Joseph Wiley, chief executive elect of Amryt Pharmaceuticals, said it was a “nice endorsement” of the company’s strategy to have Harry Stratford on board as chairman.
Amryt intends to focus on acquiring, in-licensing and then developing drugs for hard to treat, so-called orphan diseases.
Stratford pioneered this approach as founder of Shire, the world’s largest speciality pharma company.
“Having Harry on board is fantastic,” Wiley told Proactive.
“It is a nice endorsement of what we want to do, given he founded what is now the largest ‘orphans’ company in the world.”
Key to the short- and medium-term prospects of the firm is a skin treatment called Episalvan, developed by German specialist Birken - one of two acquisitions Amryt has made.
The product is already out on the market and has been signed off for use in adults in a type of gash or ulcer called a partial thickness wound.
However, the plan is to carry out a phase-III clinical trial to assess its potential to accelerate the healing process in epidermolysis bullosa (EB).
This is a rare and distressing genetic disorder affecting young children that makes the skin very fragile.
“It is a pretty horrific disease,” said Wiley. “In the severe forms the parents change the wound dressings every three to four days.”
There is a competing treatment currently undergoing phase III trials called Zorblisa.
It was discovered by Scioderm, which was then acquired by Amicus Therapeutics for up to US$847mln on the strength of phase IIb results.
Wiley is unfazed by having a rival in this space.
He sees Zorblisa as more of a pathfinder than competition and said: “The good part of Amicus being ahead of us is they have shown us the regulatory path to approval. We will follow similar protocol.”
Also, he hopes Episalvan will have a major advantage over Zorblisa in that it can be applied once every three to four days rather than every 24 hours.
The early data from a phase IIa study on EB and phase III trials in other indications for the skin treatment in adults are encouraging.
Wiley said the phase III investigation ought to get underway late this year or early next, which means Amryt will have the results some time in 2018.
Episalvan has been awarded orphan drug designation (ODD) in the US and the EU for EB, and the market for this indication alone is estimated to be US$1.5bn.
Orphan designation often means the product can be fast-tracked – usually at a fraction of the cost of developing medication destined for the mass market.
And, as they are niche areas where there are no current treatments, firms are often allowed to charge more for a breakthrough new product.
Around 19% of all prescriptions are for these rare ailments, and the market globally is estimated to be worth US$176bn.