Oral Presentation Australasian Society for Dermatological Research Annual Scientific Meeting 2017

Baseline characteristics of patients from the phase 3, double-blind, vehicle-controlled trial of the topical investigational drug SD-101 in patients with epidermolysis bullosa (ESSENCE study) (#32)

Dedee Murrell 1 , Christine Bodemer 2 , Raúl de Lucas Laguna 3 , Susanna Esposito 4 , Dimitra Kiritsi 5 , Franziska Schauer 5 , John Browning 6 , Amy Paller 7 , Hjalmar Lagast 8 , Allen Reha 8 , Jamie Gault 9 , Willistine Lenon 9 , Lindsay Reklis 9 , Richard Lazauskas 8 , Ronald V. Nardi 9 , Jay A. Barth 8 , , and the SD-101 ESSENCE Study Group
  1. St George Hospital, UNSW, Sydney, NSW, Australia
  2. The University Hospital Necker Enfants Malades, Paris, France
  3. Department of Pediatric Dermatology, University Hospital La Paz, Madrid, Spain
  4. Universita Degli Studi di Milano, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy
  5. Department of Dermatology, Medical Center- University of Freiburg, Freiburg, Germany
  6. Texas Dermatology and Laser Specialists, San Antonio, TX, USA
  7. Northwestern University Feinberg Medical School, Chicago, IL, USA
  8. Amicus Therapeutics, Inc., Cranbury, NJ, USA
  9. Scioderm - An Amicus Therapeutics Company, Durham, NC, USA

Epidermolysis bullosa (EB) is a rare genetic disorder that manifests as blistering of the skin, mucosa, and epithelial lining of organs. EB is chronic, potentially disfiguring, and, in some cases, fatal. Currently, there are no approved treatments for EB. Here, we report the baseline characteristics of patients with EB enrolled in the ESSENCE trial. ESSENCE (SD-005; NCT02384460) is an ongoing phase 3, global, multicenter, randomized, double-blind, vehicle-controlled study assessing the efficacy and safety of SD-101 (topical allantoin cream) in patients with EB (simplex, recessive dystrophic, junctional non-Herlitz). Patients were randomly assigned 1:1 to receive SD-101 6% allantoin cream or vehicle (0% allantoin) applied topically once daily to the entire body for 90 days. Eligible patients were aged ≥1 month and had a target wound size ranging from 10-50 cm2 that was present for ≥21 days. Patients completing ESSENCE were eligible to enter a separate open-label study (SD-006; NCT02670330) to assess the long-term efficacy and safety of SD-101. As of February 21, 2017, 126 patients were enrolled (target: ≈150). Proportions of randomized patients by EB subtype were: 11.1% with simplex; 67.5% with recessive dystrophic; and 21.4% with junctional non-Herlitz. Demographics were as follows: 53.2% female; 78.6% white; 5.6% African American; and 5.6% Asian. Mean (standard deviation [SD]) age was 15.1 (14.3) years (range, 0-67 years) and mean body mass index was 17.4 (4.4) (range, 9.9-31.3). Patients had a mean (SD) body surface area index (BSAi) for lesional skin of 24% (19%) and a BSAi for total body wound burden of 10.8% (11%). This study includes a broad representation of EB subtypes and is one of the largest clinical trials of an investigational drug in EB to date. Data for the primary efficacy outcome measures, time to wound closure and proportion achieving wound closure after 3 months of treatment, are expected mid-2017.

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