Amicus Therapeutics’ Phase 3 EB Clinical Trial

Below you will find updated information about the ESSENCE Study, a Phase 3 clinical trial of SD-101, an investigational topical cream being developed by Amicus, together with Scioderm, to treat skin blistering and lesions associated with Epidermolysis Bullosa (EB).

In April 2017, Amicus Therapeutics announced the completion of enrollment in Phase 3 of the ESSENCE Study, a clinical study of the novel topical medicine SD-101 for patients with 3 major types of EB (Simplex, Recessive Dystrophic, and Junctional non-Herlitz EB). Enrollment exceeded the target with more than 160 patients enrolled. With the achievement of full enrollment, top-line data from this study are expected in the third quarter of 2017.

A message from Amicus: "Completing this trial and analyzing the results is important to better understand the effect of SD-101 on healing wounds of people with EB. While there remains much work to do, the closing of enrollment in the trial is a significant step in the drug development process. We at Amicus know how hopeful the EB community is to learn of the potential for a first treatment for EB. Amicus will proceed to evaluate the results of the emerging data and determine the next steps in the development of this investigational product. If you have any further questions, please e-mail us again at info [at] ebstudy [dot] com."

What is the ESSENCE Phase 3 Study?

The ESSENCE Study is currently underway in the U.S. and Europe, with sites planned in additional geographies.

The double-blind, placebo-controlled trial will evaluate the safety and efficacy of SD-101 in patients with various subtypes of EB (Simplex, Recessive Dystrophic and Junctional [non-Herlitz]).

Participants will be randomized 1:1 to two treatment groups receiving either SD-101 6% or placebo applied over their entire body once daily for three months.

The primary efficacy endpoint will be complete closure of a target wound. Improvement in itching, pain, and full-body wound and lesion coverage will also be assessed. Investigators will also assess safety.

It is expected that everyone completing the three-month study will be offered the option to receive active SD-101 in an open-label extension trial.