Study Did Not Meet Primary Endpoints
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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).
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. In 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.
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About the ESSENCE Study
ESSENCE (SD-005) is a randomized, multicenter, double-blind, placebo-controlled Phase 3 study in patients with documented diagnosis of Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB. For study entry, target wounds were required to be chronic (at least 21 days old) and between 10 and 50 cm2 in size. A total of 169 patients were randomized on a 1:1 basis to SD-101 treatment (n=82) or placebo (n=87) for a three-month primary treatment period followed by an ongoing open-label extension study (SD-006) in which all patients receive SD-101 treatment.
About Epidermolysis Bullosa (EB)
Epidermolysis bullosa is a rare, devastating genetic disorder that leads to severe skin blistering and open wounds often beginning at birth. There are no approved therapies to treat epidermolysis bullosa, which affects tens of thousands of children and adults throughout their lifetimes. EB is chronic, potentially disfiguring, and in some cases fatal. 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.
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to the clinical results from, and our future development plans with respect to, our product candidate, SD-101 for Epidermolysis Bullosa. The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved or will not change. Any or all of the forward-looking statements in this press release can be affected by assumptions we might make based on information available at this time, including our evaluation of the Phase 3 clinical results, or by other known or unknown risks and uncertainties. In addition, all forward-looking statements are subject to other risks and factors detailed in our Annual Report on Form 10-K for the year ended
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.
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