Amryt, the pharmaceutical company focused on treatments for rare and orphan diseases, has enroled first patient into “EASE”, the company’s pivotal Phase 3 clinical trial, which is evaluating AP101 as a potential treatment for the rare, genetic skin disorder, Epidermolysis Bullosa (“EB”).
The study will include 164 patients across approximately 30 sites in 15 countries, with the first site initiated in Sydney, Australia, the company said. Additionaly, patients will be randomised in a double-blind fashion to AP101 or placebo, with the treatment applied as a topical gel when dressing changes would routinely occur but no less frequently than every four days.
The company said that the proportion of patients with completely healed target wounds within 45 days will be evaluated as the primary efficacy endpoint. The trial is being conducted by INC Research as the contract research organisation. An interim analysis will be conducted when 50% of the study patients have completed 45 days of treatment. The results from this interim analysis of this trial are expected in the first quarter of 2018.
EB is a chronic and debilitating disease which causes the skin to tear and blister at the slightest touch. There are approximately 500,000 people living with EB worldwide and the global market for a treatment in EB is estimated to be in excess of EUR 1.3 billion. Currently, there is no approved therapy to treat EB, Amryt noted.
Mark Sumeray, Chief Medical Officer of Amryt, commented: “The trial will evaluate the efficacy and safety of our lead drug candidate, AP101, as a potential treatment for Epidermolysis Bullosa, a distressing and rare skin disorder. We look forward to further patients joining the study, which we expect to be one of the largest studies of its kind in this rare disease.”