Amryt Pharma enrols first patient on EB drug trial

Irish company says therapy for skin blistering condition is its prime pipeline candidate

Amryt Pharma says it has made substantial progress since its admission to trading on AIM in April 2016. Photograph: iStock

Amryt Pharma says it has made substantial progress since its admission to trading on AIM in April 2016. Photograph: iStock


Specialty drug group Amryt Pharma told shareholders Thursday that it had made “substantial progress” since listing on the London’s junior AIM market last year.

In a trading statement released ahead of its annual general meeting, chairman Harry Stratford also gave an upbeat assessment of performance in the current year of the company which specialises in finding therapies for rare or “orphan” disease for which no medicine is available.

Amryt said it had enrolled the first patient in a phase III trial for its treatment of genetic skin disorder epidermolysis bullosa (EB), that affects an estimated one in 18,000 babies born in Ireland, according to the charity Debra Ireland, which provides support to families living with the condition.

More than 500,000 people worldwide suffer from the condition in which the skin blisters at the slightest touch for which there is currently no cure. EB is also known as “butterfly skin” in reference to the fragile nature of butterfly wings.

Global market

Amryt’s AP101 drug is the Irish company’s lead development asset and the phase III trial is one of the largest studies into EB medication.

The company says the global market for a treatment for EB is believed to be in excess of €1.3 billion.

The company also reiterated guidance that cholesterol drug Lojuxta will generate revenues of €10.5 million on an annualised basis.

Lojuxta is a treatment for an inherited cholesterol disorder called homozygous familial hypercholesterolaemia. Patients with familial hypercholesterolaemia have high levels of LDL, or “bad” cholesterol unrelated to lifestyle, which can lead to premature cardiovascular disease.

The condition affects as many as one in 250 people. Men with the condition have a 50 per cent higher chance of suffering a heart attack by the age of 50 if the condition is not treated. In women, the risk of a coronary heart attack by the age of 60 is 30 per cent higher, according to a 2003 study.

Homozygous familial hypercholesterolaemia is a particularly aggressive form of the condition which scientists now believe could affect as many as one in 160,000 people and can cause heart disease in teenagers and even younger children.