Ae­glea's en­gi­neered en­zyme flops out­comes test in rare meta­bol­ic dis­ease. Is re­duc­ing a key bio­mark­er enough for an ap­proval?

Austin’s Ae­glea Bio­Ther­a­peu­tics is hop­ing to blaze a path re­duc­ing a key amino acid bio­mark­er for a rare wast­ing meta­bol­ic dis­ease with its en­gi­neered en­zyme ap­proach. But with a piv­otal out­comes read­out bod­ing poor­ly for the drug’s ben­e­fit, Ae­glea hopes it still has enough da­ta on hand to catch the FDA’s eyes.

Ae­glea’s pegzi­larginase, a re­com­bi­nant en­zyme de­signed to de­grade the amino acid argi­nine, hit its pri­ma­ry end­point re­duc­ing argi­nine lev­els in the blood over place­bo in pa­tients with the rare meta­bol­ic dis­ease arginase 1 de­fi­cien­cy (ARG1-D), the Austin-based biotech said Mon­day.

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