene
dezaparvovec. Data on file.3 ASH Clinical News. New HOPE for
Hemophilia B: One-Time Gene Therapy Abolishes Bleeding in Most
Patients. Available at:
https://ashpublications.org/ashclinicalnews/news/5441/New-HOPE-for-Hemophilia-B-One-Time-Gene-Therapy.
[Accessed December 2022]4 ClinicalTrials.gov. HOPE-B: Trial of
AMT-061 in Severe or Moderately Severe Hemophilia B Patients.
Available at: https://clinicaltrials.gov/ct2/show/NCT03569891.
[Accessed December 2022]5 Miesbach W et al. Poster presentation at
ASH meeting, December 20226 Pipe S et al. Poster presentation at
ASH meeting, December 2022
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