Amicus announces planned analysis of ESSENCE trial primary endpoints
Amicus Therapeutics announced it 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. 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. FDA, 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, 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 less than or equal to 0.05. Amicus said it is on track to report top-line data from the ESSENCE study during the third quarter of 2017. To date, more than 95% of patients completing the 3-month primary treatment period have elected to continue in the open-label extension study. Jay Barth, Chief Medical Officer of Amicus, said, "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."