Ox­ford spin­out rais­es $113M in bid to beat Sarep­ta's Ex­ondys 51 in DMD. But will we ever know if any of the drugs work?

It’s been five years since the FDA de­cid­ed to ap­prove Sarep­ta’s Duchenne mus­cu­lar dy­s­tro­phy drug Ex­ondys 51 on a sliv­er of bi­o­log­i­cal ev­i­dence, and still there are lit­tle da­ta to show the drug can ac­tu­al­ly slow pa­tients’ de­cline.

That pauci­ty, though, hasn’t scared off in­vestors from in­vest­ing in com­pa­nies try­ing to treat the dis­ease with the same ap­proach. On the con­trary, it has giv­en such com­pa­nies a clear path to gain ap­proval af­ter min­i­mal test­ing, while the $400-plus mil­lion in sales Sarep­ta has raked in from Ex­ondys 51 has shown just how large a mar­ket there could be for any com­pa­ny that can show more bi­o­log­i­cal ef­fect than the large biotech.

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