TWi Biotechnology has enrolled the first patient in a Phase II clinical trial of AC-203 for the treatment of inherited epidermolysis bullosa (EB).
EB is caused by mutations in genes that comprise long strands of DNA responsible for making and expressing proteins.
The double-blind, intra-individual comparison, proof-of-concept trial is expected to enrol patients as young as two years old.
The trial’s efficacy and safety objectives are a reduction of lesion surface area and blister number, improvement in pruritus and pain, and decreased levels of pro-inflammatory cytokines and tolerability of AC-203 in enrolled patients.
TWi Biotechnology CEO and president Calvin Chen said: "We are hopeful the unique activities of AC-203 in inhibiting the activation of NLRP3 inflammasome and decreasing the production of pro-inflammatory cytokines including IL-1beta and IL-18 could reduce the formation of blisters and the severity of skin lesions of EB patients.”
On the basis of specific cleavage site within the skin layers, EB can be classified into four types, which are simplex, dystrophic, junctional, and Kindler syndrome.
The genetic basis of different types of EB is different, and inflammation is believed to play an important role in modulating the symptoms of the diseases.
Currently, no drug is approved to treat any type of EB. Wound care for EB requires a lot of effort and can be very traumatic to patients and caregivers.
TWi Biotechnology has formed a partnership with Castle Creek Pharmaceuticals (CCP), which is leading the global clinical development using AC-203 for the treatment of EB Simplex.
AC-203 is a topical formulation of 1% diacerein that has demonstrated the ability to inhibit the activity of caspase-1 and generation of cytokine Interleukin-1Beta (IL-1Beta) through inhibition of NLRP3 inflammasome activation.