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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