Two years ago, when investigators for Daiichi Sankyo gave its experimental Duchenne muscular dystrophy drug to the very first patient in a clinical trial, the company confidently hailed the move as a big step toward a marketing approval and launch in 2020. But DMD drug development isn’t that simple.
Daiichi today said that DS-5141 — designed to spur the production of incomplete but functional dystrophin by inducing exon 45 skipping through dystrophin messenger RNA — failed an early Phase I/II study. But they’re not about to throw in the towel.
Dystrophin protein expression was “partially identified, but was not be clearly detected as a whole,” researchers said. They added that a secondary endpoint — the production of messenger RNA with exon 45 skipping of the dystrophin gene — did hit. And now they’ll keep studying the drug, which was given Japan’s Sakigake designation — read fast-track status with a shot at cutting the regulatory review time in half — last year.
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