Amicus Therapeutics Completes Enrollment in ESSENCE Phase 3 Epidermolysis Bullosa Clinical Study
April 03 2017 - 7:30AM
Target Exceeded with More than 160
Patients Enrolled
Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company
at the forefront of therapies for rare and orphan diseases, has
completed enrollment in the ongoing Phase 3 clinical study
(ESSENCE) of the novel topical medicine SD-101 for patients with
all 3 major types of epidermolysis bullosa (EB) (Simplex, Recessive
Dystrophic, and Junctional non-Herlitz EB). With the achievement of
full enrollment, top-line data from this study are expected in the
third quarter of 2017.
John F. Crowley, Chairman and Chief Executive
Officer of Amicus Therapeutics, Inc. stated, “The completion of
enrollment in our global Phase 3 ESSENCE study of SD-101 for
epidermolysis bullosa is a significant accomplishment for our team
at Amicus as well as for the EB community. This is the most
advanced clinical study for EB, and we look forward to announcing
top-line data from this study in the third quarter of this
year.”
The FDA has granted Breakthrough Therapy
designation for SD-101 based on results from a Phase 2a study for
the treatment of lesions in patients suffering with EB. SD-101 is
the first-ever treatment in clinical studies to show improvements
in wound closure across all major EB types.
John C. Browning, Chief of Dermatology at
Children's Hospital of San Antonio, stated, “The full enrollment in
this Phase 3 trial is a significant step forward as we look to
advance new wound healing treatments for EB. With no currently
approved treatment options, there is an urgent need among people
living with EB, as well as their caregivers. There has been
tremendous commitment among patients and families, advocacy
organizations and study investigators in working alongside Amicus
to raise awareness of and drive enrollment in this important
study.”
The ESSENCE Study is a Phase 3 double-blind,
placebo-controlled study that enrolled more than 160 patients who
have a documented diagnosis of Simplex, Recessive Dystrophic, or
Junctional non-Herlitz EB. To date, more than 95 percent of
patients completing the 3-month primary treatment period have
elected to continue in the open-label extension study.
About Epidermolysis Bullosa
(EB)EB is a rare, genetic disorder that manifests as
blistering or erosion of the skin, and, in some cases, the
epithelial lining of other organs. EB is chronic, potentially
disfiguring, and in some cases fatal. Individuals with EB have
painful wounds and blisters that can lead to infection and
scarring. There are many genetic and symptomatic variations of EB,
but all forms share the common symptom of fragile skin that
blisters and tears, sometimes from the slightest friction or
trauma. There is currently no approved treatment for EB. Current
standard of care consists of pain management and the cleaning and
bandaging of open wounds to prevent infection.
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq:FOLD) is a global biotechnology company
at the forefront of therapies for rare and orphan diseases. The
Company has a robust pipeline of advanced therapies for a broad
range of human genetic diseases. Amicus’ lead programs in
development include the small molecule pharmacological chaperone
migalastat as a monotherapy for Fabry disease, SD-101 for
Epidermolysis Bullosa (EB), as well as novel enzyme replacement
therapy (ERT) and biologic products for Fabry disease, Pompe
disease, and other rare and devastating diseases.
Forward-Looking StatementsThis
press release contains "forward-looking statements" within the
meaning of the Private Securities Litigation Reform Act of 1995
relating to clinical development of our product candidate and the
timing and reporting of results from our clinical trial. The
inclusion of forward-looking statements 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 potential goals,
progress, and timing of results of our clinical trial, 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 our
clinical study indicates that the product candidate is unsafe or
ineffective; 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 the clinical study
could be delayed because we identify serious side effects or other
safety issues; and the potential that we will need additional
funding to complete our study. Further, the results of earlier
preclinical studies and/or clinical trials may not be predictive of
future 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, 2016. 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.
FOLD–G
CONTACTS:
Investors/Media:
Amicus Therapeutics
Sara Pellegrino
Senior Director, Investor Relations
spellegrino@amicusrx.com
(609) 662-5044
Media:
MWW PR
Sid Dinsay
sdinsay@mww.com
(646) 381-9017
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