Amicus Announces Additional Positive Interim Clinical Data for CLN6 Batten Disease Gene Therapy at 48th Annual Meeting of the...
October 24 2019 - 7:00AM
Amicus Therapeutics (Nasdaq: FOLD) today announced additional
positive interim results from its CLN6 Batten disease gene therapy
program licensed from the Abigail Wexner Research Institute (AWRI)
at Nationwide Children’s Hospital. The results are featured in a
poster presentation at the 48th Annual Meeting of the Child
Neurology Society to be held October 23-26, 2019 in Charlotte, NC.
The poster is available in the Events and Presentations section of
the Amicus Therapeutics corporate website at
http://ir.amicusrx.com/events-and-presentations.
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.
Clinical Data
Highlights:Interim safety data are available for 12
patients. 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.
- Safety (n=12): as previously announced,
treatment with AAV-CLN6 gene therapy was generally well tolerated.
The majority of adverse events (AEs) were mild and unrelated to
treatment. Additional details are provided in the poster.
- Hamburg Motor & Language Aggregate Score
(n=8): as previously announced, as of the interim analysis
from this ongoing study, the Hamburg Motor & Language Score, an
assessment of ambulation and speech, shows a positive impact on
motor and language function for 7 of 8 patients treated (16-25
months post-administration of the gene therapy as of this data
cutoff). Additional details are provided in the poster.
- Hamburg Motor, Language, Seizure and Vision Subscores
(n=8): new data on motor, language, seizure and vision
subscores suggest stabilization of these individual components in
most patients from baseline to months 12 and 24, in particular
those patients treated at a younger age, further supporting the
positive impact of one-time intrathecal AAV gene therapy in
children with CLN6 Batten disease. Additional details are provided
in the poster.
- Natural History and Sibling Pair Comparisons:
as previously announced, the AAV-CLN6 gene therapy demonstrated a
positive impact on motor and language function compared to a
natural history data set, including matched comparisons, as well as
in comparisons within sibling pairs. Additional details are
provided in the poster.
Jay Barth, Chief Medical Officer of Amicus
Therapeutics, stated, “We are pleased to share these positive
interim clinical data for our intrathecal AAV gene therapy with the
CLN6 physician community for the first time at Child Neurology
Society. The aggregate and individual Hamburg components, in
addition to the natural history data and sibling comparisons,
continue to suggest that our gene therapy in CLN6 Batten disease
has the potential to halt the progression of this devastating
disease that causes loss of brain function and is fatal in
childhood.”
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, “I
continue to be pleased with the progress of this trial as well as
our collection of matched natural history data to further inform
the results for the AAV-CLN6 gene therapy. I look forward to
following these initial study participants as well as collecting
new data for additional participants as we continue to advance the
program in CLN6 and move additional Batten disease programs through
the clinic in collaboration with Amicus.”
Upcoming Amicus Milestones in Next 12
Months:
- 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.
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 June 30, 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:Sara Pellegrino, IRCVice President,
Investor Relationsspellegrino@amicusrx.com(609) 662-5044
Media:Christopher
ByrneExecutive Director, Corporate
Communicationscbyrne@amicusrx.com 609-662-2798
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