BioCryst enrolls first patient in proof-of-concept trial for oral Factor D inhibitor BCX10013

BIOCRYST PHARMACEUTICALS INC

First patient enrolled in open-label, multicenter dose-escalation trial of BCX10013 in up to 15 PNH patients.
Primary endpoints: treatment-emergent AEs, graded lab abnormalities; secondary: LDH, hemoglobin, transfusion-free rate.
If best-in-class profile confirmed in PNH, company plans to advance to pivotal program in renal complement diseases like IgAN.
Trial conducted in countries without standard-of-care complement inhibitor therapy; NCT06100900.

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