TWi Biotechnology, a Taiwanese biotech company, has announced the enrollment of the first patient in a phase 2 trial evaluating AC-203 for the treatment of inherited Epidermolysis Bullosa (EB).
The phase 2 trial of AC-203 is a double-blind, intra-individual comparison, proof-of-concept clinical trial and will feature patients as young as two years of age.
The assessment of efficacy and safety outcomes of AC-203 includes reduction of lesion surface area and blister number apart from improvement in pruritus and pain and reduced levels of pro-inflammatory cytokines, and tolerability in patients with Epidermolysis Bullosa, said TWi Biotechnology.
Epidermolysis bullosa is a group of genetic conditions that make the skin very fragile and to blister easily. Depending on the specific cleavage site within the skin layers, Epidermolysis bullosa is classified typically into four types: Simplex, Dystrophic, Junctional, and Kindler Syndrome.
The genetic basis causing various subtypes of Epidermolysis bullosa is distinct and inflammation recently has been thought to play major roles in modulating the symptoms of the conditions.
TWi Biotechnology has partnered with Castle Creek Pharmaceuticals, which is heading the global clinical development using AC-203 for treating Epidermolysis bullosa Simplex.
Calvin Chen – CEO and president of TWi Biotechnology, commenting on the phase 2 trial of AC-203, 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.
“We are grateful for the support of local EB patient groups. They have guided us since the beginning of the program.”
Currently, no drug has been approved for any type of Epidermolysis bullosa. The wound care of Epidermolysis bullosa patients has to be very careful and could be very traumatic to both patients and caregivers.
Se-Meng Cheng – founder and CEO of the patient group Taiwan Debra Home Care Promotion Association, said: “Because EB is a devastating disease to the patients and their caregivers, everyday life could be a struggle, especially for the families with children suffering from EB.
“An effective treatment in reducing lesion would be wonderful for people affected by the disease not only in physical but also in mental and moral. We are extremely excited for TWi Biotech in developing treatment for EB patients and its decision in conducting the clinical study in Taiwan. We are looking forward to working with TWi Biotech and doctors in finding effective treatments for all EB patients and their caregivers in the world.”