Ear­ly func­tion­al da­ta on limb gir­dle pa­tients buoy ex­pec­ta­tions for Sarep­ta's gene ther­a­py

Sarep­ta’s lousy sum­mer has come to a close — with some en­cour­ag­ing fresh func­tion­al da­ta from its limb-gir­dle mus­cu­lar dy­s­tro­phy (LGMD) gene-ther­a­py pro­gram.

In Feb­ru­ary, the drug­mak­er di­vulged da­ta from three pa­tients in the first co­hort of an open-la­bel Phase I/II study test­ing the use of an ex­per­i­men­tal gene ther­a­py — MYO-101 (now called SRP-9003) — in LGMD pa­tients aged four to 15 years.

Da­ta showed the ther­a­py re­ju­ve­nat­ed the pro­duc­tion, by an av­er­age of 51%, of be­ta-sarco­gly­can — the pro­tein re­quired for mus­cle func­tion that is miss­ing in this pa­tient pop­u­la­tion. On Fri­day, Sarep­ta broke out da­ta that showed that the in­duced uptick in be­ta-sarco­gly­can did, in fact, trans­late to func­tion­al im­prove­ments, nine months post-in­fu­sion.

Based on the nat­ur­al his­to­ry of the dis­ease, these changes were def­i­nite­ly not ex­pect­ed at these time points, Sarep­ta ex­ec­u­tives un­der­scored on a con­fer­ence call with an­a­lysts.

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