BG04 Oleogel-S10 (birch triterpenes) in the treatment of epidermolysis bullosa wounds: 24-month efficacy and safety data from the EASE study

Abstract

Abstract Epidermolysis bullosa (EB) is a rare debilitating disorder characterized by skin fragility, blister formation and impaired wound healing. The abnormal wound healing seen in EB is a key driver for systemic disease and can lead to serious clinical complications, deformities and symptoms with a devastating impact on quality of life (QoL). The 90-day double-blind phase (DBP) of the phase III EASE study previously demonstrated accelerated wound healing for oleogel-S10 (birch triterpenes) vs. control gel in EB. Here, we report the safety and total wound burden results from the 24-month open-label phase (OLP), in which all patients received treatment with oleogel-S10. EB Disease Activity and Scarring Index (EBDASI) and body surface area percentage (BSAP) data are reported without visit windows to reflect a real-world situation more accurately, particularly considering the COVID-19 pandemic. The patient population was made up of those with dystrophic EB (n = 178; 86.8%) and junctional EB (n = 25; 12.2%). Further baseline characteristics are available in Table 1. In total, 141 patients (68.8%) completed the OLP. The mean (SD) treatment duration for all patients was 584.7 (246.1) days. Adverse events were reported in 77.1% of all patients in the OLP vs. 81.7% of those receiving oleogel-S10 in the DBP. Mean BSAP for patients treated with oleogel-S10 in the DBP reduced from 12.1% at study entry to 6.1% on completion of the EASE study. Similarly, the mean EBDASI skin activity score in the oleogel-S10 group improved from 19.6 to 15.1 at the end of the OLP. In addition, reductions in both BSAP and EBDASI from OLP baseline were observed in patients who transitioned from control gel to oleogel-S10 in the OLP. Oleogel-S10 has a reassuring long-term safety profile. Furthermore, the long-term impact of accelerated wound healing is evident through the sustained reduction in wound burden over time. This is encouraging given the regular reoccurrence of patients’ fragile wounds in the natural course of this chronic disease. Table 1Baseline characteristics