Drug Development, Setbacks

Daiichi Sankyo concedes an early setback on Duchenne MD drug, but doubles down on development

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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RAPS Convergence 2018

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Magenta Therapeutics Cambridge, MA
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