22 October 2018 | News
There is no approved drug for any type of EB. The wound care of EB patients has to be meticulous and can be very traumatic to patients and caregivers.
TWi Biotechnology, Inc. a clinical stage biotechnology company focused on developing novel inhibitors of inflammasomes has announced that it has enrolled the first patient in a phase 2 trial of AC-203 for the treatment of inherited Epidermolysis Bullosa (EB). This study 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 assessment of efficacy and safety outcomes includes reduction of lesion surface area and blister number, improvement in pruritus and pain, decreased levels of pro-inflammatory cytokines and tolerability of AC-203 in patients with EB.
Based on the specific cleavage site within the skin layers, EB is typically classified into 4 types: Simplex, Dystrophic, Junctional, and Kindler Syndrome. The genetic basis underlying different EB subtypes is distinct and recently inflammation is thought to play important roles in modulating the symptoms of the diseases. TWi Biotech has partnered with Castle Creek Pharmaceuticals, LLC. (CCP), which is leading the global clinical development using AC-203 (CCP code name: CCP-020) for treating EB Simplex. "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," said Calvin Chen, Ph.D., CEO and president of TWi Biotech. "We are grateful for the support of local EB patient groups. They have guided us since the beginning of the program."
There is no approved drug for any type of EB. The wound care of EB patients has to be meticulous and can be very traumatic to patients and caregivers. "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," said Ms. Se-Meng Cheng, founder and CEO of the patient group Taiwan Debra Home Care Promotion Association, who also is the mother of a EB Junctional patient. "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."