Amryt Pharma plc: Update on AP101 EASE Phase III trial

Amryt  a revenue generating orphan drug company focused on acquiring, developing & commercialising products that help to improve the lives of patients where there is a high unmet medical need, today announces an update on its pivotal Phase III EASE trial for AP101 as a potential treatment for Epidermolysis Bullosa (“EB”).

Following an assessment by the trial’s Independent Data Monitoring Committee (“IDMC”), the Company can now enrol infants and children with EB between the ages of 21 days to 4 years of age in to the trial. The IDMC’s analysis was conducted using pharmacokinetic (“PK”) data received from patients already enrolled in the trial (aged four years and older). This follows the announcement of the unblinded interim efficacy analysis in January.

Amryt will begin the recruitment process for infants and children into EASE immediately. The EASE trial is the largest ever global Phase III study conducted in patients with EB and Amryt expects top-line data read out in H2 2019.

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.1  It is estimated that the market potential for AP101 is more than €1 billion.

Joe Wiley, CEO of Amryt Pharma, commented: “Extending the age eligibility criteria for this trial is positive news and a significant step forward in addressing the unmet need in this devastating condition, which is present from birth in most cases. This development represents another milestone for Amryt as we build on our vision of becoming a global leader in rare and orphan diseases.”


Amryt Pharma plc


+353 (1) 518 0200


Joe Wiley, CEO

Rory Nealon, CFO/COO


Shore Capital


+44 (0) 20 7408 4090


NOMAD and Joint Broker


Edward Mansfield, Mark Percy, Daniel Bush




+44 (0) 20 7710 7600


Joint Broker


Jonathan Senior, Ben Maddison




+353 (1) 679 6363


ESM Adviser and Joint Broker


John Frain, Anthony Farrell


Consilium Strategic Communications


+44 (0) 20 3709 5700


Matthew Neal, David Daley, Nicholas Brown



About Amryt

Amryt is a biopharmaceutical company focused on developing and delivering innovative new treatments to help improve the lives of patients with rare or orphan diseases.  

Lojuxta is an approved treatment for adult patients with the rare cholesterol disorder - Homozygous Familial Hypercholesterolaemia ("HoFH"). This disorder impairs the body's ability to remove low density lipoprotein ("LDL") cholesterol ("bad" cholesterol) from the blood, typically leading to abnormally high blood LDL cholesterol levels in the body from before birth - often ten times more than people without HoFH - and subsequent aggressive and premature narrowing and blocking of blood vessels, heart attacks and strokes, even at a very young age if not properly diagnosed or receiving adequate treatment.  Lojuxta is indicated as an adjunct to a low-fat diet and other lipid-lowering medicinal products with or without LDL apheresis in adult patients with HoFH.

Amryt is the marketing authorisation holder and has an exclusive licence to sell Lojuxta (lomitapide) across the European Economic Area, Middle East and North Africa, Switzerland, Turkey, Israel, Russia, the Commonwealth of Independent States and the non-EU Balkan states.

Amryt's lead drug candidate, AP101, is a potential treatment for Epidermolysis Bullosa ("EB"), a rare and distressing genetic skin disorder affecting young children and adults for which there is currently no treatment.  It is currently in Phase III clinical trials. The European and US market opportunity for EB is estimated to be in excess of €1 billion.

In March 2018, Amryt in-licenced a pre-clinical gene-therapy platform technology, AP103, which offers a potential treatment for patients with Recessive Dystrophic Epidermolysis Bullosa, a subset of EB, and is also potentially relevant to other genetic disorders.

For more information on Amryt, please visit

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