TWi Biotech Gets U.S. And Taiwan Approval For AC-201 Phase 2 Trial
TWi Pharmaceuticals announced that its subsidiary TWi Biotechnology has scored regulatory approvals from both U.S. and Taiwan authorities for the protocol of a Phase 2 clinical trial investigating its lead drug AC-201.
AC-201 is first-in-class small molecule that works to inhibit the activity and production of caspase-1 and the cytokine Interleukin-1Beta (IL-1Beta). The drug is also designed to down-regulate the receptors of IL-1Beta. Hindering IL-1Beta signaling has shown potential for the treatment of autoimmune diseases, such as diabetes mellitus, arthritis, and gout. TWi Biotechnology has filed two investigational new drug (IND) applications with the U.S. Food and Drug Administration (FDA) for the drug as a gout treatment and as a method to control blood sugar in patients with Type 2 diabetes.
The company reported that it has received the go-ahead signal from both regulatory offices to begin a Phase 2 trial for AC-201 in high blood uric acid level and gout. Dr. Calvin C. Chen, President of TWi Biotechnology, said, “We are very pleased with the approval of the Phase 2 clinical trial protocol for AC-201 CR. The purpose of this Phase 2 trial is to determine the effectiveness of AC-201 CR in reducing uric acid levels in the blood of gout patients… We developed a safer and higher bioavailability formulation than the original instant-release formulations. The upcoming trial will assess the efficacy and safety of this new formulation.”
Gout is a potentially curable disease that affects an estimated 8.3 million patients in the U.S. alone. The disease is caused by the deposition of monosodium urate (MSU) crystals in patients with hyperuricemia and marked by severely painful and recurrent acute attacks of arthritis. The number of gout sufferers worldwide is projected to balloon to 18 million by 2021.
TWi Biotech said it will begin enrollment of patients for the Phase 2 trial of AC-201 in gout as soon as possible.
Aside from gout, the company is also investigating the drug in the rare genetic disease epidermolysis bullosa (EB), for which AC-201 received Orphan Drug Designation from the FDA in October this year.