Amryt Pharma – Phase 3 Clinical Trial for AP101 in EB. First Patient Enrolled

24 April 2017

Amryt Pharma plc

 Phase 3 Clinical Trial for AP101 in EB

First Patient Enrolled


Amryt, the pharmaceutical company focused on best-in-class treatments for rare and orphan diseases, is pleased to announce that the first patient has now been enrolled into “EASE”, the Company’s pivotal Phase 3 clinical trial, which is evaluating AP101 as a potential treatment for the rare, genetic skin disorder, Epidermolysis Bullosa (“EB”).

As previously reported, EASE intends to study 164 patients across approximately 30 sites in 15 countries, with the first site initiated in Sydney, Australia. Patients will be randomised in a double-blind fashion to AP101 or placebo, with the treatment applied as a topical gel when dressing changes would routinely occur but no less frequently than every four days.

The proportion of patients with completely healed target wounds within 45 days will be evaluated as the primary efficacy endpoint. The trial is being conducted by INC Research as the contract research organisation. An interim analysis will be conducted when 50% of the study patients have completed 45 days of treatment. The results from this interim analysis of this trial are expected in the first quarter of 2018.

EB is a chronic and debilitating disease which causes the skin to tear and blister at the slightest touch. 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. Currently, there is no approved therapy to treat EB.

Mark Sumeray, Chief Medical Officer of Amryt, commented:

“We are delighted to enrol the first patient in EASE, our pivotal Phase 3 clinical trial. The trial will evaluate the efficacy and safety of our lead drug candidate, AP101, as a potential treatment for Epidermolysis Bullosa, a distressing and rare skin disorder. We look forward to further patients joining the study, which we expect to be one of the largest studies of its kind in this rare disease.”


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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 holds an exclusive licence to sell LOJUXTA (lomitapide) for adults, across the EU and other territories including the Middle East, North Africa, Turkey and Israel. LOJUXTA is used to treat a rare life-threatening, genetic disorder called Homozygous Familial Hypercholesterolemia, which impairs the body’s ability to remove LDL cholesterol (“bad” cholesterol) from the blood. This typically results in extremely high blood LDL cholesterol levels leading to aggressive and premature narrowing and blocking of arterial blood vessels.  If left untreated, heart attack or sudden death may occur in childhood or early adulthood.

Amryt’s lead drug candidate, AP101 (Episalvan), is a potential treatment for Epidermolysis Bullosa (“EB”), a rare and distressing genetic skin disorder for which there is currently no treatment. It is currently in Phase 3 clinical trials. 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.

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

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