TWi Biotechnology, Inc. Announced Regulatory Clearance to Begin Phase 2 Proof-of-Concept Clinical trial of AC-203 for treatment of Inherited Epidermolysis Bullosa

TAIPEI, Taiwan, Feb. 26, 2018 /PRNewswire/ -- TWi Biotechnology, Inc. ("TWi Biotech", TWO: 6610), a clinical stage biotechnology company focused on developing inhibitors of inflammasomes, today announced that it has received approval from the Taiwan Food and Drug Administration (TFDA) to proceed a phase 2 clinical trial of AC-203 for the treatment of inherited Epidermolysis Bullosa (EB). This trial 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 primary efficacy endpoint is the reduction of lesion surface in patients with EB. TWi Biotech expects the first patient will be enrolled in early April this year.

"While our partner for the territories outside of Asia, Castle Creek Pharmaceuticals (CCP), is conducting the global pivotal phase 2/3 DELIVERS clinical trial for the treatment of Epidermolysis Bullosa Simplex (EBS) using AC-203 (code name CCP-020 by CCP), we would like to evaluate the safety and efficacy of AC-203 in other types of EB," said Dr. Calvin C. Chen, president and CEO of TWi Biotech. "Because although most of EB patients are type Simplex (70%), and type Dystrophic (25%) and type Juncture (5%) are the minority among EB patients, their symptoms are more severe than the type Simplex according to DEBRA International, the worldwide patient group organization of EB." "The approval of the trial can enroll very young patients, 2 year old, is very important, because the younger the age of patients, the disease symptoms are more severe, and the risks of patient developing chronic complications and even death are higher. We are grateful for the support of the regulatory agencies, TFDA and the Center for Drug Evaluation (CDE), the principal investigators of the trial, and the local patient group, DEBRA Taiwan." The pivotal trial DELIVERS is approved to test in patient equal or more than 4 years old.

There isn't any 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 Managing Director of DEBRA Taiwan, who also is the mother of a JEB 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 the 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 the finding of effective treatments of EB for all the patients and their caregivers in the world."

About AC-203

AC-203 is a proprietary topical formulation of 1% diacerein, which has shown the ability to inhibit the production and activity of caspase-1 and the cytokine Interleukin-1Beta (IL-1Beta) through the inhibition of the assembly of the NLRP3 inflammasome. Inhibition of the NLRP3 inflammasome associated signaling pathway and IL-1Beta has been demonstrated to be effective in treating a variety of diseases including arthritis, gout, and diabetes mellitus (DM). The oral formulations containing diacerein has been approved for treating patients with 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 the DNA code that make up genes. Genes are responsible for making and expressing proteins. EB can result from a mutation in any one of the 18 genes that have been found to cause the disorder. These mutations, or errors in the genetic code, may not allow the affected gene to produce a specific protein, or it may result in the incorrect formation of that protein. Either way, the mutation does not enable the affected protein to work correctly thus resulting in extremely fragile skin, and other manifestations of EB including: 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 could 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 the diseases associated with innate immunity. The company is building its product pipeline through in-licensing and internal research. TWi Biotech's product development pipeline includes two drug candidates for treating epidermolysis bullosa, gout, type II diabetes, hemophilic arthropathy, and immunodermatology diseases.

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