An Independent Data Monitoring Committee (IDCM) has recommended that Amryt’s pivotal Phase III EASE trial for AP101 - a potential treatment for the currently incurable Epidermolysis Bullosa (EB) - should continue.
The IDMC recommended that the trial should continue with an increase of 48 patients in the study, creating a total of 230 evaluable patients, in order to achieve 80% statistical power.
The news is a significant milestone for Amryt, who will begin the recruitment process for the additional patients required and now expects a readout of the top line data from the EASE study in the second half of 2019.
The EASE trial is the largest ever global study to investigate a treatment for EB, and the continuation means there fortunately there is real hope that the results show that the treatment is efficacious and ultimately works.
EB is a devastating, rare genetic skin disorder that can cause skin to blister and tear from the slightest friction or trauma and can, in some cases, cause blistering and erosion of the epithelial lining of internal organs.
There are approximately 500,000 people living with EB worldwide, 5,000 in the UK, with around 2,000 being managed in specialist centres, with no existing cure for the condition.
The US Food and Drug Administration (FDA) granted Investigational New Drug (IND) clearance for AP101 in September 2018 and US trial sites, alongside other international sites, are expected to contribute to the additional patient recruitment required.
Joe Wiley, CEO of Amryt Pharma, commented: “The IDMC’s recommendation is good news for Amryt as it allows us to continue the trial with only a modest increase in the size of the study. This brings us closer to potentially delivering a treatment for patients with EB. We would like to thank sincerely the patients, families and clinical investigators involved in the EASE trial to date.”