Amicus Therapeutics Announces Planned Analysis of Primary Endpoints in Phase 3 Epidermolysis Bullosa Study
May 19 2017 - 7:30AM
Amicus Therapeutics, Inc. (Nasdaq:FOLD), has completed the
analysis plan for the primary endpoints in the blinded ongoing
Phase 3 clinical study (ESSENCE) of the novel topical medicine
SD-101 for epidermolysis bullosa (EB). SD-101 was one of the first
treatments to receive the FDA’s Breakthrough Therapy designation.
ESSENCE is a double-blind, placebo-controlled registration study
that completed enrollment of more than 160 patients who have a
documented diagnosis of Simplex, Recessive Dystrophic, or
Junctional non-Herlitz EB.
Based on discussions and written communication
from the Dermatology Division of the U.S. Food and Drug
Administration, Amicus will analyze the primary endpoints for the
Phase 3 ESSENCE study as follows:
- The endpoint of “time to wound closure within 3 months” will be
analyzed first. If the difference between SD-101 6% and placebo is
statistically significant (p ≤ 0.05), then the study will be
considered a success.
- If the first endpoint is statistically significant, then
“proportion of patients with target wound closure at month 3” will
be analyzed and considered statistically significant at p ≤
0.05.
Time to Wound Closure is a clinically relevant
endpoint in EB, given the constant and often chronic nature of EB
wounds. Time to Wound Closure is as an acceptable efficacy endpoint
for wound-related diseases, per FDA guidance.1
Jay Barth, MD, Chief Medical Officer of Amicus
Therapeutics, Inc. stated, “We believe that the planned analysis of
the primary endpoints increases our overall likelihood for success
in the Phase 3 ESSENCE study of SD-101 for epidermolysis bullosa.
Time to Wound Closure accounts for wound healing across all study
time points, offering greater precision for discriminating
treatment effects and treatment differences between SD-101 and
placebo. We have been very pleased with the collaborative
interactions with the Dermatology Division of the FDA under our
breakthrough therapy designation for SD-101. We look forward to
announcing data from this study.”
Amicus is on track to report top-line data from
the ESSENCE study during the third quarter of 2017. 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. Patients 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 bandaging and cleaning of open wounds to
prevent infection.
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq:FOLD) is a 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, the
timing and reporting of results from a clinical trial, the
prospects and timing of the potential regulatory approval of our
product candidates. 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 goals, progress, timing, and outcomes of
discussions with regulatory authorities, and in particular the
potential goals, progress, timing, and results of 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 studies indicate that the product candidates are 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 we may not
be successful in commercializing our product candidates if and when
approved; the potential that 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. 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 as well as our Quarterly Report on Form
10-Q for the quarter ended March 31 2017. 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.
1http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm071324.pdf
FOLD–G
CONTACTS:
Investors/Media:
Amicus Therapeutics
Sara Pellegrino
Senior Director, Investor Relations
spellegrino@amicusrx.com
(609) 662-5044
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