Amryt Pharma – Phase 3 Clinical Trial for AP101 in EB, First Site Initiated

28 March 2017

Amryt Pharma plc

Phase 3 Clinical Trial for AP101 in EB

First Site Initiated

Amryt, the pharmaceutical company focused on best-in-class treatments for rare and orphan diseases, is pleased to announce the commencement of “EASE”, its Phase 3 clinical trial of AP101, Amryt’s lead drug candidate, which offers a potential treatment for Epidermolysis Bullosa (“EB”). EB is a rare, genetic skin disorder, which causes exceptionally fragile skin.

The Company has initiated the first site for EASE in Sydney, Australia, and will randomise the first patient within the next few days. As previously reported, Amryt expects to conduct EASE in approximately 15 countries at over 30 sites to enrol a total of 164 evaluable patients. Patients will be randomised in a double-blind fashion to AP101 or placebo, and the proportion of patients with completely healed target wounds within 45 days will be evaluated as the primary efficacy endpoint.

EB is a chronic and debilitating condition that causes the skin to blister and tear at the slightest touch and for which there is currently no known cure. There are approximately 500,000 people living with EB worldwide and the global market for a treatment in EB is estimated to be in excess of EUR 1.3 billion.

Mark Sumeray, Chief Medical Officer of Amryt, commented:

“We are delighted to have initiated the first site participating in our Phase 3 clinical for AP101, which offers a potential treatment for the rare, genetic skin condition, Epidermolysis Bullosa or EB. Our study, EASE, is of substantial size for such a rare disease and offers the opportunity to evaluate a new topical treatment with the potential to accelerate wound healing in this devastating disorder.”


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About Amryt Pharma plc – see

Amryt Pharma is a specialty pharmaceutical company focused on developing and delivering innovative new treatments to help improve the lives of patients with rare or orphan diseases. The Company is building a diversified portfolio of commercially attractive, best-in-class, proprietary new drugs to help address some of these rare and debilitating illnesses for which there are currently no available treatments.

The Company recently acquired an exclusive licence to sell LOJUXTA (lomitapide), across the EU and other territories including the Middle East, North Africa, Turkey and Israel. LOJUXTA is used to treat a rare life-threatening disease called Homozygous Familial Hypercholesterolemia.

Amryt’s product, AP101 (Episalvan), received marketing approval for the treatment of partial-thickness wounds from the European Commission in January 2016. Amryt intends to develop AP101 (Episalvan) as a new treatment for Epidermolysis Bullosa (“EB”), a rare and distressing genetic skin disorder affecting young children for which there is currently no treatment. The Company recently received regulatory clarity from the FDA and the EMA regarding  its Phase 3 study of AP101 (Episalvan) in EB, which has been granted US and EU orphan drug designation, and is on track to commence this study towards the end of March 2017. The global market opportunity for EB is estimated to be in excess of EUR 1.3 billion.

Amryt’s earlier stage product AP102 is focused on developing novel, next generation somatostatin analogue (“SSA”) peptide medicines for patients with rare neuroendocrine diseases, where there is a high unmet medical need, including acromegaly and Cushing’s disease. AP102 was recently granted orphan designation in the US in acromegaly by the FDA.

The Company joined AIM and Dublin’s ESM in April 2016 following the reverse takeover of Fastnet Equity PLC.

This information is provided by RNS

The company news service from the London Stock Exchange