TAIPEI, Taiwan, Jan. 22, 2019 /PRNewswire/ -- TWi Biotechnology Inc. (TWiB), a clinical stage biopharmaceutical company focused on development of novel products for rare diseases associated with chronic inflammation, announced today that it has completed patient enrollment in its Phase 2 clinical trial (Study code: AC-203-EBS-005) of AC-203 for the treatment of inherited Epidermolysis Bullosa (EB), including Epidermolysis Bullosa Simplex (EBS), Dystrophic Epidermolysis Bullosa (DEB) and Junctional Epidermolysis Bullosa (JEB). The Phase 2 clinical trial is the first multi-center, double-blind, randomized, placebo-control, intra-individual comparison study conducted to evaluate the efficacy, safety and tolerability of AC-203 in different EB sub-types. Launched in October 2018, enrollment was completed within 3 months with 9 subjects randomized at 2 sites in Hsinchu and Tainan. The company expects to report top-line results in second quarter of 2019. The trial results will help support TWIB's global development partner, Castle Creek Pharma (CCP), to extend the indication in both US and EU.
Enrolled patients will complete 8 weeks of once daily topical treatment in two designated lesion areas (one with AC-203 and one with placebo) followed by 4 weeks of follow-up period with no treatment. To evaluate efficacy, the study measures change of lesion surface areas in blisters, erythema, erosion and crust within the designated lesion areas from baseline by treatment. Other assessments include safety, pruritus and pain scale, levels of pro-inflammatory cytokines, and analysis of daily digital skin images of lesions taken by patients.
"EB is a debilitating skin disease without any effective or FDA-approved treatment. Some EB sub-types could be lethal and patients are more at risk for developing squamous cell carcinoma. It is a huge burden for the patients and families. Completing patient enrollment for AC-203-EBS-005 at such a short time is an important milestone for TWiB and triumph for EB patients, and we are grateful for the support of trial participants and their families, patient groups and investigators to achieve our enrollment goal," commented Calvin Chen, Ph.D., Chief Executive Officer of TWiB. "We are committed to bring AC-203 treatment to EB patients and believe the results of this study will be an essential step for future global registration trial in partnership with CCP. In addition, the study will offer a good opportunity to allow our local investigators to integrate into the international EB research community."
For more information on the Phase 2 study with AC-203 for the treatment of EB, please visit ClinicalTrials.gov (reference identifier NCT03468322).
AC-203 is a proprietary topical formulation of 1% diacerein, which has been shown to mechanistically inhibit NLRP3 inflammasome activation. The blockade of the NLRP3 inflammasome-associated signaling pathway suppresses activity of caspase-1 and subsequent production of the major pro-inflammatory cytokine Interleukin-1Beta (IL-1Beta), therefore reducing chronic inflammation. AC-203 has been demonstrated to be effective in treating a variety of inflammatory diseases including arthritis, gout, and diabetes mellitus (DM). The oral formulation of diacerein has been approved for the treatment of chronic rheumatic diseases in France, Spain, Italy and other EU and Middle Eastern countries since the mid-1990s. AC-203 was granted Orphan Drug Designation by both USFDA and TFDA for EB indication.
About Epidermolysis Bullosa (EB)
EB is a heterogenous group of rare genetic diseases characterized by skin fragility due to separation between layers of skin. EB patients develop blisters in response to minor trauma or injury. In DEB and JEB, wounds are often chronic and difficult to heal. At least 18 genes have been identified to cause EB. Other clinical manifestations can include anemia, cardiomyopathy, syndactyly (fusion of the fingers and toes), renal insufficiency, dysphagia (difficulty swallowing), malnourishment, cancer, constipation, osteoporosis, muscular dystrophy and pyloric atresia. Recent research has indicated that inflammation plays a critical role in EB and thus offers a new direction to treat and manage the disease. 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, specializing in development of repositioned drugs for rare diseases with high unmet medical needs. The company is expanding its pipeline products through in-licensing, internal research, and drug repositioning screen to identify and to develop novel inflammasome inhibitors to treat rare diseases associated with chronic inflammation triggered by innate immunity. TWiB's product development pipeline includes two leading drug candidates for treating epidermolysis bullosa, hemophilic arthropathy, and immunodermatological diseases.
SOURCE TWi Biotechnology, Inc.