uniQure Announces Clinical Update on First Patients in Phase I/II
Clinical Trial of AMT-130 Gene Therapy for the Treatment of
Huntington’s Disease
uniQure N.V. (NASDAQ: QURE), a leading gene therapy company
advancing transformative therapies for patients with severe medical
needs, today announced initial observations on the first four
patients enrolled in the lower-dose cohort of its ongoing Phase
I/II clinical trial of AMT-130 for the treatment of Huntington’s
disease. Two of the four enrolled patients received AMT-130, and
two patients experienced an imitation (sham) surgery in this
randomized, blinded clinical trial being conducted in the United
States.
“We are pleased with these data that support the
tolerability of AMT-130, a potential one-time gene-therapy approach
for Huntington’s disease,” stated Ricardo Dolmetsch, Ph.D.,
president of research and development at uniQure. “Neurofilament
light chain, a key biomarker of injury in the brain, trended
downward and returned to baseline levels in the treated patients,
and structural MRI data are consistent with the safety profile of
AMT-130. Based on recommendations of the DSMB and steering
committee, we will disclose efficacy data including volumetric MRI
changes once all patients in the first two cohorts are unblinded.
We look forward to evaluating safety, biomarker and functional data
on a larger number of patients and over a longer period during the
ongoing study.”
“AMT-130 appears to be generally well tolerated
both immediately after surgical administration and at one year of
follow up in these four patients,” stated Jody Corey-Bloom, M.D.,
PhD, professor of neurosciences at University of California, San
Diego and chairperson of the Data Safety Monitoring Board (DSMB)
for AMT-130. “We are encouraged by the early data in two treated
patients and will continue to provide comprehensive safety
oversight of the program as additional patients are enrolled and
the trial expands into Europe. The DSMB will continue to
oversee the conduct of this important program, and will evaluate
safety, biomarker, imaging, and functional data as the program
advances.”
One-Year Observations on AMT-130
The first four patients were a heterogeneous
group representing a spectrum of Stage I-II early manifest disease
with 41-44 CAG repeats, baseline Total Functional Capacity scores
of 10-13 and Total Motor Scores of 7-23.
- AMT-130 was generally well
tolerated in the treated patients at the lower dose of 6x1012
vector genomes (vg).
- There have been no serious adverse
events (SAEs) to date related to AMT-130 in these patients.
- NfL increased as expected
immediately following the AMT-130 surgical procedure and returned
to baseline in the two treated patients. NfL remained relatively
constant in the two untreated control patients.
- Structural magnetic resonance
imaging did not reveal any clinically meaningful safety findings in
either treated or control patients at one year of follow-up.
- Measurements of total and mutant
HTT protein in the cerebral spinal fluid of the four patients were
highly variable and inconclusive.
- The safety profile of AMT-130 in
the low-dose cohort to date supports continued enrollment of
patients in the higher-dose cohort of 6x1013 vg. Nineteen patients
have been enrolled in the clinical trial to date, including 9 of 16
in the higher-dose cohort.
A clinical update on the low-dose cohort of ten
patients, largely focused on safety, is expected in the second
quarter of 2022 after patient unblinding. Full safety and efficacy
data from the first two cohorts in the Phase I/II clinical trial
are expected in the first half of 2023 after all patients in the
higher-dose cohort have achieved one year of follow-up.
Planned Expansion of Phase I/II Studies of
AMT-130
The U.S. Phase I/II clinical trial of AMT-130
for the treatment of Huntington’s disease is exploring the safety,
tolerability, and efficacy signals in a planned 26 total patients
with early manifest Huntington’s disease split into a 10 patient,
low-dose cohort followed by a 16 patient, higher-dose cohort;
patients will be randomized to treatment with AMT-130 or an
imitation (sham) surgery. The multi-center trial consists of a
blinded 12-month core study period followed by unblinded long-term
follow-up for five years. A total of 16 patients in the clinical
trial will receive a single administration of AMT-130 through
MRI-guided, convection-enhanced stereotactic neurosurgical delivery
directly into the striatum (caudate and putamen).
uniQure also plans to initiate a third cohort in
the ongoing U.S. Phase I/II clinical trial in the second half of
2022 that will explore the use of alternative stereotactic
navigation systems to simplify placement of catheters for infusions
of AMT-130. This will be explored in two steps and include up to 18
additional randomized patients who will receive the higher dose of
6x1013 vg.
The European, open-label Phase Ib/II study of
AMT-130 will enroll 15 patients with early manifest Huntington’s
disease across two dose cohorts. Screening has been initiated, and
the first procedures in the lower-dose cohort are expected to be
complete in early 2022. 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.
AMT-130 is uniQure’s first clinical program
focusing on the central nervous system (CNS) incorporating its
proprietary miQURE® platform.
Investor Conference Call and Webcast
Information
uniQure management will host an investor
conference call and webcast today, Thursday, December 16, 2021, at
8:30 a.m. ET. The conference call may be accessed by dialing (833)
962–1471 for domestic callers and +44 0800 0288 438 for
international callers. The conference call ID: 2866185. Please
specify to the operator that you would like to join the “uniQure
Conference Call.” If you are joining the conference call, please
dial-in 15 minutes before the start time. The webcast of the
conference call may also be accessed through the Investors &
Newsroom section of the uniQure website. Following the live
webcast, a replay of the call will be archived for several
weeks.
About Huntington’s Disease
Huntington’s disease is a rare, inherited
neurodegenerative disorder that leads to motor symptoms including
chorea, and behavioral abnormalities and cognitive decline
resulting in progressive physical and mental deterioration. The
disease is an autosomal dominant condition with a disease-causing
CAG repeat expansion in the first exon of the huntingtin gene that
leads to the production and aggregation of abnormal protein in the
brain. Despite the clear etiology of Huntington’s disease, there
are no currently approved therapies to delay the onset or to slow
the disease’s progression.
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 temporal lobe epilepsy, Alzheimer’s,
Parkinson’s and ALS. 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
we will be able to evaluate data on a larger number of patients or
over a longer period during the ongoing study; whether safety data
on the first cohort of ten patients will be available in the second
quarter of 2022 or ever; whether full safety and efficacy data from
the Phase I/II clinical trial will be available for cohorts 1 and 2
in the first half of 2023 or ever; whether 16 patients or any
patients in the clinical trial will receive a higher dose
administration of AMT-130; whether we will initiate a third cohort
in the ongoing U.S. Phase I/II clinical trial in the second half of
2022 or ever; whether we will enroll 15 patients or any patients in
a European, open-label Phase Ib/II study of AMT-130; whether we
will complete the first procedures in the low-dose cohort of the
European study in early 2022 or ever; whether the European study
will establish safety, proof of concept, or the optimal dose of
AMT-130 to take forward into Phase III development or into a
confirmatory study or for any purpose; and whether an accelerated
registration pathway will be feasible or available at all. Our
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 and our collaborators’
clinical development activities, clinical results, collaboration
arrangements, corporate reorganizations and strategic shifts,
regulatory oversight, product commercialization and intellectual
property claims, as well as the risks, uncertainties and other
factors described under the heading “Risk Factors”
in uniQure’s periodic securities filings,
including its Annual Report on Form 10-K filed March 1,
2021 and Quarterly Report on Form 10-Q filed on October 25,
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: |
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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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