Amicus Therapeutics Announces Additional Positive Interim Clinical Data for CLN6 Batten Disease Gene Therapy at 49th Annual M...
October 12 2020 - 7:00AM
Amicus Therapeutics (Nasdaq: FOLD) today announced additional
positive interim results from its CLN6 Batten disease gene therapy
program, AT-GTX-501. The results are featured in a virtual poster
presentation at the Joint 16th International Child Neurology
Congress and 49th Annual Child Neurology Society Meeting being held
October 12-23, 2020. The presentation is also available in the
Events and Presentations section of the Amicus Therapeutics
corporate website at
http://ir.amicusrx.com/events-and-presentations.
The Abigail Wexner Research Institute (AWRI) at
Nationwide Children’s Hospital is conducting the ongoing Phase 1/2
clinical study of a single one-time intrathecal administration of
AT-GTX-501 gene therapy for variant late-infantile neuronal ceroid
lipofuscinosis 6 (vLINCL6) disease, also known as 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.
Clinical Data
Highlights:Interim safety data are available for 13
children with CLN6 Batten disease. Interim efficacy data are
available for the first 12 children reaching the 12-month timepoint
and for eight children up to 24 months, post-administration of the
AAV-CLN6 gene therapy.
- Safety
(n=13): Treatment with
AT-GTX-501 was generally well tolerated. The majority of adverse
events (AEs) were mild and unrelated to treatment. No pattern of
adverse events related to AAV or CLN6 immunogenicity was observed.
Additional details are provided in the presentation.
- Hamburg Motor &
Language Aggregate Score
(n=12): The Hamburg
Motor & Language Score, an assessment of ambulation and speech,
shows a meaningful effect in slowing disease progression at 12 and
24 months:
- On a combined scale of 0 to 6, the
mean rate of decline was 0.4 vs 1.2 points over 12 months in
treated patients (n=12) vs subjects from the natural history cohort
(n=16).
- On a combined scale of 0 to 6, the
mean rate of decline was 0.6 vs 2.4 points over 24 months in
treated patients (n=8) vs subjects from the natural history cohort
(n=16).
- Natural History
(n=16): Within
the natural history cohort, two years from their first decline on
the Hamburg Motor & Language Score 63% of subjects experienced
an additional 2-point decline (10 out of 16), compared to only 13%
of treated patients (1 out of 8).
Jeff Castelli, Ph.D., Chief Development Officer
of Amicus Therapeutics, stated, “We are pleased to share these
positive interim clinical data for our intrathecal AAV gene therapy
with the CLN6 community. The data continues to suggest that our
gene therapy has the potential to be a treatment option for
children living with CLN6 Batten disease, an ultra-rare,
debilitating condition that leads to progressive declines in
cognitive and motor function, and often results in death early in
life.”
Emily de los Reyes, M.D., Ph.D., 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, “I remain
pleased with the progress of this trial as well as our collection
of natural history data to further inform the results for the
AAV-CLN6 gene therapy. The interim results show that this
investigational gene therapy has the potential to slow the
neurological disease progression in children with CLN6 Batten
disease.”
Regulatory interactions for AT-GTX-501 are
ongoing and the Company expects to provide feedback on the path
forward in 2021.
Amicus has exclusive rights to the CLN6 gene
therapy program developed at the Abigail Wexner Research Institute
at Nationwide Children’s Hospital.
About AT-GTX-501AT-GTX-501 is a
novel gene therapy in Phase 1/2 development for CLN6 Batten
disease, a rare, fatal, inherited lysosomal disorder with no
approved treatment that primarily affects the nervous system.
AT-GTX-501 is dosed in a one-time intrathecal infusion to deliver a
functional copy of the CLN6 gene to cells of the central nervous
system. The therapy is designed to address the underlying enzyme
deficiency that results in progressive cell damage and
neurodevelopmental and physical decline. In the U.S., AT-GTX-501
was granted Rare Pediatric Disease and Orphan Drug designations by
the United States Food and Drug Administration. In the EU, the
Company holds PRIME and orphan medicinal product designations.
About Batten DiseaseBatten
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 disorders, 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 StatementsThis
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, 2019
and Quarterly Report on Form 10-Q for the quarter ended June 30,
2020. 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:Amicus
TherapeuticsAndrew FaughnanDirector, Investor
Relationsafaughnan@amicusrx.com(609) 662-3809
Media:Amicus TherapeuticsDiana
MooreHead of Global Corporate
Communicationsdmoore@amicusrx.com(609) 662-5079
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