HealthPharma & Biotech
Amryt Pharma PLC

Amryt Pharma adds new element to promising portfolio

“If successful, this platform [AP103] has potential in other genetic skin conditions and beyond”

EB often occurs in children


  • Junior with one drug on the market and second in phase III trial

  • Cholesterol treatment Lojuxta on sale in five major European countries

  • AP101 in phase III trial for Epidermolysis Bullosa (EB), a condition which occurs prominently in children and makes skin fragile to even the faintest touch.

  • Independent committee recommends trial continues with modest increaser in number of patients

  • No other available treatment in EB market worth US$1bn


How is it doing

AP101 is a skin disorder treatment for epidermolysis bullosa, a rare, inherited condition which occurs predominantly in children and makes skin fragile to even the faintest touch.

It is chronic, potentially disfiguring and in some cases fatal. There are around 500,000 people living with EB worldwide and there are currently no approved treatments.

After analysing interim efficacy data, the Independent Data Monitoring Committee (IDMC) has recommended Amryt continues the phase III study but with the addition of 48 new patients. That will take the total up to 230 and the larger pool should yield better, more reliable data further down the line.

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.

Amryt has exclusive licences for the European Economic Area, Middle East and North Africa, Switzerland, Turkey, Israel, Russia, the Commonwealth of Independent States and the non-EU Balkan states.

Lojuxta’s revenues for the six months to June amounted to €6.6mln or a 14.6% increase from the same period in 2017. 

Group revenues rose by 13.6% to €7mln from €6.2mln a year earlier.

Losses in half-year were €11.4mln while cash holdings were €12.2mln.


In January, Amryt Pharma PLC (LON:AMYT) said a pre-clinical study of a cream for a severe and very difficult to treat skin condition has delivered encouraging results.

AP103 is a putative treatment for recessive dystrophic epidermolysis bullosa (RDEB), which is a subset of epidermolysis bullosa (EB), a condition that means the skin is so fragile it breaks at the lightest contact.

Researchers carried out in vitro, or lab tests as well as applying AP103 onto a 3-D matrix of human RDEB skin.

The headline results revealed the cream restored the production of collagen VII to levels exceeding those created by healthy people.

Collagen VII is critical in the wound healing process and scientists believe it could have a central role to play in tackling REDB.

AP103 has been awarded grant funding totalling €8.4m over three years by the Irish government.


What the boss says: Joe Wiley

"Initiation of the first trial site in the US allows us to further accelerate patient enrolment for EASE and crucially, means that eligible American sufferers of this rare condition will be able to take part in this potentially life-changing trial.”



Blue Sky

AP103, developed via a gene delivery system in-licensed by Amryt last March, showed no cellular toxicity after repeated administration.

“If successful, this platform has potential in other genetic skin conditions and beyond,” said chief executive Joe Wiley.

“Unlike other gene therapies that rely on viral vectors, AP103 is a novel synthetic delivery technology, which provides a potential competitive advantage in the gene therapy field.”

What the brokers say: Shore Capital

“Today’s announcement is further momentum for Amryt following last week’s (4th January) significant announcement that an independent data monitoring committee (IDMC) had recommended the Phase III EASE study of AP101 in EB should continue with only a modest (48) increase in patients.

“The announcement was highly encouraging in our view, as it suggests AP101 is showing signs of efficacy and was an unblinded analysis on the primary efficacy outcome of the trial.

"Significantly, there are no approved treatments for EB in an annual market that could be worth over €1bn.”

At 16.4p Amryt is worth £45mln.

Quick facts: Amryt Pharma PLC

Price: £0.12

Market: AIM
Market Cap: £5.67 m


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