Taiho’s DMD Therapy Falls Short in Phase III Trial

Taiho Pharmaceutical’s pizuglanstat failed to meet the primary endpoint in a Phase III trial for Duchenne muscular dystrophy (DMD). The REACH-DMD study found no significant improvement in time to rise from the floor in ambulatory male patients aged five and above. The trial was placebo-controlled and double-blinded, with data from the non-ambulatory cohort still pending.

Pizuglanstat inhibits HPGDS, aiming to reduce muscle necrosis by lowering prostaglandin D2 levels.

The setback follows other recent disappointments in DMD, including Sarepta/Roche’s Elevidys and Pfizer’s fordadistrogene, both failing in late-stage trials—some tied to patient deaths.

Still, hope remains: Satellos’ SAT-3247 showed early promise, and Entrada’s ENTR-601-45 has begun EU trials post-FDA clearance.

Despite setbacks, the DMD market is projected to reach $5.2B by 2033 across major global markets.

10-07-2025