Amryt Reports Positive Interim Analysis Data from Open Label Phase 3 Trial in EB

Rare Daily Staff

Rare disease focused biotech Amryt reported new positive clinical data from EASE, the largest phase 3 trial in Epidermolysis Bullosa, which were presented during a late-breaking oral presentation at the American Academy of Dermatology Annual Meeting 2022.

Epidermolysis Bullosa (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. 

The EASE trial is the largest ever global phase 3 trial conducted in patients with EB, performed across 58 sites in 28 countries. It comprises a 3-month double-blind randomized controlled phase followed by a 24-month open-label, single-arm phase. Patients with dystrophic and junctional EB target wounds of between 10 and 50cm2 in size that 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 wound dressings applied according to standard of care.  223 patients were enrolled into the trial including 156 pediatric patients.  Of those that completed the double-blind phase, all entered the open label safety follow up phase.

The 12-month interim analysis of the two-year open-label phase (OLP) (n=205) from the EASE trial evaluating the safety and efficacy of Oleogel-S10 in patients with EB found that the trial

met its primary endpoint in the double-blind phase (DBP; 90 days) with target wounds treated with Oleogel-S10 reaching first complete wound closure by Day 45 significantly more frequently than wounds treated with control gel.

Patients treated with Oleogel-S10 in the DBP (n=109) experienced a reduction in total body surface area percentage wounding (BSAP) from 12.1 percent at study entry in the DBP to 7.4 percent at the end of the DBP. In patients who continued Oleogel-S10 treatment in the OLP (100 of whom were on Oleogel-S10 in the DBP) and who had an OLP Month 12 assessment (n=56), BSAP was further reduced to 5.4 percent (equates to at least 15 months on Oleogel-S10 treatment). Patients treated with Oleogel-S10 in the DBP (n=109) experienced a reduction in EBDASI skin activity from 19.6 at study entry in the DBP to 16.5 at the end of the DBP. In the patients who continued Oleogel-S10 treatment in the OLP (100 of whom were on Oleogel-S10 in the DBP) and who had an OLP Month 12 assessment (n=55), EBDASI skin activity was further reduced to 15.2 (equates to at least 15 months on Oleogel-S10 treatment).

Treatment with Oleogel-S10 was well tolerated with continued use and no new safety signals were observed in the interim OLP analysis.

 “These important long-term interim safety and efficacy data demonstrate an average 55 percent reduction in the body surface area percentage with partial thickness wounds in those patients who continued on Oleogel-S10 for at least 15 months of treatment, which builds on the previously reported positive data from the DBP of EASE,” said Mark Sumeray, chief medical officer of Amryt Pharma. “We remain committed to our goal of delivering a clinically approved treatment to patients in need.”

Photo: Mark Sumeray, chief medical officer of Amryt Pharma