SD 101 fails to meet endpoints in Phase III ESSENCE study to treat epidermolysis bullosa.- Amicus Therapeutics.

- Amicus Therapeutics, Inc. reported that top-line data from the randomized, double-blind, placebo-controlled Phase III clinical study (ESSENCE, SD-005) to assess the efficacy and safety of the novel topical wound-healing agent SD 101 did not meet the primary endpoints or secondary endpoints in participants with epidermolysis bullosa (EB). The Phase III ESSENCE study randomized 169 participants with a documented diagnosis of Simplex, Recessive Dystrophic, or Junctional non-Herlitz epidermolysis bullosa 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 3 months, was not different between groups (Hazard ratio=1.004, p=0.985). The second primary endpoint, the percentage of patients achieving target wound closure by month 3, was also not different 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 observed in certain sub-populations. Treatment-emergent adverse events (TEAEs) were similar across both the SD 101 and placebo groups. The most common TEAEs were nasopharyngitis (common cold), pruritis (itchy skin), and pyrexia (fever).

Based on these top-line data Amicus has no current plans to invest in any additional clinical studies or commercial preparation activities for SD 101.