OnlyBioLover
$DYN now in here. the sept 8 dump came from novartis missing the primary endpoint in harbor, a dm1 trial. fair point, dyne’s dm1 program uses the same delivery idea and the same vhot endpoint, so that read-across stays open until achieve topline in q1 2027. but the jan 21 pdufa is a different drug in a different disease: z-rostudirsen, exon 51 dmd, priority review. deliver showed 5.46% muscle-adjusted dystrophin, 2.87% unadjusted, p<0.0001, functional data nominal on 24 vs 8. exondys 51, approved for the same patients, shows 0.93% on its label. cash 898.5m at june 30 plus about 405m net from july, runway guided into q2 2028. risk i’d put first: fda’s view on dystrophin as a surrogate after sarepta’s confirmatory misses. wms sept 30 next. not advice.
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