Amicus Therapeutics (Nasdaq: FOLD) today announced positive interim
results from its CLN6 Batten disease gene therapy program licensed
from the Abigail Wexner Research Institute (AWRI) at Nationwide
Children’s Hospital. AWRI is conducting the ongoing Phase 1/2
clinical study of a single one-time intrathecal administration of
AAV-CLN6 gene therapy for CLN6 Batten disease. With no approved
treatments, CLN6 Batten disease is a fatal neurologic disease that
rapidly robs children of their ability to walk, speak, think, and
see.
Interim efficacy data are available for the
first eight children with CLN6 Batten disease for up to 24 months
post-administration of the AAV-CLN6 gene therapy. The Hamburg Motor
& Language Score, an assessment of ambulation and speech,
showed that the AAV-CLN6 gene therapy demonstrated a positive
impact on motor and language function compared to a natural history
dataset, as well as in comparisons within sibling pairs. Treatment
with AAV-CLN6 gene therapy was generally well tolerated. This
intrathecal AAV-CLN6 gene therapy uses the same capsid as the
recently FDA approved systemic gene therapy, also initially
developed at AWRI, for the fatal neurologic disease Spinal Muscular
Atrophy Type 1.
More detailed information on the initial
clinical results can be found in the Events and Presentations
section of the Amicus Therapeutics corporate website at
http://ir.amicusrx.com/events-and-presentations.
Clinical Data Highlights:
- Safety (n=12): The majority of adverse events
(AEs) were mild and unrelated to treatment in a total of 12
patients in the clinical study (exposure duration 6 to 39 months).
No pattern of AEs related to anti-AAV capsid or anti-CLN6
immunogenicity were observed.
- Hamburg Motor & Language (n=8): As of the
interim analysis from this ongoing study, efficacy data show a
positive impact on motor and language function for 7 of 8 patients
treated. These eight patients are from 16-25 months
post-administration of the gene therapy as of this data cutoff.
Seven patients (treated at 19 to 66 months of age) maintained their
Hamburg score or had an initial change (ranging from +1 to -1
points) followed by stabilization. The oldest patient in the study
(treated at 79 months of age) had a 2 point decline.
- Hamburg Motor & Language in Sibling Pairs:
Three treated patients demonstrated stabilization relative to their
untreated siblings in the natural history data set who experienced
substantial declines in motor and language ability or died over the
same time period. For the two pairs of in-study siblings, the
younger siblings demonstrated an increase or stabilization in their
score compared to their older siblings who had an initial change
followed by stabilization.
- Hamburg Motor & Language Natural History
(n=14): An initial natural history cohort from Nationwide
Children’s shows disease progression in all untreated patients,
with at least a 2- to 3-point decline in Hamburg Motor &
Language score from the initial point of decline over 24
months.
John F. Crowley, Chairman and Chief Executive
Officer of Amicus Therapeutics, stated, “This program and
these initial results represent the heart and soul of who we are
and why we do what we do at Amicus. These interim clinical data
suggest that our gene therapy in CLN6 Batten disease has the
potential to halt the progression of this devastating fatal disease
that untreated destroys brain function and kills children. It is
remarkable that most children in this study appear to show
stabilization, particularly the younger children who were able to
maintain high baseline motor and language scores for up to two
years. We know that brain damage here is irreversible, and early
intervention will be critical to preserve the ability to speak and
walk. We look forward to presenting additional data throughout this
year and continuing to advance our CLN6 and other Batten disease
gene therapy programs that all apply the same AAV technology
platform developed by Dr. Brian Kaspar and his former colleagues at
Nationwide Children’s. Early intervention is crucial, so we move
forward with a great sense of urgency here for these children and
their families.”
Tauna Batiste, Executive Director of the Batten
Disease Support and Research Association (BDSRA) commented, “We are
hopeful the clinical results presented today represent a notable
step forward towards finding a treatment for children suffering
from CLN6 disease. This data also provides encouragement for many
families within the broader Batten disease community who are
following the progress of the Amicus gene therapy programs for
CLN3, CLN8, and CLN1 disease. We look forward to seeing additional
data in CLN6 disease and to the continued advancement of all the
Batten disease programs.”
The Hamburg Motor & Language Score (0-6)
separately measures performance of mobility (0-3) and speech (0-3).
For each domain, a 3 represents the child’s normal function and a 0
represents no ability to walk or speak, with each point decline
representing significant impairment. Children living with CLN6
Batten disease often experience typical development for the first
few years of life. Following symptom onset, natural history
indicates that there often is a rapid progression from a 6 to a 0
score within 3-4 years (or a 1-2 point annual decline).
Emily de los Reyes, MD, PhD, Principal
Investigator of the CLN6 clinical trial at AWRI at Nationwide
Children’s and Professor of Clinical Pediatrics and Neurology
at The Ohio State University College of Medicine stated, “Comparing
the data collected during the CLN6 Batten disease clinical trial
with the natural history data collected for Batten’s disease
patients, I am pleased with the progress of this trial. It is
powerful to have pairs of siblings as clinical trial participants
since siblings are expected to have similar progression of the
disease.”
Dr. Brian Kaspar, PhD, former Professor of
Pediatrics, Faculty at AWRI, Dr. Kathrin Meyer, Principal
Investigator at AWRI, and their team at AWRI, in collaboration with
Dr. Jill Weimer’s laboratory at Sanford Research, developed the AAV
gene replacement strategy that is the basis for the AAV-CLN6 gene
therapy.
Dr. Kaspar added, “I am thrilled to see these
clinical results for AAV-CLN6 gene therapy. It is gratifying to see
the teamwork between Nationwide Children’s and Sanford researchers
and clinicians to translate gene therapy from preclinical animal
studies to humans, leading to the current data which demonstrate
evidence for safety and initial efficacy in the first children
treated. I believe this AAV-CLN6 gene therapy has the potential to
make a very meaningful impact for children with CLN6 Batten
disease, and provides great promise to address many types of Batten
disease and other neurologic disorders.”
Upcoming Amicus Milestones in Next 12
Months:
- Presentation of additional data measures in the eight initial
patients at the Amicus Analyst Day this Fall and in a poster
presentation by Dr. de los Reyes at the Child Neurology Society
Annual Meeting, October 23-26, 2019 (Charlotte, NC).
- Collection and presentation of additional natural history data
in CLN6 Batten disease
- Dosing of additional patients
- Advance regulatory discussions
- Manufacturing of additional AAV-CLN6 gene therapy underway at
Thermo Fisher (Brammer Bio)
- Continued advancement of AAV gene therapy programs in CLN3,
CLN8 and CLN1 Batten disease.
Conference Call and Webcast on August 1,
2019 at 8:30 a.m. ET
Amicus Therapeutics will host a conference call
and audio webcast today, August 1, 2019 at 8:30 a.m. ET. Members of
the Amicus leadership team to discuss the positive interim Phase
1/2 CLN6 Batten disease data. Interested participants and investors
may access the conference call by dialing (877) 303-5859
(U.S./Canada) or (678) 224-7784 (international), conference ID:
1186207.
A live audio webcast can also be accessed via
the Investors section of the Amicus Therapeutics corporate website
at http://ir.amicusrx.com/, and will be archived for 30 days. Web
participants are encouraged to register on the website 15 minutes
prior to the start of the call. A replay of the call will be
available for seven days beginning at 11:30 a.m. ET on August 1,
2019. Access numbers for this replay are (855) 859-2056
(U.S./Canada) and (404) 537-3406 (international); conference ID:
1186207.
About Batten Disease
Batten disease is the common name for a broad
class of rare, fatal, inherited disorders of the nervous system
also known as neuronal ceroid lipofuscinoses, or NCLs. In these
diseases, a defect in a specific gene triggers a cascade of
problems that interferes with a cell’s ability to recycle certain
molecules. Each gene is called CLN (ceroid lipofuscinosis,
neuronal) and given a different number designation as its subtype.
There are 13 known forms of Batten disease often referred to as
CLN1-8; 10-14. The various types of Batten disease have similar
features and symptoms but vary in severity and age of onset.
Most forms of Batten disease/NCLs usually begin
during childhood. The clinical course often involves progressive
loss of independent adaptive skills such as mobility, feeding, and
communication. Patients may also experience vision loss,
personality changes, behavioral problems, learning impairment, and
seizures. Patients typically experience progressive loss of
motor function and eventually become wheelchair-bound, are then
bedridden, and die prematurely.
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq: FOLD) is a global,
patient-dedicated biotechnology company focused on discovering,
developing and delivering novel high-quality medicines for people
living with rare metabolic diseases. With extraordinary patient
focus, Amicus Therapeutics is committed to advancing and expanding
a robust pipeline of cutting-edge, first- or best-in-class
medicines for rare metabolic diseases. For more information please
visit the company’s website at www.amicusrx.com, and follow on
Twitter and LinkedIn.
Forward-Looking Statements
This press release contains "forward-looking
statements" within the meaning of the Private Securities Litigation
Reform Act of 1995 relating to preclinical and clinical development
of our product candidates, the timing and reporting of results from
preclinical studies and clinical trials and the prospects and
timing of the potential regulatory approval of our product
candidates. In particular, this press release relates to
interim data from an ongoing Phase 1/2 study to investigate
intrathecal administration of AAV-CLN6 gene therapy. The
inclusion of forward-looking statements arising from this interim
data, ongoing study and natural history preliminary data should not
be regarded as a representation by us that any of our plans will be
achieved. Any or all of the forward-looking statements in this
press release may turn out to be wrong and can be affected by
inaccurate assumptions we might make or by known or unknown risks
and uncertainties. For example, with respect to statements
regarding the goals, progress, timing, and outcomes of discussions
with regulatory authorities, and in particular the potential goals,
progress, timing, and results of preclinical studies and clinical
trials, actual results may differ materially from those set forth
in this release due to the risks and uncertainties inherent in our
business, including, without limitation: the potential that results
of clinical or preclinical studies indicate that the product
candidates are unsafe or ineffective; the potential that it may be
difficult to enroll patients in our clinical trials; the potential
that regulatory authorities, including the FDA, EMA, and PMDA, may
not grant or may delay approval for our product candidates; the
potential that preclinical and clinical studies could be delayed
because we identify serious side effects or other safety issues;
and the potential that we will need additional funding to complete
all of our studies. Further, the results of earlier preclinical
studies and/or clinical trials may not be predictive of future
results. The interim data and Phase 1/2 study discussed herein is
inherently preliminary and early in the study, derived from a
limited patient set, and later trial results with this patient set
or others may not be consistent with these preliminary results. In
addition, all forward-looking statements are subject to other risks
detailed in our Annual Report on Form 10-K for the year ended
December 31, 2018 and Quarterly Report on Form 10-Q for the quarter
ended March 31, 2019. You are cautioned not to place undue reliance
on these forward-looking statements, which speak only as of the
date hereof. All forward-looking statements are qualified in their
entirety by this cautionary statement, and we undertake no
obligation to revise or update this news release to reflect events
or circumstances after the date hereof.
CONTACTS:
Investors/Media:Amicus TherapeuticsSara
Pellegrino, IRCVice President, Investor Relations & Corporate
Communicationsspellegrino@amicusrx.com(609) 662-5044
Media:
Amicus TherapeuticsMarco Winkler Director, Corporate
Communications mwinkler@amicusrx.com(609) 662-2798
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