Amicus Therapeutics Announces Planned Analysis of Primary Endpoints in Phase 3 Epidermolysis Bullosa Study
Based on discussions and written communication from the Dermatology Division of the
- 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
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.
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Forward-Looking Statements
This 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
1 http://www.fda.gov/downloads/drugs/guidancecomplianceregulatoryinformation/guidances/ucm071324.pdf
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CONTACTS: Investors/Media:Source:Amicus Therapeutics Sara Pellegrino Senior Director, Investor Relations spellegrino@amicusrx.com (609) 662-5044
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