Amryt Pharma given go-ahead to continue Phase III EASE trial

Revenue-generating orphan drug company Amryt Pharma 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) on Friday.

The London-listed firm said the analysis was conducted by an independent data monitoring committee, which 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.

It said the analysis was conducted using unblinded efficacy data received by the IDMC for the primary endpoint from the first half of the study.

Amryt said it would begin the recruitment process for the additional patients required, and now expected a readout of the top line data from the EASE study in the second half of 2019.

Following the announcement, Amryt said it was now in a position to draw down the remaining €5m of its European Investment Bank facility.

“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,” said chief executive officer Joe Wiley.

“This brings us closer to potentially delivering a treatment for patients with EB.”

Wiley thanked 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.”