Amryt has announced the results of an unblinded interim efficacy analysis on its pivotal Phase III EASE trial for AP101 as a potential treatment for Epidermolysis Bullosa (EB).
The analysis was conducted by an Independent Data Monitoring Committee (IDMC). The IDMC recommended that the trial should continue with an increase of 48 patients in the study to a total of 230 evaluable patients, in order to achieve 80% statistical power.
The analysis was conducted using unblinded efficacy data received by the IDMC for the primary endpoint from the first half of the study. Amryt 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 H2 2019.
Following today’s announcement, Amryt is now in a position to draw down the remaining €5m of its European Investment Bank (EIB) facility.
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. The EASE trial is the largest ever global Phase III study conducted in patients with EB and follows favourable data from a Phase IIa study in EB.
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. EB is chronic, potentially disfiguring and life limiting. There are approximately 500,000 people living with EB worldwide and there are currently no approved treatments. It is estimated that the annual market potential for AP101 is more than €1 billion.
Joe Wiley, CEO of Amryt Pharma, said: “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. Today’s news is a significant milestone for Amryt and our shareholders in building towards our vision of becoming a global leader in rare and orphan diseases.”
Dr Helen Philips, Global Head of Medical Affairs for Amryt Pharma, said: “A cure is definitely something to aspire to achieving but short-term solutions are a priority. The IDMC’s recommendation to continue the global EASE trial is positive news for people living with the condition as it brings us closer to potentially delivering the first ever approved treatment for patients with EB.”
AP101 is Amryt’s lead development asset in its EB franchise which also includes AP103, a novel topical gene therapy, initially being developed in Recessive Dystrophic EB (RDEB). The company also announced positive results from two pre-clinical studies which support the development of AP103, as a potentially disease-modifying therapy for patients with
Recessive Dystrophic Epidermolysis Bullosa (RDEB), a particularly severe form of EB.