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, 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.”
The revenue-generating drug developer is currently in Phase III clinical trials with a cream called AP101 for EB.
On Friday, it said it had been asked to add a further 48 patients to its study, suggesting the treatment is providing some therapeutic effect.
In a note to clients, analysts at ‘house’ broker Shore Capital commented: “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.”
In afternoon trading, Amryt Pharma shares held steady at 17.10p.
-- Adds analyst comment, share price --