Clinical Trials

Date: 2017-04-03

Type of information: Results

phase: 3

Announcement: results

Company: Amicus Therapeutics (USA - NJ)

Product: SD-101

Action mechanism:

• SD-101 is a novel, late-stage, proprietary topical treatment  developed by Scioderm.  The FDA has granted Breakthrough Therapy designation for SD-101 based on results from a Phase 2a study for the treatment of lesions in patients suffering with epidermolysis bullosa.

Disease: epidermolysis bullosa

Therapeutic area: Rare diseases - Genetic diseases - Dermatological diseases

Country: Australia, Austria, France, Germany, Israel, Italy, Lithuania, The Netherlands, Poland, Serbia, Spain, UK, USA

Trial details:

• ESSENCE is a double-blind, placebo-controlled registration study that completed enrollment of more than 160 patients who have a documented diagnosis of Simplex, Recessive Dystrophic, or Junctional non-Herlitz Epidermolysis Bullosa.
• The aim of the ESSENCE trial is to assess the efficacy and safety of SD-101-6.0 cream versus SD-101-0.0 (placebo) in the treatment of patients with Epidermolysis Bullosa. (NCT02384460)

Latest news:

• • On September 13, 2017, Amicus Therapeutics reported that top-line data from the randomized, double-blind, placebo-controlled Phase 3 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 .
• The Phase 3 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).
• Amicus plans to further analyze and share the Phase 3 ESSENCE results with key stakeholders in the epidermolysis bullosa community including physicians, patient organizations and regulators. In the interim, in consultation with their physicians, participants in the ongoing extension studies (SD-004 and -006) will have the opportunity to continue being treated with SD-101. 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.
• • On May 19, 2017, Amicus Therapeutics has completed the analysis plan for the primary endpoints in the blinded ongoing Phase 3 clinical study (ESSENCE) of the novel topical medicine SD-101 for epidermolysis bullosa (EB). Based on discussions and written communication from the Dermatology Division of the FDA, Amicus will analyze the primary endpoints for the Phase 3 ESSENCE study as follows:
• The endpoint of "time to wound closure within 3 months" will be analyzed first. If the difference between SD-101 6% and placebo is statistically significant (p ? 0.05), then the study will be considered a success.
• If the first endpoint is statistically significant, then "proportion of patients with target wound closure at month 3" will be analyzed and considered statistically significant at p ? 0.05.
• Amicus is on track to report top-line data from the ESSENCE study during the third quarter of 2017. To date, more than 95 percent of patients completing the 3-month primary treatment period have elected to continue in the open-label extension study.
• • On April 3, 2017, Amicus Therapeutics has completed enrollment in the ongoing Phase 3 clinical study (ESSENCE) of the novel topical medicine SD-101 for patients with all 3 major types of epidermolysis bullosa (simplex, recessive dystrophic, and junctional non-Herlitz). With the achievement of full enrollment, top-line data from this study are expected in the third quarter of 2017. The ESSENCE Study has enrolled more than 160 patients who have a documented diagnosis of simplex, recessive dystrophic, or junctional non-Herlitz epidermolysis bullosa. To date, more than 95 percent of patients completing the 3-month primary treatment period have elected to continue in the open-label extension study.

   

Is general: Yes