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Amicus Therapeutics Announces Top-Line Phase 3 Results for SD-101 in Epidermolysis BullosaStudy Did Not Meet Primary Endpoints Conference Call and Webcast Today at 8:30am ET CRANBURY, N.J., Sept. 13, 2017 (GLOBE NEWSWIRE) -- Amicus Therapeutics, Inc. (Nasdaq:FOLD) reported that top-line data from the randomized, double-blind, placebo-controlled Phase 3 clinical study (ESSENCE, SD-005) to assess the efficacy and safety of the novel topical wound-healing agent SD-101 did not meet the primary endpoints or secondary endpoints in participants with epidermolysis bullosa (EB). The Phase 3 ESSENCE study randomized 169 participants with a documented diagnosis of Simplex, Recessive Dystrophic, or Junctional non-Herlitz epidermolysis bullosa to SD-101 (n=82) or placebo (n=87) during a three-month primary treatment period. SD-101 did not show a statistically significant difference from placebo in the intent to treat (ITT) population (n=169). The first primary endpoint, the time to target wound closure within 3 months, was not different between groups (Hazard ratio=1.004, p=0.985).1 The second primary endpoint, the percentage of patients achieving target wound closure by month 3, was also not different between groups (49% SD-101; 54% placebo; p=0.390).2 Similarly, the secondary endpoints did not reach statistical significance versus placebo. Encouraging trends in wound closure were observed in certain sub-populations. Treatment-emergent adverse events (TEAEs) were similar across both the SD-101 and placebo groups. The most common TEAEs were nasopharyngitis (common cold), pruritis (itchy skin), and pyrexia (fever). John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. stated, “We are disappointed that this Phase 3 study of SD-101 did not meet the primary endpoints in epidermolysis bullosa, an utterly devastating rare genetic disorder with no approved treatment options. In keeping with our Amicus mission, we have a strong commitment to the EB community and will work closely with investigators and other leading experts to understand and to share these data. We would like to sincerely thank the patients, families, clinical investigators, regulators and our Amicus team involved in this EB program. In seeking to develop novel, high quality therapies for those living with devastating rare diseases we may sometimes fail. But we would rather be the first to fail, than the last to try. Our vision at Amicus remains steadfast and is focused on building a leading global biotechnology company that delivers significant benefits for people living with rare diseases.” Amicus plans to further analyze and share the Phase 3 ESSENCE results with key stakeholders in the epidermolysis bullosa community including physicians, patient organizations and regulators. I the interim, in consultation with their physicians, participants in the ongoing extension studies (SD-004 and -006) will have the opportunity to continue being treated with SD-101. Based on these top-line data Amicus has no current plans to invest in any additional clinical studies or commercial preparation activities for SD-101. Conference Call and Webcast An audio webcast can also be accessed via the Investors section of the Amicus Therapeutics corporate web site at http://ir.amicusrx.com/, and will be archived for 30 days. Web participants are encouraged to go to the web site 15 minutes prior to the start of the call to register, download and install any necessary software. A telephonic replay of the call will be available for seven days beginning at 11:30 a.m. ET today. Access numbers for this replay are 855-859-2056 (U.S./Canada) and 404-537-3406 (international); participant code 85441850. About the ESSENCE Study About Epidermolysis Bullosa (EB) About Amicus Therapeutics Forward-Looking Statements 1Analyzed in all patients randomized (intent to treat (ITT)) using cox-proportional statistical model with baseline target wound size, target wound age, and EB type as covariates. 2Analyzed in ITT population using a logistic regression statistical model with baseline target wound size, target wound age, and EB type as covariates. CONTACTS: Investors/Media: Jennifer Paganelli FOLD–G |