uniQure Announces FDA Removes Clinical Hold on Hemophilia B Gene Therapy Program
April 26 2021 - 7:05AM
uniQure Announces FDA Removes Clinical Hold on Hemophilia B Gene
Therapy Program
uniQure N.V. (NASDAQ: QURE), a leading gene therapy company
advancing transformative therapies for patients with severe medical
needs, announced today that the U.S. Food and Drug Administration
(FDA) has removed the clinical hold on the Company’s hemophilia B
gene therapy program after determining the Company satisfactorily
addressed all issues identified by the FDA related to a single
patient diagnosed with hepatocellular carcinoma (HCC) in the HOPE-B
pivotal trial.
“Patient safety is our top priority, and we are
grateful to our advisors and the FDA for their help in resolving
this clinical hold,” stated Ricardo Dolmetsch, Ph.D., president of
research and development at uniQure. “Our comprehensive
investigation showed that AMT-061 (etranacogene dezaparvovec) is
very unlikely to have contributed to the HCC in our patient. We
look forward to announcing top-line 52-week data from the HOPE-B
pivotal trial later this quarter.”
uniQure previously announced on December 21,
2020, that the FDA placed the hemophilia B program on clinical hold
following the diagnosis of HCC in one patient in the HOPE-B trial.
The patient had multiple risk factors associated with HCC,
including a twenty-five-year history of hepatitis C (HCV), history
of hepatitis B (HBV). Chronic infections with hepatitis B and C
have been associated with approximately 80% of HCC cases.1
Following a surgical resection of both tumor and
adjacent liver tissue, multiple analyses conducted by an
independent laboratory and reviewed by leading external experts in
the field show that AAV vector integration in the patient’s tissue
sample was extremely rare and accounted for 0.027% of the cells in
the sample. The integration events were distributed randomly across
the genome, and there was no evidence of clonal expansion or any
dominant integration event. Additionally, whole genome sequencing
of the tumor confirmed that the tumor had genetic mutations that
are characteristic of HCC and are independent of vector
integration. Finally, gene expression analysis of the tumor and
adjacent tissue suggested a precancerous state in the liver that
may have predisposed this patient to HCC.
All patients in uniQure’s hemophilia B gene
therapy program, including the 54 patients in HOPE-B, have had
abdominal ultrasounds performed one year after dosing, and each
will continue to be monitored by their care teams. Patients will
continue to receive abdominal ultrasounds every six-months. No
other cases of HCC have been reported in uniQure clinical trials
conducted in more than 100 patients in hemophilia B and other
indications, with some patients dosed more than 10 years ago.
About Etranacogene
Dezaparvovec
Etranacogene dezaparvovec consists of an AAV5
viral vector carrying a gene cassette with the patent-protected
Padua variant of Factor IX (FIX-Padua). uniQure holds multiple
issued patents in the United States and Canada broadly covering
methods of treating bleeding disorders, including hemophilia B,
using AAV gene therapy with the FIX-Padua variant. Etranacogene
dezaparvovec has been granted Breakthrough Therapy Designation by
the United States Food and Drug Administration and access to
Priority Medicine (PRIME) regulatory initiative by the European
Medicines Agency. In June 2020, the Company and CSL
Behring entered into a licensing agreement providing CSL Behring
with exclusive global rights to etranacogene dezaparvovec. This
licensing agreement has cleared antitrust regulatory review in
Australia and the United Kingdom and is subject to ongoing review
in the United States.
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
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," "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 the Company
will present top line 52-week data in the second quarter of 2021,
or at all. Our actual results could differ materially from those
anticipated in these forward-looking statements for many reasons,
including, without limitation, the risk that the Company has not
and may not be able to definitively determine whether etranacogene
dezaparvovec caused the reported case of hepatocellular carcinoma
(HCC); the risk that additional cases of HCC or other serious
adverse events will be discovered or reported in patients treated
with etranacogene dezaparvovec over time; as well as the risks,
uncertainties and other factors described under the heading "Risk
Factors" in uniQure’s Quarterly Report on Form 10-K filed on March
1, 2021. 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 |
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 |
___________________________
1 El-Serag HB. Epidemiology of viral hepatitis
and hepatocellular carcinoma. Gastroenterology. 2012
May;142(6):1264-1273.e1. doi: 10.1053/j.gastro.2011.12.061. PMID:
22537432; PMCID: PMC3338949.
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