Taiho Pharmaceutical’s investigational Duchenne muscular dystrophy (DMD) therapy, pizuglanstat, has shown no benefit in a Phase III trial.
The REACH-DMD study (NCT04587908) showed no significant difference in the mean change in time to rise from the floor after one year, the primary endpoint in the ambulatory cohort of the study.
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This cohort enrolled male DMD patients aged five years and older from Japan, and the trial was conducted as a placebo-controlled, double-blind comparative study.
Taiho Pharmaceutical is a subsidiary of the Otsuka Group, which is listed on the Tokyo Stock Exchange. On 8 July, Otsuka shares closed 1.62% lower (Y6,622.00) ($45.08) compared to 7 July (Y6,731.00) ($45.82).
Taiho’s study has also enrolled a non-ambulatory DMD cohort, with investigators evaluating the safety of pizuglanstat in the population. Data from this population has not yet been released.
The company said that detailed results from this study will be presented at an appropriate upcoming academic conference, but it is unclear whether that will include data from the non-ambulatory cohort.
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By GlobalDataPizuglanstat acts by inhibiting hematopoietic prostaglandin D synthase (HPGDS), which is involved in the progression of muscular necrosis in DMD. As a result, inhibition of this leads to decreased synthesis of prostaglandin D2, which results in a reduction in the necrotic muscle fibres.
Another major setback for DMD research
This latest readout is another difficult moment for the DMD community. In October 2023, Sarepta and Roche’s DMD gene therapy Elevidys (delandistrogene moxeparvovec-rokl) failed to meet the primary endpoint in a Phase III, confirmatory trial. As well as this, there have been several patient deaths after the therapy was administered, with the US Food and Drug Administration (FDA) launching an investigation into the therapy in June 2025.
In June 2024, Pfizer pulled its DMD gene therapy, fordadistrogene movaparvovec, after a patient in a Phase II trial died last year and a Phase III trial failed to show any benefit.
There have recently been some promising studies, including Satello’s SAT-3247, which showed a doubling of grip strength in a Phase Ib study.
Entrada Therapeutics has also received authorisation from the Health Authorities and Ethics Committees within the European Union (EU) to begin a randomised Phase I/II trial of ENTR-601-45, an endosomal escape vehicle (EEV)-conjugated phosphorodiamidate morpholino oligomer. This news came after the FDA released a two-year hold on the therapy after requesting additional data.
GlobalData predicts the DMD market will grow to $5.2bn in the seven major markets (7MM: US, France, Germany, Italy, Spain, the UK, and Japan) by 2033. This expansion is primarily driven by the recent approvals of innovative therapies, notably Sarepta Therapeutics/Roche’s Elevidys and Santhera Pharmaceuticals’ Agamree.
