Summit Pursues A Different Approach to Treating Duchenne

RARECast

Duchenne Muscular Dystrophy, a rare genetic disease, causes progressive muscle wasting that slowly robs people of abilities and leads to death. In recent years, much attention has focused on the use of antisense oligonucleotides to bypass defective portions of the exon that codes for the dystrophin gene to restore its production. Dystrophin is a protein that is essential to healthy muscle. Summit Therapeutics is taking a different approach. Instead of restoring dystrophin, Summit is developing a drug that activate utrophin, a related protein that serves a similar function to dystrophin during fetal development, but then shuts off. We spoke to Glyn Edwards, CEO of Summit, about the company’s utrophin activator ezutromid, its licensing deal with Sarepta Therapeutics, and why, unlike the exon-skipping drugs that target specific subpopulaitons of Duchenne patients, ezutromid could provide benefits to patients broadly.