Taiho Pharmaceutical’s investigational treatment for Duchenne muscular dystrophy (DMD) has failed to impact the time it takes patients to rise from the floor over 52 weeks, missing the phase 3 study’s main goal.
The late-stage efficacy data included 82 male patients with DMD aged 5 years and older, according to a July 8 release. Participants received either Taiho’s TAS-205—an orally administered candidate also known as pizuglanstat—or placebo twice a day for a year.
The double-blind study was split into two cohorts: an ambulatory group and a non-ambulatory group. The primary endpoint for the ambulatory cohort measured the mean change from baseline for the time it takes to get up from the ground.
TAS-205 failed to demonstrate a significant difference in time to rise from the floor over 52 weeks. Further findings from the study, which was conducted in Japan and known as REACH-DMD, are expected to be presented at an upcoming academic conference, Taiho said in the release.
Taiho didn’t share information about the non-ambulatory group, which was being measured for adverse events and reactions. In an early phase 2 study assessing TAS-205, no obvious differences in the incidences of adverse events were reported between treatment and placebo groups.
When asked by Fierce Biotech, Taiho said both cohorts of the trial have been discontinued. The company said it was considering all possibilities regarding the future of TAS-205.
Taiho-developed TAS-205 is designed to selectively inhibit hematopoietic prostaglandin D synthase (HPGDS), an enzyme related to inflammation responses in patients with DMD. The serious genetic condition causes muscle strength loss because the body isn’t able to produce normal dystrophin protein.
The Japan-based biopharma is owned by Otsuka Holdings, parent company to Otsuka Pharmaceutical.
Editor's note: This article was updated at 9 a.m. ET on July 9 with a response from Taiho.