Successful phase III trial of Filsuvez to treat epidermolysis bullosa.- Amryt.

Amryt announces positive top line results from its pivotal Phase III EASE trial of Filsuvez (previously AP101 /Oleogel-S10) for the treatment of dystrophic and junctional Epidermolysis Bullosa ("EB"). The primary endpoint of the trial was met (p-value = 0.013). The EASE trial (NCT03068780) is the largest ever global Phase III trial conducted in patients with EB, performed across 58 sites in 28 countries. It comprises a 3 month double-blind randomised controlled phase followed by a 24 month open-label, single-arm phase. Patients with EB target wounds of between 10 and 50cm in size that were present for greater 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, 100% entered the open label safety follow up phase. The primary endpoint of the trial was to compare the efficacy of Filsuvez versus control gel according to the proportion of patients with complete closure of the target wound within 45 days of treatment. The primary endpoint was achieved with statistical significance (p-value = 0.013). This represents the first ever successful Phase III top line readout in EB. It is also the fourth time Filsuvez has demonstrated accelerated wound healing in a Phase III trial. While the key secondary endpoints did not achieve statistical significance, a number of favourable differences were observed. In addition, substantial further secondary endpoint data is expected and will be analysed over the coming weeks. The Company will now evaluate and analyse the full data set from the trial and will present results at an upcoming scientific symposium. Amryt intends to complete the submission of its rolling New Drug Application ("NDA") to the FDA and request priority review for Filsuvez. Filsuvez previously received Fast Track Designation and Rare Paediatric Disease Designation from the FDA. This means that if an NDA for Filsuvez is approved, the Company expects to be eligible to apply for a Rare Pediatric Disease Priority Review Voucher that can be used, sold or transferred. Amryt also intends to pursue an accelerated assessment in the EU. Regulatory submissions in the US and the EU are expected to be filed by late Q1 2021..