TWi Biotechnology Receives Orphan Drug Designation for its Lead Drug Candidate AC-201 in United States.

2014.10.21 TWi Pharmaceuticals, Inc. today announced that its fully owned subsidiary, TWi Biotechnology, Inc., has received the designation of Orphan Drug by US FDA for using AC-201 to treat epidermolysis bullosa (EB). TWi Biotechnology has developed proprietary formulations to be used topically for the indication of EB.


"We are very pleased with the approval of orphan drug designation for treating epidermolysis bullosa. It fulfills the mission of our new drug business for meeting unmet medical needs.” said Dr. Calvin C. Chen, President of TWi Biotechnology. “Epidermolysis Bullosa, or EB, is a very rare genetic connective tissue disorder that affects 1 child out of every 50,000 births. It is estimated there are approximately 30,000 patients in US, and a few hundreds children in Taiwan suffered from the deliberating disease, which is also known as “Butterfly Children” in the United States and is called “Bubble Dragan Children” in Taiwan because patients have extremely fragile skin that blisters and tears from friction or trauma. We hope this orphan drug designation can result in a speedy development of topical AC-201. Current treatments of EB are focusing in relief of symptoms such as itching and pain and wound care to prevent infection, which are expensive such as artificial skin and may require frequent visiting to hospitals. In contrast, the topical formulation of AC-201 is developed to prevent or reduce the blisters that, if success, may alleviate the suffering of the patients and their families from this deliberating disease.”


Added Dr. Chen: “We will soon proceed to the clinical trials for the topical formulation of AC-201.”


About AC-201
AC-201 is a, first-in-class, small molecule which has shown the ability to inhibit the production and activity of caspase-1 and the cytokine Interleukin-1Beta (IL-1Beta), and to down-regulate IL-1Beta receptors. Inhibition of IL-1Beta signaling has been demonstrated to be effective in treating a variety of diseases including arthritis, gout, and diabetes mellitus (DM). The active ingredient of AC-201 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 mid-1990.


TWi Biotechnology holds two US INDs for AC-201–one for controlling blood glucose in patients with type II diabetes and the other for treating gout in patients under taking urate-lowering therapy. In addition to the good safety record of its active ingredient used in treating another chronic disease, AC-201 has undergone testing in several human clinical trials including 3 phase II trials for up to 6 months treatment period with a satisfactory safety profile demonstrated.


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 one genetic 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 like: anemia, cardiomyopathy, syndactyly (fusion of the fingers and toes), renal insufficiency, dysphagia (difficulty swallowing), malnourishment, cancer, constipation, osteoporosis, muscular dystrophy and pyloric atresia.


About TWi Biotechnology, Inc.
TWi Biotechnology, Inc., a fully owned subsidiary of TWi Pharmaceuticals, Inc., is a leading clinical stage biopharmaceutical company based in Taipei, Taiwan, specializing in the development of innovative new 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 Biotechnology's product development pipeline includes three drug candidates for treating type II diabetes, retinopathy, arthritis, and immunodermatology diseases.


Contact :
Eric Lin
Tel: +886-2-2657-3350 #407
email: eric.lin@twipharma.com