This trial is designed to be a double-blind, intra-individual comparison, proof-of-concept clinical trial, and will enroll patients as young as 2 year old.
The primary efficacy endpoint is the reduction of lesion surface in patients with EB.
AC-203 is a proprietary topical formulation of 1% diacerein, which has shown the ability to inhibit the production and activity of caspase-1 and the cytokine Interleukin-1Beta (IL-1Beta) through the inhibition of the assembly of the NLRP3 inflammasome.
Inhibition of the NLRP3 inflammasome associated signaling pathway and IL-1Beta has been demonstrated to be effective in treating a variety of diseases including arthritis, gout, and diabetes mellitus.
TWi biotechnology specializes in the development of repositioned drugs for unmet medical needs, especially in diseases associated with innate immunity.
The company is building its product pipeline through in-licensing and internal research. Its product development pipeline includes two drug candidates for treating epidermolysis bullosa, gout, type II diabetes, hemophilic arthropathy, and immunodermatology diseases.
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