News Releases

Taiho Pharmaceutical Co., Ltd. announced that the results of a phase III, randomized, placebo-controlled, double-blind and open-label, extension study of TAS-205 in patients with Duchenne muscular dystrophy (DMD) (REACH-DMD) showed no significant difference in the mean change from baseline to 52 weeks in the time to rise from the floor, which was the primary endpoint in the ambulatory cohort of the study.

The ambulatory cohort of this study was conducted in Japan as a placebo-controlled, multi-center, double-blind comparative study in male DMD patients aged 5 years and older. The purpose of this study is to evaluate the efficacy of TAS-205 by orally administering TAS-205 or a placebo twice daily for 52 weeks. A total of 82 patients were enrolled in 26 sites from November 2020 to December 2023. Detailed results from this study will be presented at an appropriate upcoming academic conference.
For more information on this study (NCT04587908/jRCT2041200055), please visit: www.clinicaltrials.gov/ / https://jrct.mhlw.go.jp/en-top

In December 2019, development of TAS-205 was selected as one of the 4th Cyclic Innovation for Clinical Empowerment (CiCLE*) program for fiscal 2019, under the application title: Development of an innovative therapeutic drug for DMD with a novel mechanism of action originating from Japan by utilizing patient registry. This program is operated by the Japan Agency for Medical Research and Development (AMED).
*CiCLE：Cyclic Innovation for Clinical Empowerment

TAS-205 (INN: pizuglanstat) is a selective hematopoietic prostaglandin D synthase (HPGDS*) inhibitor discovered by Taiho Pharmaceutical. It is under development as a DMD treatment which can be used regardless of the dystrophin gene mutation type, controlling the decline in motor function in DMD patients by inhibiting HPGDS, which exacerbates the inflammatory response in DMD patients’ muscles.
*HPGDS：Hematopoietic prostaglandin D synthase

This program aims at creating a foundation (including human resources) for a radical transformation in the way research and development is conducted in response to needs arising in clinical settings and for accelerating the practical application to pharmaceutical products. It also seeks to drive the creation of an environment that strongly promotes the fostering of open innovation in the field of medical research and development through the combined efforts of government, academia, and the private sector.

DMD is a genetic disorder with a higher prevalence in young males. It is caused by a genetic mutation in a gene, which codes dystrophin protein supporting the framework of muscle cells. It is a refractory, serious illness leading to a loss of muscle strength caused by the inability to produce normal dystrophin protein. The prevalence of DMD is 1.9 to 3.4 per 100,000 individuals¹, and there are an estimated 3,000 to 4,000 patients in Japan. Currently, oral steroids and viltolarsen are approved and delandistrogene moxeparvovec has received conditional and time-limited approval for DMD in Japan, however new treatment options are being awaited.

1. Nonaka I. Muscle Pathology for Clinical Practice. 4th ed. Japan Medical Journal; 2011: 46-59.