Scoring Pfizer v Sarepta: This round on Duchenne gene therapy goes to the little biotech, but the pharma Goliath has plenty of punch left
Pfizer this morning is presenting its first cut of the Phase Ib data from a small study of its mini-dystrophin gene therapy designed to cure Duchenne muscular dystrophy. And they’re dropping it straight into the middle of a well defined clinical battleground, with lines clearly drawn between the pharma giant and Sarepta, the little biotech with mighty big ambitions.
Sarepta — which posted impressive but variable results from its early-stage study last year — is coming out of this round scoring ahead of its giant rival. But by Pfizer’s own assessment, this is at worst a draw with data that looks much like Sarepta’s, with lots of potential for coming out ahead as they move toward a pivotal showdown.
Taken on its own, the early Pfizer efficacy data are positive, with clear signs of a dose-dependent response for the handful of evaluable DMD boys. On all key measures related to dystrophin expression, safety and perhaps assays used, though, Pfizer’s data has a worse overall profile when compared to the competition.
But that doesn’t mean they are out of the game.
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