Armed with a batch of pos­i­tive bio­mark­er da­ta, a rare dis­ease play­er reaps $88M for the next big push in the clin­ic

Omega Funds and Abing­worth teamed up to lead an $88 mil­lion round for Spruce, which is pri­mar­i­ly fo­cused on de­vel­op­ing tildac­er­font to treat clas­sic con­gen­i­tal adren­al hy­per­pla­sia.

The da­ta they came up with was based on pa­tients who lack en­zyme 21-hy­drox­y­lase, which throws a mon­key wrench in the pro­duc­tion of cor­ti­sol while in­creas­ing pro­duc­tion of male sex hor­mones to dan­ger­ous lev­els. Their drug — a CRF-1 re­cep­tor an­tag­o­nist — tar­gets ACTH and a down­stream bio­mark­er dubbed A4, and in the Phase IIa in­ves­ti­ga­tors tracked a mean re­duc­tion of 74% for ACTH, 82% for 17-OHP and 55% for A4.

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