TWi Biotechnology Inc. Reports Positive Interim Results from AC-201 CR Phase 2 Proof-of-Concept Study in Gout Patients

TAIPEI, Taiwan, Dec. 24, 2015 /PRNewswire/ -- TWi Biotechnology Inc. today announced interim results from the ongoing Phase 2 proof of concept clinical study evaluating AC-201 CR as an oral uricosuric and anti-inflammatory agent for the treatment of hyperuricemia and prevention of gout flares combining with febuxostat, a xanthine oxidase inhibitor.

The interim analysis is for the first enrollment cohort (18 per arm completing Week 8), if the number of subjects in the AC-201 CR arm achieving the clinical target of sUA <6.0 mg/dL at Week 8 is at least 2 greater than the number in the placebo arm, then the remaining subjects  will be enrolled in the second cohort; if not, enrollment will be stopped. Based on the report, the responders in AC-201 CR arm is at least 2 greater than the number in the placebo arm. In addition to producing the efficacy of AC-201 CR in gout patients, there have been no serious adverse events reported to date and AC-201 CR was generally safe and well tolerated in the study patients. Therefore, TWIB is able to continue to enroll second cohort subjects and complete this clinical trial in the end of 2016.

"We are encouraged to see the potential efficacy of AC-201 CR in the Phase 2 proof-of-concept study results to date," said Dr. Calvin C. Chen, President and CEO of TWIB. "Current evidence of AC-201 CR efficacy and the preliminary results for tolerance support the potential of AC-201 CR as a dual uricosuric and anti-inflammatory agent for the treatment of hyperuricemia and the prevention of gout flares when combining with xanthine oxidase inhibitor. I am very pleased with the interim data as it indicates AC-201 CR may increase the successful rate of the treatment of gout without increase or even decrease the flare rate. The poor compliance of the urate lowering therapy due to frequent gout flares at the initiation and low treatment successful rate offered by existing therapies are the major unmet needs for the management of sUA and gout."

About the Study

The ongoing Phase 2a study (study code: AC-201-GOU-002) is an randomized, double-blind, placebo-controlled, multicenter study. This study is designed to test the urate-lowering effects, safety and tolerability of AC-201 controlled-release tablets (vs.placebo) in an initial dosing period as a monotherapy, followed by addition of febuxostat to test the efficacy and safety of the combination. At up to a maximum of 14 clinical sites in Taiwan, the study aims to enroll 140 eligible subjects, who are confirmed with gout, and who will be randomized in a 1:1 allocation ratio to either AC-201 CR or Placebo over a with a 12-week treatment period and a 4-week follow-up period.

The primary efficacy endpoint is the proportion of subjects achieving serum uric acid concentration <6.0 mg/dL at Week 8. The major secondary efficacy endpoints are proportion of subjects achieving serum uric acid concentration <6.0 mg/dL or <5.0 mg/dL at each visit; number of gout flares per subject during each period and overall.

About Hyperuricemia and Gout

Gout is a medical condition usually characterized by recurrent attacks of acute inflammatory arthritis -- a red, tender, warm, swollen joint caused by an inflammatory response to uric acid crystals deposited in joints and soft tissues as a result of elevated levels of uric acid in the blood (hyperuricemia). Urate crystals in joints can trigger acute arthritic flares, chronic destructive arthropathy, and formation of tophi. According to the U.S. National Health and Nutrition Examination Survey (NHANES) study, the prevalence of gout among US adults in 2007-2008 was 3.9% (8.3 million individuals).

About AC-201/ AC-201 CR

AC-201 is a first-in-class, orally available small molecule which has shown the dual abilities to inhibit URAT1 as well as 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 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. AC-201 CR is the controlled release oral formulation of AC-201.

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 with up to 6 months treatment period with a satisfactory safety profile demonstrated.

About TWi Biotechnology, Inc.
TWi Biotechnology, Inc., a 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 two drug candidates for treating type II diabetes, arthritis, gout, and immunodermatology diseases. TWi Biotechnology holds US and Taiwan orphan drug designations for AC-203 for Epidermolysis Bullosa treatment as well as two US INDs for the oral dosage form of the molecule for type 2 diabetes and gout treatment.

Contact :
Weishu Lu 
Tel: +886-2-2657-1788 #300
email: [email protected]

SOURCE TWi Biotechnology, Inc.