Phase 3 Results of Epidermolysis Bullosa Trial Published in British Journal of Dermatology

Amryt Pharma’s phase 3 trial, EASE, is the largest phase 3 trial in epidermolysis bullosa.

The British Journal of Dermatology published phase 3 trial results from Amryt Pharma's EASE (NCT03068780) trial of Oleogel-S10 gel for the treatment of epidermolysis bullosa (EB).1 The EASE trial met its primary endpoint of complete target wound closure achieved in 41.3% of target wounds treated with Oleogel-S10 compared to control gel, demonstrating a 44% increase in the probability of wound closure with Oleogel-S10. 

Currently, Oleogel-S10 is approved in the European Union and Great Britain for the treatment of partial thickness wounds associated with junctional and dystrophic EB in patients aged 6 months and older.

EASE is the largest phase 3, randomized, controlled study evaluating treatment options for EB. The trial was performed across 58 sites in 28 countries and was comprised of a 3-month double-blind, randomized, controlled phase followed by a 24-month open-label, single-arm phase.

Patients with dystrophic EB and junctional EB target wounds of between 10 and 50cm in size who were present for more than 21 days and less than 9 months were randomized in the double-blind phase to study treatment in a 1:1 ratio and had wound dressings applied according to the standard of care. The trial enrolled 223 patients, including 156 pediatric patients. Out of the patients who completed the double-blind phase, 100% entered the 24-month open-label follow-up phase.

Key trial endpoints include:

  • A reduction in overall wound burden using two different measures: Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI) and Body Surface Area Percentage (BSAP)
  • Using EBDASI, the mean change from baseline in the skin activity score exceeded the clinically important threshold of a 3-point reduction for patients treated with Oleogel-S10
  • Using BSAP, the mean change in total BSAP showed greater reduction at day 60 and day 90 for Oleogel-S10 compared to the control gel
  • Patients treated with Oleogel-S10 had a reduced requirement for daily dressing changes vs control gel
  • Oleogel-S10 significantly improved pain associated with dressing changes at day 14 and remained numerically lower throughout the 90-day treatment period of the trial
  • Oleogel-S10 was well tolerated and had a similar safety profile to the control gel

According to Amryt, “EB is a rare and devastating group of hereditary disorders of the skin, mucous membranes, and internal epithelial linings characterized by extreme skin fragility and blister development. Patients with severe forms of EB suffer from severe, chronic blistering, ulceration, and scarring of the skin, mutilating scarring of the hands and feet, joint contractures, strictures of the esophagus and mucous membranes, a high risk of developing aggressive squamous cell carcinomas, infections and risk of premature death.” 2

References

  1. Johannes S Kern, Eli Sprecher, Maria Florencia Fernandez, Franziska Schauer, Christine Bodemer, Tracy Cunningham, Sandra Löwe, Charles Davis, Mark Sumeray, Anna L Bruckner, Dedee F Murrell, for the EASE investigators, Efficacy and safety of Oleogel-S10 (birch triterpenes) for epidermolysis bullosa – results from the phase 3, randomised, double-blind phase of the ‘EASE’ study, British Journal of Dermatology, 2022;, ljac001, https://doi.org/10.1093/bjd/ljac001
  2. The British Journal of Dermatology published results from Amryt’s EASE phase 3 trial in epidermolysis bullosa. Amryt Pharma. Published October 25, 2022. Accessed October 26, 2022. https://otp.tools.investis.com/clients/uk/amryt_pharmaceuticals_dac1/usn/usnews-story.aspx?cid=1375&newsid=86200