Advancing utrophin modulator SMTC1100 into clinical proof of concept trials for DMD

Around 1,500 boys and young men in the UK are living with Duchenne Muscular Dystrophy (DMD), a fatal genetic disease that progressively destroys their muscles, and no drug currently exists that can alter its course. Summit, a UK drug company, is developing SMTC1100, a compound that works by boosting a protein called utrophin to compensate for the missing dystrophin that causes the disease. Unlike some experimental treatments that only work for specific genetic mutations, this approach could treat all forms of DMD. The drug has already shown strong results in disease models and passed a Phase 1 safety trial in healthy volunteers in 2012. This grant from the Technology Strategy Board will fund the next step: testing SMTC1100 in patients to establish whether it actually works in people. If the trials succeed, SMTC1100 could become the first disease-modifying therapy for DMD, offering a treatment option for every boy and young man with the condition, regardless of their specific genetic mutation. That would change the outlook for a disease that currently has no effective medical intervention.