Heart in­flam­ma­tion emerges in a Sarep­ta Duchenne pa­tient, cloud­ing an oth­er­wise pos­i­tive read­out

Hav­ing al­ready se­cured sev­er­al ap­provals for Duchenne mus­cu­lar dy­s­tro­phy drugs, Sarep­ta aimed to cap­i­tal­ize on its lead in the field with a new da­ta read­out Wednes­day. And while many of the hard num­bers are pos­i­tive, a new side ef­fect is putting a damper on the mood.

Sarep­ta re­port­ed Wednes­day that its ex­per­i­men­tal gene ther­a­py, dubbed SRP-9001, sig­nif­i­cant­ly im­proved pa­tients’ mo­tor func­tion af­ter one year com­pared to a con­trol. The da­ta came from a 20-pa­tient co­hort of boys aged four to sev­en years old and al­so passed sev­er­al key sec­ondary tests. The biotech al­so re­vealed four-year da­ta from a small, four-pa­tient co­hort in an ear­li­er study sug­gest­ing the treat­ment’s ef­fect can re­main durable over that time.

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