Neu­ro­crine breaks out proof-of-con­cept da­ta for CAH drug; Ake­bia, MT­PC work to­ward Japan­ese NDA af­ter ane­mia drug scores in twin PhI­I­Is

→ More than a month af­ter its big tick­et deal with Voy­ager Ther­a­peu­tics $VY­GR, Neu­ro­crine Bio­sciences $NBIX an­nounced pos­i­tive in­ter­im da­ta from a Phase II proof-of-con­cept study eval­u­at­ing its ex­per­i­men­tal drug, NBI-74788, in adults with a ge­net­ic dis­or­der that re­sults in an en­zyme de­fi­cien­cy that al­ters the pro­duc­tion of adren­al steroids called clas­sic con­gen­i­tal adren­al hy­per­pla­sia (CAH). Da­ta showed a re­duc­tion of at least 50% from base­line in 17-hy­drox­yprog­es­terone (17-OHP) — used as a di­ag­nos­tic bio­mark­er in CAH — in more than half the treat­ed pa­tients. “But what does a 50% ‘re­spon­der’ rate mean in clin­i­cal terms? This study wasn’t re­al­ly de­signed to an­swer this ques­tion, and while we don’t know the pa­tients’ 17-OHP base­line val­ues in this study, our guess from look­ing at oth­er CAH tri­als (ahead of the da­ta to­day) is that it was prob­a­bly very high. Thus, it is un­clear to us to­day how these bio­mark­er changes will or will not trans­late in­to an im­prove­ment in pa­tient out­comes,” Stifel’s Paul Mat­teis wrote.

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