Amryt Pharma PLC (LON:AMYT) said it will begin phase III clinical trial on a drug for people a rare skin disorder following the completion of discussions with the US Food & Drug Administration and European Medicines Agency over the design of the study.
The company’s AP101 has been developed to treat Epidermolysis Bullosa (EB), which leaves suffers with exceptionally fragile skin.
INC Research has been employed to carry out the trials and has a choice of 30 sites in 15 countries that have been pre-qualified.
WATCH: CEO Wiley 'delighted' to be getting trial underway soon
A total of 164 patients will be treated for a 90 days. The proportion of patients with completely healed target wounds within 45 days will be evaluated as the main outcome of the study.
However researchers will also assess the time to achieve wound healing and changes in pain and itching.
Amryt has also agreed to conduct further non-clinical studies in parallel with this phase III trial.
"We are delighted to have reached this critical milestone in the development of Amryt,” said chief executive Joe Wiley.
“Reaching agreement with the regulatory authorities for our phase III study in EB was a precursor to commencing our pivotal study, which we now expect will enrol its first patients in the coming weeks.
“Significantly, we now have agreement from the regulatory agencies in both the US and in Europe for a single phase III study, which we hope in due course will lead to approval in EB.
“There remains substantial unmet need for drugs which can address the symptoms and we are delighted to be progressing our potential treatment to this pivotal phase."
There are an estimate 500,000 people living with EB worldwide, with 35,000 in Europe and 30,000 in the US. The global market is estimated to be worth in the order of €1.3bn a year.
In December, Amryt revealed it had secured a €20mln European Investment Bank facility that will help phase III trial of AP10, also known as Episalvan.
At 12.50pm, the shares were changing hands for 19.5p each, up 7%.
"We see encouraging momentum in the business and have confidence in management's ability to deliver on its strategic priorities as set out at the IPO in April 2016," said City broker Shore Capital.
"We look to the imminent start of the pivotal phase III trial for the lead asset Episalvan in EB in the next few weeks as the next key catalyst."
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