TAIPEI, Taiwan, Nov. 15, 2018 /PRNewswire/ — TWi Biotechnology, Inc. (TWiB, 6610.TWO), a clinical stage drug development company targeting rare, chronic and inflammatory diseases, announced today that it has completed patient enrollment in its Phase 2 clinical trial (Study code: AC-201CR-HA-001) of AC-201CR for the treatment of hemophilic arthropathy. The Company expects to release top-line results from the interim analysis of this study in the second quarter of 2019.
The multi-center, double-blind, randomized, placebo-control proof-of-concept Phase 2 study was launched in Taiwan in the fourth quarter of 2016 and designed to evaluate AC-201CR’s efficacy, safety and tolerability in up to 22 individuals with hemophilic arthropathy. AC-201CR-HA-001 study was composed of 3 sequential treatment periods of total 48 weeks. In Period 1, subjects were randomized in a 1:1 ratio to receive QD treatment of AC-201CR or placebo for 4 weeks. In Period 2, subjects titrated to BID regimen of blinded study medication for 20 weeks. In Period 3, all subjects discontinued blinded study medication and started open-label QD treatment with AC-201CR for 4 weeks followed by dose titration to BID for the remaining 20 weeks. The primary endpoint of the study will be assessed by change from baseline in IPSG (MRI) score for primary knee to evaluate joint structure-modifying effects of AC-201CR. Other secondary endpoints in the Phase 2 study to evaluate efficacy include MRI-measured synovial membrane thickness by knee, knee pain (by VAS), quality of life and safety.
Calvin Chen, Ph.D., Chief Executive Officer of TWiB commented, "Completing patient enrollment for AC-201CR-HA-001 is an important milestone for TWIB. Our development strategy for hemophilic arthropathy treatment is to provide not only a non-opioid/ non-traditional NSAID pain treatments for rare diseases like hemophilic arthropathy, but also slow down its disease progression in the future." "In hemophilic arthropathy, imaging plays a crucial role in accurately monitoring the disease process in all phases and evaluating treatment. In AC-201CR-HA-001 study, we selected MRI as the tool to measure the progress of hemophilic arthropathy, and the trial results will help us to design the next clinical trial to improve patient’s quality of life."
About Hemophilic Arthropathy (HA)
Hemophilia A or B patients suffer from spontaneous and recurrent joint bleedings (hemarthroses) that often progress to a debilitating disease called hemophilic arthropathy. Pathologically, HA is characterized by chronic synovitis, cartilage damage, bone destruction and ultimately joint deformation. Both inflammatory and degenerative mechanisms are thought to play pivotal roles in the pathophysiology of the disease. HA is the leading cause of disability in hemophilia patients. The pain caused by the intra-articular bleeding and HA significantly impacts daily activities and quality of life of the patients. Although many hemophilia patients are treated with clotting factors prophylactically, the risk of hemarthrosis is not completely eliminated. A high percentage of hemophilia patients (30-50%) present HA despite access to prophylactic treatment and majority of severe hemophilia patients develop HA early in their young adulthood There are estimated 45,000 people diagnosed with hemophilia in the US, EU and Japan combined. Hemophilic arthropathy is classified as a rare disease by US FDA, and there are currently no approved treatments in the United States.
About AC-201/AC-201 CR
AC201CR, a control released formulation of AC-201, demonstrates anti-inflammatory effects by inhibiting NLRP3 inflammasome activation and thus interleukin-1β signaling pathway. In additional, AC-201 influences both the anabolism and catabolism of chondrocytes in vitro and has shown cartilage-sparing properties in animal. The API of AC-201 was approved in Europe and Asia to treat osteoarthritis (OA), which bears resemblance to progressive degeneration of the cartilage in HA.
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 rare diseases associated with innate immunity. The company is expanding its product pipeline through in-licensing and internal research. TWiB’s product development pipeline includes two drug candidates for treating epidermolysis bullosa, gout, hemophilic arthropathy, and immunodermatology diseases.
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