Amryt Pharma reports ‘very positive’ results in largest EB clinical trial
(AIM: AMYT ) has reported “very positive” results from its Phase 3 EASE trial, the largest international clinical trial conducted in patients with Epidermolysis Bullosa (EB).
The biopharmaceutical company said the positive outcome of the trial marks “a very important milestone” as it seeks approval for FILSUVEZ®, a potential treatment for EB, a group of rare genetic conditions for which there is currently no approved treatment.
If FILSUVEZ®, formerly known as AP101 /Oleogel-S10 is approved, Amryt said it will leverage its existing global infrastructure to commercialise the product as a treatment for EB.
The Phase 3 EASE trial is the largest ever global Phase 3 trial conducted in patients with EB and was performed across 58 sites in 28 countries and comprised a three-month double-blind randomised controlled phase followed by a 24 month open-label, single-arm phase.
223 patients were enrolled into the trial including 156 pediatric patients. Of those that completed the double-blind phase, 100% entered the open label safety follow up phase.
Shares in Amryt Pharma have jumped over 50% since July and were trading 13.44% higher this morning at 181.5p following the announcement.
The primary endpoint of the trial, which was achieved ‘with statistical significance’, was to compare the efficacy of FILSUVEZ® versus control gel according to the proportion of patients with complete closure of the target wound within 45 days of treatment.
Amryt said this represents the first ever successful Phase 3 top line readout in EB. It is also the fourth time FILSUVEZ® has demonstrated accelerated wound healing in a Phase 3 trial.
While the key secondary endpoints did not achieve statistical significance, a number of favourable differences were observed, and substantial secondary endpoint data is expected.
“We are proud to present these positive and encouraging results, demonstrating that FILSUVEZ® could make an important difference to the lives of patients,” said CEO, Dr Joe Wiley.
Amryt intends to complete the submission of its rolling New Drug Application ("NDA") to the US Food and Drug Administration ("FDA") and request priority review for FILSUVEZ®.
If an NDA is approved, Amryt will be eligible to apply for a Rare Pediatric Disease Priority Review Voucher that can be used, sold or transferred because it already possesses Fast Track Designation and Rare Paediatric Disease Designation from the FDA for FILSUVEZ®.
The group said it also intends to pursue an accelerated assessment in the EU. Meanwhile, regulatory submissions in the US and EU are expected to be filed by late Q1 2021.
The global market opportunity for EB is estimated by Amryt to be in excess of $1.0 billion.
“The proven ability to address the hallmark manifestation of this devastating disease, chronic wounds that don't heal, in such an efficacious manner and as a therapy applied as part of the standard of care will undoubtedly lead to a meaningful quality of life improvement for patients living with the "worst disease you've never heard of,” said Brett Kopelan, Executive Director of debra of America and President of Debra International.
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