BridgeBio’s long-term limb-girdle muscular dystrophy data; QHP buys a CDMO

Bridge­Bio shares longer-term PhII da­ta in limb-gir­dle: Bridge­Bio said that pa­tients with limb-gir­dle mus­cu­lar dy­s­tro­phy type 2I/R9 treat­ed with BBP-418 saw “sta­bi­liza­tion in NSAD scores and am­bu­la­to­ry mea­sures” over 21 months in the 14-pa­tient Phase II study. The com­pa­ny is al­ready run­ning a Phase III study that it hopes to use for an ac­cel­er­at­ed ap­proval of the limb-gir­dle treat­ment, with gly­co­sy­lat­ed α-dy­s­tro­gly­can lev­els at 12 months as a sur­ro­gate end­point. The con­fir­ma­to­ry re­sults from the study would be a clin­i­cal test for limb-gir­dle dy­s­tro­phies called the North Star As­sess­ment for Dys­fer­linopa­thy, or NSAD. — Lei Lei Wu

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