Amicus Therapeutics' SD-101 fails to meet goals of Phase III study for epidermolysis bullosa

Amicus Therapeutics announced Wednesday that a late-stage study of SD-101 in patients with epidermolysis bullosa (EB) failed to meet its primary or secondary endpoints. The company, whose shares fell as much as 15 percent on the news, gained the experimental drug in 2015 via the purchase of Scioderm for an upfront payment of $229 million.

The ESSENCE trial randomised 169 patients with documented diagnosis of simplex, recessive dystrophic or junctional non-Herlitz EB to received treatment with SD-101 or placebo for three months. According to Amicus, top-line results showed that SD-101 did not demonstrate a significant difference from placebo in the intent to treat population for either the first main goal, the time to target wound closure within three months, or the second primary endpoint, the percentage of patients achieving target wound closure by month three.

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The company added that secondary endpoints did not reach statistical significance versus placebo, while "encouraging trends" in wound closure were observed in certain sub-populations. Amicus said that "based on these top-line data [it] has no current plans to invest in any additional clinical studies or commercial preparation activities for SD-101."

CEO John Crowley said "we are disappointed" in the results, adding "in seeking to develop novel, high quality therapies for those living with devastating rare diseases we may sometimes fail. But we would rather be the first to fail, than the last to try."

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