Amryt Pharma PLC (NASDAQ:AMYT) (LON:AMYT) has hailed as a “significant milestone” the phase III success of FILSUVEZ, a gel for a rare and extremely painful skin condition called epidermolysis bullosa (EB).
Shares in the company soared almost 50% to a new high of 240p on Wednesday morning.
The study achieved its main goal, known in the scientific parlance as the primary endpoint, of accelerating the wound healing process
The next step is a rolling submission programme to the regulatory authorities, with the company applying for speedy approval in both the US and EU.
The company previously received fast track designation and rare paediatric disease designation from the US Food & Drug Administration (FDA), and it expects to be eligible to apply for a rare paediatric disease priority review voucher.
Submissions to the US and EU regulators are expected to be made late in the first quarter of next year.
The full set of data will be fully assessed and presented to an upcoming scientific symposium, Amryt said.
In all 223 people were enrolled into the phase III EASE trial, 156 of them children or infants. It was a double-blind study in which one group received the active treatment while the other didn’t. Only at the end did doctors know which group was which.
The Amryt team then compared total wound closure over 45 days of treatment across the two groups. The results were highly statistically significant.
In a statement, Amyrt chief executive, Dr Joe Wiley said: “We are proud to present these positive and encouraging results, demonstrating that FILSUVEZ could make an important difference to the lives of patients.
“We would like to extend our gratitude to all of the patients, their families, carers and physicians for their participation in the EASE trial and we look forward to working with regulatory authorities to make FILSUVEZ available as the first approved therapeutic treatment for EB patients. All of the team at Amryt are very excited by today's news and the impact this may have in our efforts to help patients with this very distressing condition," he added.
EB is a rare, chronic and distressing genetic skin disorder that causes the skin layers and internal body linings to separate.
Amryt estimates there are as many as 30,000 youngsters and adults affected by it in the US and there are 500,000 sufferers worldwide. The potential market for FILSUVEZ, therefore, is in excess of US$1bn.
House broker Shore Capital said if regulatory submissions are approved, Amryt is also eligible for a priority review voucher (PRV), which could be worth up to US$100mn and is not yet reflected in its forecasts and hence represents potential upside.
“We see this as a transformational milestone for Amryt and its management team, with the positive headline data a hugely de-risking event.”
The shares trade at a significant discount to fair value, the ShoreCap analysts said, representing less than 2x sales versus global peers on at least five times sales, and with “significant upside from EB and the dual-listing on Nasdaq as key drivers for a re-rating”.
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