Amryt Pharma PLC (LON:AMYT) has said the read-out from its phase III study of its AP101 cream, a potential breakthrough treatment for a rare skin condition is likely late in the third quarter, or early in the fourth quarter of this year.
The timeline was provided in a statement in which Amryt said its Global EASE trial would be concluded slightly earlier than anticipated against the backdrop of the coronavirus (COVID-19) outbreak. Independent advice suggested the impact at this advanced stage of the recruitment process the would be “statistically negligible”.
The company’s AP101 cream is being developed for epidermolysis bullosa (EB), a chronic and distressing genetic skin disorder that causes the skin layers and internal body linings to separate.
Researchers are carrying out what’s called a double-blind assessment of the potential treatment, which means the experts won’t know who is receiving the AP101 and who has been given a placebo preparation until the end of the trial. The programme will close 90 days from the last patient being recruited.
Amryt chief executive, Dr Joe Wiley, said: “Operating clinical trials in rare diseases is always challenging and we are proud to have conducted the largest ever global study in EB.
“We are fortunate that our patient recruitment was already near conclusion before the onset of COVID-19, and having taken expert advice that the statistical impact of further patient recruitment would most likely be negligible, we have decided to close enrollment at this point."
“I would like to take this opportunity to thank all of our patients, their carers and physicians for their participation in and commitment to the EASE trial. We look forward to announcing top line data from this study in the coming months," he added.
AP101 has US Food & Drug Administration fast-track designation because it is designed to treat a serious disease for which there is an unmet medical need.
The incidence of EB is one in 20,000, which implies that there are as many as 30,000 people with it in the US and over 500,000 worldwide.