TWi Biotechnology, Inc. today announced that it has enrolled the first patient in a phase 2 trial of AC-203 for the treatment of inherited Epidermolysis Bullosa (EB).
TAIPEI, Taiwan, Oct. 20, 2018 /PRNewswire/ -- TWi Biotechnology, Inc. (6610 TTO), a clinical stage biotechnology company focused on developing novel inhibitors of inflammasomes, today 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.”
About AC-203
AC-203 is a proprietary topical formulation of 1% diacerein, which has been shown to inhibit the activity of caspase-1 and production of cytokine Interleukin-1Beta (IL-1Beta) through inhibition of theNLRP3 inflammasome activation. Blockade of the NLRP3 inflammasome-associated signaling pathway and subsequent IL-1Beta production has been demonstrated to be effective in treating a variety of diseases including arthritis, gout, and diabetes mellitus (DM). The oral formulation containing diacerein has been approved for with the treatment of chronic rheumatic diseases in France and subsequently in other EU and Middle Eastern countries such as Spain and Italy since the mid-1990s.
About Epidermolysis Bullosa (EB)
EB is caused by mutations in genes which consist of long strands of DNA responsible for making and expressing proteins. At least 18 genes have been identified to cause EB. These mutations, or errors in the genetic code, may not allow the affected gene to produce a specific protein at all, or to produce a defective protein with deleterious functions. Either way, the affected protein does not work correctly and thus resulting in extremely fragile skin. Clinical manifestations of other EB subtypes include anemia, cardiomyopathy, syndactyly (fusion of the fingers and toes), renal insufficiency, dysphagia (difficulty swallowing), malnourishment, cancer, constipation, osteoporosis, muscular dystrophy and pyloric atresia. The number of EB patients is estimated to be around 60,000 in US and EU combined.
About TWi Biotechnology, Inc.
TWi Biotechnology, Inc. is a leading clinical stage biopharmaceutical company based in Taipei, Taiwan, specializing in the development of repositioned drugs for unmet medical needs, especially in rare diseases associated with innate immunity. The company is expanding its product pipeline through in-licensing and internal research. TWi Biotechnology’s product development pipeline includes two drug candidates for treating epidermolysis bullosa, gout, hemophilic arthropathy, and immunodermatology diseases.
Media Contact
Contact Person: Weishu Lu
Phone Number: +886-2-26571788 ext.300
Email: Weishu.Lu@twibiotech.com
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