Drug for Rare Skin Disease Scrapped After Trial Failure

Amicus won’t pursue wound-healing agent SD-101 for epidermolysis bullosa

Amicus Therapeutics, Inc., will end development of a medication meant to treat the devastating rare skin disorder epidermolysis bullosa (EB) after the drug failed to help patients in a phase 3 trial.

Amicus said that top-line data from the randomized, double-blind, placebo-controlled clinical study ESSENCE to assess the efficacy and safety of its novel topical wound-healing agent SD-101 did not meet primary or secondary endpoints.

The ESSENCE study randomized 169 participants with a documented diagnosis of simplex, recessive dystrophic, or junctional non-Herlitz EB to SD-101 (n = 82) or placebo (n = 87) during a three-month primary treatment period. SD-101 did not show a statistically significant difference from placebo in the intent-to-treat (ITT) population (n = 169).

The first primary endpoint, the time to target wound closure within three months, did not differ between groups (hazard ratio, 1.004; P = 0.985). The second primary endpoint, the percentage of patients achieving target wound closure by month 3, also did not differ between groups (49%, SD-101; 54%, placebo; P = 0.390). Similarly, the secondary endpoints did not reach statistical significance versus placebo.

Encouraging trends in wound closure were seen in certain subpopulations. Treatment-emergent adverse events were similar across both the SD-101 and placebo groups, with the most common being nasopharyngitis, pruritis, and pyrexia.

“We are disappointed that this phase 3 study of SD-101 did not meet the primary endpoints in epidermolysis bullosa, an utterly devastating rare genetic disorder with no approved treatment options,” said  John F. Crowley, Chairman and Chief Executive Officer of Amicus, which will work closely with investigators and other experts to understand and share these data. Based on these results, Amicus has no current plans to invest in any additional clinical studies or commercial preparation activities for SD-101.

In consultation with their physicians, participants in the ongoing extension studies (SD-004 and SD-006) can continue being treated with SD-101.

EB is a rare, devastating genetic disorder that leads to severe skin blistering and open wounds, often beginning at birth. It affects tens of thousands of children and adults throughout their lifetimes. EB is chronic, potentially disfiguring, and in some cases fatal.

There are many genetic and symptomatic variations of EB, but all forms share the common symptom of fragile skin that blisters and tears, sometimes from the slightest friction or trauma. Current standard of care consists of pain management and the bandaging and cleaning of open wounds to prevent infection.

Source: Amicus Therapeutics, Inc.; September 13, 2017.