Amicus' SD-101 misses in Phase III for skin blistering disorder

Amicus Therapeutics Inc. (NASDAQ:FOLD) reported top-line data showing that SD-101 missed the two primary endpoints in the Phase III ESSENCE trial to treat simplex, recessive dystrophic or junctional non-Herlitz epidermolysis bullosa (EB). Based on the data, the company said it has "no current plans to invest in any additional clinical studies or commercial preparation activities for SD-101."

On the first primary endpoint, once-daily topical SD-101 for 90 days did not significantly improve time to target wound closure within 3 months vs. placebo (HR=1.004, p=0.985). On the second primary endpoint, SD-101 did not significantly improve the proportion of patients who achieved target wound closure by 3 months vs. placebo (49% vs. 54%, p=0.39). Amicus said SD-101 also missed the secondary endpoints vs. placebo. The most common treatment-emergent adverse events reported were nasopharyngitis, pruritus and pyrexia...