Shares of Amryt Pharma were up nearly 15% in premarket trading after the company released positive topline results from its Phase III trial assessing Filsuvez as a treatment for dystrophic and junctional Epidermolysis Bullosa (EB).
This morning, Dublin-based Amryt said the primary endpoint of the late-stage study was met. The Phase III study, the largest in Epidermolysis Bullosa, compared Filsuvez, formerly known as AP101, against a control gel according to the proportion of patients with complete closure of the target wound within 45 days of treatment. Amryt said the statistically significant results of the study marks the first successful Phase III top-line readout in this indication. It is also the fourth time Filsuvez has demonstrated accelerated wound healing in a Phase III trial, the company added.
Amryt’s lead development candidate, Filsuvez is a potential treatment for the cutaneous manifestations of EB, a rare and distressing genetic skin disorder affecting young children and adults for which there is currently no approved treatment. Filsuvez has been granted Rare Pediatric Disease Designation and has also received a Fast Track Designation from the
“It is very gratifying to see the first results from the trial provide evidence of the effect of FILSUVEZ® on the speed of wound healing in such a complex clinical situation. We look forward to further data over the next few weeks,” Mark Sumeray, Amryt’s chief medical officer said in a statement.
Amryt Chief Executive Officer Joe Wiley said the Phase III readout represents an important advancement for patients and families living with EB, a genetic skin disorder that causes the skin layers and internal body linings to separate. It is estimated there are approximately 500,000 people across the globe affected by EB, a disease for which there is no approved treatment. If Filsuvez is approved, Amryt expects the global market for the drug to be in excess of $1 billion.
“If approved, we intend to leverage our existing global infrastructure to commercialize Filsuvez,” Wiley said in a statement. “We look forward to working with regulatory authorities to make Filsuvez available as the first approved therapeutic treatment for EB patients.”
Amryt will submit a rolling New Drug Application to the U.S. Food and Drug Administration and will request a priority review for its product. Amryt also intends to pursue an accelerated assessment in the European Union. Regulatory submissions in the U.S. and the EU are expected to be filed by the end of the first quarter of 2021.
Although Amryt reported that Filsuvez hit the mark with its primary endpoint, it did not fare as well with its secondary goal. While the key secondary endpoints did not achieve statistical significance, a number of favorable differences were observed. In addition, substantial further secondary endpoint data is expected and will be analyzed over the coming weeks, the company said.
Full trial results will be presented at a future medical conference once the data has been fully analyzed.