US FDA, Taiwan FDA Approve TWi Biotechnology's Phase 2 Gout Trial
By Cyndi Root
TWi Pharmaceuticals is approved to run a Phase 2 trial of AC-201, according to the Food and Drug Administration (FDA) and the Taiwan FDA. The company announced the approvals in a press release, stating that AC-201 is a controlled-release (CR) tablet to reduce the high blood uric acid levels in gout patients. TWi Pharmaceuticals and its fully owned subsidiary, TWi Biotechnology will enroll patients as soon as possible. Dr. Calvin C. Chen, President of TWi Biotechnology, said, “The currently available drugs either have safety concerns or increase the frequency of gout flares, reducing the compliance of regular drug dosing by patients. We developed a safer and higher bioavailability formulation.”
AC-201 CR
AC-201 is a small molecule that inhibits caspase-1 and the cytokine Interleukin-1Beta (IL-1Beta). The agent also down-regulates IL-1Beta receptors, which shows promise in treating arthritis, gout, and diabetes mellitus (DM). The active ingredient in the IL-1β modulator is approved to treat osteoarthritis in many countries. The agent has a demonstrated safety profile due to its testing in other indications. TWi Biotechnology now holds two INDs for the agent as a urate-lowering therapy in gout patients and as a method to control blood glucose in type 2 diabetes patients.
TWi states that it has developed a controlled release formulation of the agent that is safer and more bioavailable than the instant-release formulations. The trials approved by the U.S. and Taiwan are an effort to evaluate the safety and efficacy of the new formulation. The company hopes to prove that its treatment is more effective, safer, and more acceptable to patients than other treatments.
About TWi
TWi Biotechnology is based in Taipei, Taiwan where it focuses on new drugs for unmet medical needs and diseases associated with innate immunity. Product candidates treat type 2 diabetes, arthritis, and immunodermatology diseases. In October 2014, the company received Orphan Drug status from the FDA for AC-201 to treat epidermolysis bullosa (EB), a rare genetic connective tissue disorder. TWi estimates that 30,000 patients in U.S. have the disease, and in Taiwan, several hundred are what the Taiwanese call "Bubble Dragon Children" due to fragile, blistering skin in patients. The company hopes that its topical application of AC-201 will prevent or reduce the blisters and reduce suffering.