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 conditional marketing authorization (CMA)
from the European Commission for HEMGENIX® (etranacogene
dezaparvovec), the first and only one-time gene therapy for the
treatment of severe and moderately severe hemophilia B.
HEMGENIX is approved for the treatment of adults
with severe and moderately severe hemophilia B (congenital Factor
IX deficiency) in adult patients without a history of Factor IX
inhibitors. It is the first approved gene therapy for hemophilia B
in the European Union (EU) and European Economic Area (EEA).
“The European approval of HEMGENIX represents
another major milestone in the field of genomic medicine and
innovation in the treatment of people living with hemophilia B,”
said Matt Kapusta, chief executive officer of uniQure. “This
achievement is based on more than a decade of research and clinical
development led by uniQure, and we are grateful for the tireless
dedication of our employees, clinicians, patients and their
families who made this possible.”
Hemophilia B is a rare, lifelong bleeding
disorder caused by a single gene defect, resulting in insufficient
production of factor IX, a protein primarily produced by the liver
that helps blood clots form. Treatments for moderate to severe
hemophilia B include prophylactic infusions of factor IX
replacement therapy to temporarily replace or supplement low levels
of blood-clotting factor and, while these therapies are effective,
those with hemophilia B must adhere to strict, lifelong infusion
schedules. They may also still experience spontaneous bleeding
episodes as well as limited mobility, joint damage or severe pain
as a result of the disease. For appropriate patients, HEMGENIX has
been shown in clinical trials to allow people living with
hemophilia B to produce their own factor IX, which can lower the
risk of bleeding.
“The approval of HEMGENIX in Europe is the
essence of great science delivering a medicine that we believe can
transform the treatment paradigm for both people living with
hemophilia B and the healthcare professionals who treat them,” said
Dr. Bill Mezzanotte, head of research & development and chief
medical officer, CSL. “HEMGENIX, and our partnership with uniQure,
underscore CSL’s promise to pursue, develop and deliver disruptive
innovations when patients can benefit, particularly in disease
states we know well like hemophilia B.”
The European Commission’s decision follows the
CHMP’s positive opinion in December 2022, based on findings from
the pivotal HOPE-B trial, the largest gene therapy trial in
hemophilia B to date. These findings showed that hemophilia B
patients treated with HEMGENIX demonstrated stable and durable
increases in mean Factor IX activity levels (with a mean Factor IX
activity of 36.9%) which led to an adjusted annualized bleed rate
(ABR) reduction of 64%. Following infusion, 96% of patients
discontinued routine Factor IX prophylaxis and mean Factor IX
consumption was reduced by 97% at 18 months post-treatment,
compared to the lead-in period.
The HOPE-B study 24-month analysis continued to show a sustained
and durable effect of HEMGENIX. In a clinical setting, the
treatment is generally well-tolerated with no serious
treatment-related adverse events.
uniQure conducted the research and clinical
development for the product, which included three clinical trials
across 34 global sites and involving 67 adults with hemophilia B.
In May 2021, uniQure and CSL completed a licensing transaction
providing CSL Behring with exclusive rights to commercialize and
continue clinical development of HEMGENIX globally. uniQure is
responsible for the global manufacturing of the product at its
licensed Lexington, Massachusetts facility. Under the terms of the
agreement, uniQure has received payments from CSL totaling
approximately $500 million and is eligible to receive up to an
additional $1.5 billion in commercial milestone payments and
tiered, double-digit royalties in a range up to a low-twenties
percentage of net product sales arising from the collaboration.
“This approval marks an important step forward
in the treatment of hemophilia B, which could be transformative for
people who are debilitated by bleeds into their muscles, joints and
internal organs, alleviating the burden of lifelong intravenous
infusions of Factor IX products,” said Professor Wolfgang Miesbach,
head of coagulation disorders at the Comprehensive Care Center,
University Hospital of Frankfurt. “Data from the HOPE-B study
demonstrate the potential of HEMGENIX to remove the need for
routine prophylaxis, by providing durable Factor IX activity, as
well as improved bleeding outcomes and quality of life for people
with hemophilia B.”
The multi-year clinical development of HEMGENIX
was led by uniQure and sponsorship of the clinical trials
transitioned to CSL after it licensed global rights to
commercialize the treatment. In the United Kingdom, The Medicines
and Healthcare products Regulatory Agency is currently reviewing
CSL’s submission for HEMGENIX. HEMGENIX was approved by the U.S.
Food and Drug Administration in November 2022. Product information
on HEMGENIX, including its prescribing information, will be
provided by CSL Behring.
About Hemophilia BHemophilia 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 HEMGENIXHEMGENIX is a
gene therapy that reduces the rate of abnormal bleeding in eligible
people with hemophilia B by enabling the body to continuously
produce factor IX, the deficient protein in hemophilia B. It uses
AAV5, a non-infectious viral vector, called an adeno-associated
virus (AAV). The AAV5 vector carries the Padua gene variant of
Factor IX (FIX-Padua) to the target cells in the liver, generating
factor IX proteins that are 5x-8x more active than normal. These
genetic instructions remain in the target cells, but generally do
not become a part of a person’s own DNA. Once delivered, the
new genetic instructions allow the cellular machinery to produce
stable levels of factor IX.
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 HEMGENIX. Fifty-four adult hemophilia B patients
classified as having a diagnosis of moderately severe or severe
hemophilia B and requiring prophylactic Factor IX 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 HEMGENIX® at the 2x10^13 gc/kg
dose. Patients with pre-existing neutralizing antibodies (NAbs) to
AAV5 were not excluded from the trial. A total of 54 patients
received a single dose of HEMGENIX 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 Factor IX 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 Factor IX transgene
expression.
Results from the pivotal HOPE-B study
demonstrated that HEMGENIX produced mean Factor IX activity of 36.9
IU/dL at 18 months post infusion. At 24 months follow-up, Factor IX
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
Factor IX-treated bleeds was reduced by 77 percent (3.65 to 0.83;
p<0.0001) over months seven to 18. From day 21 through to months
7 to 24, 52 of 54 (96.3%) treated patients remained free of
continuous routine Factor IX prophylaxis. The mean consumption of
Factor IX replacement therapy significantly decreased by 248,392.6
IU/year/patient (96.52%; 1-sided p< 0.0001) between month 7 to
24 following treatment with HEMGENIX® compared to standard of care
routine Factor IX prophylaxis during the lead-in period.
Further analyses showed that there was no
clinically meaningful correlation between patient AAV5 NAb levels
at baseline and Factor IX activity.
No serious adverse reactions were identified.
One death resulting from urosepsis and cardiogenic shock in a
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 HEMGENIX® by independent molecular
tumor characterization and vector integration analysis. No
inhibitors to Factor IX were reported.
About uniQure uniQure is
delivering on the promise of gene therapy – single treatments with
potentially curative results. The recent approval of our gene
therapy for hemophilia B – an historic achievement based on more
than a decade of research and clinical development – represents a
major milestone in the field of genomic medicine and ushers in a
new treatment approach for patients living with hemophilia. We are
now leveraging our modular and validated technology platform to
advance a pipeline of proprietary gene therapies for the treatment
of patients with Huntington's disease, refractory temporal lobe
epilepsy, ALS, Fabry disease, and other severe
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 we are able to bring AMT-061 to people
living with hemophilia B and whether the treatment will be
transformational. 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 |
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 |
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