EASE Study Enrollment Closed; Top Line Data Expected in Late Q3/Early Q4 2020

Amryt, a global, commercial-stage biopharmaceutical company dedicated to commercializing and developing novel therapeutics to treat patients suffering from serious and life-threatening rare diseases, today provides the following update with regard to its Global Phase 3 clinical study (“EASE”) in Epidermolysis Bullosa (“EB”).

EASE Trial Update:

In recent weeks, the COVID-19  pandemic has had a material impact on clinical trials globally, including patient recruitment. Given that the EASE study was already close to full enrollment, Amryt has taken advice from an independent expert and concluded that the statistical impact of further patient recruitment would most likely be negligible.  Amryt has therefore decided to close the EASE study to further enrollment.

Shortly after the last patient completes the end of the double-blind treatment period (Day 90), the study data will be cleaned and the database locked.  Statistical analyses will then be performed and the Company now anticipates top line data read out in late Q3 / early Q4 2020.

Dr Joe Wiley, CEO of Amryt Pharma, commented: “We are pleased to have completed recruitment into our EASE study. Operating clinical trials in rare diseases is always challenging and we are proud to have conducted the largest ever global study in EB. We are fortunate that our patient recruitment was already near conclusion before the onset of COVID-19, and having taken expert advice that the statistical impact of further patient recruitment would most likely be negligible, we have decided to close enrollment at this point.

I would like to take this opportunity to thank all of our patients, their carers and physicians for their participation in and commitment to the EASE trial. We look forward to announcing top line data from this study in the coming months.”

About AP101 and EASE

AP101 is a potential treatment for EB, a rare, chronic and distressing genetic skin disorder that causes the skin layers and internal body linings to separate, affecting infants, children and adults, for which there is currently no approved treatment.  It is estimated that the incidence of EB is approximately one in 20,000, which implies that there are as many as 30,000 affected individuals in the US and over 500,000 worldwide.

In March 2017 Amryt commenced EASE, a Phase 3 prospective double-blind randomised placebo controlled efficacy and safety study of AP101 in patients with EB. EASE is the largest ever global Phase 3 study conducted in patients with EB, operating across 55 sites in 27 countries globally.  In January 2019, Amryt reported the outcome of an unblinded interim efficacy analysis, at which point an Independent Data Monitoring Committee (“IDMC”) recommended continuing the trial.

In July 2018, AP101 was granted Rare Pediatric Disease designation by the U.S. Food and Drug Administration (“FDA”). This means that if a New Drug Application (“NDA”) for AP101 is approved, the Directors expect Amryt to be eligible to receive a Rare Pediatric Disease Priority Review Voucher (“PRV”) that can be used, sold or transferred.

AP101 was granted Fast Track designation by the FDA in September 2019. The Fast Track programme is designed to accelerate the development and review of products such as AP101, which are intended to treat serious diseases and for which there is an unmet medical need.  Fast Track designation enables more frequent communication with the FDA.  Amryt intends to request and submit a rolling-NDA in the coming months to the FDA and will request a priority review in the US.  Amryt also intends to pursue an accelerated assessment in the EU.

AP101 has been granted Orphan Drug status for the treatment of EB in the EU and the US. Should AP101 be granted approval it should be entitled to Orphan Drug exclusivity for the treatment of EB, extending seven years in the US and ten years in the EU from the date of approval in the respective jurisdictions.

Amryt’s next scheduled market update will be the release of FY 2019 Audited Results and Q1 Trading Update in early May.