Our Programs

Complement-Mediated Diseases

BioCryst discovered and is developing BCX9930, a novel, oral, potent and selective small molecule Factor D inhibitor that could offer a significant advance in therapy for patients with paroxysmal nocturnal hemoglobinuria (PNH) and other complement-mediated diseases.

The complement system is part of the body’s natural immune system and is responsible for helping the body eliminate microbes and damaged cells. It is comprised of proteins that are primarily produced in the liver and circulate in the blood. Once activated, the complement system stimulates inflammation, phagocytosis and cell lysis. Excessive or uncontrolled activation of the complement system can cause severe, and potentially fatal, immune and inflammatory disorders.

The complement system comprises biological cascades of amplifying enzyme cleavages involving more than 30 proteins and protein fragments and may be activated through three pathways:

  1. Classical pathway (initiated by antibody-antigen complexes)
  2. Lectin pathway (initiated by microbial surfaces)
  3. Alternative pathway (constitutively active)

 

The alternative pathway also provides a critical amplification loop for all three pathways, regardless of the initiating mechanism. Factor D is an essential enzyme in the alternative pathway, thus making Factor D an attractive target to address complement-mediated diseases.

Clinical Program

BCX9930 is currently being evaluated in clinical trials for the treatment of complement-mediated diseases.

REDEEM-1 is a randomized, open-label, active comparator-controlled study of the efficacy and safety of oral BCX9930 monotherapy in PNH patients with an inadequate response to a C5 inhibitor. Learn more about REDEEM-1.

REDEEM-2 is a randomized, placebo-controlled study to evaluate the efficacy and safety of oral BCX9930 as monotherapy versus placebo in PNH patients not currently receiving complement inhibitor therapy. Learn more about REDEEM-2.

BioCryst is also preparing to initiate a proof-of-concept trial of BCX9930 in renal complement-mediated diseases. The trial will be a basket study to evaluate BCX9930 for the potential to treat patients with C3 glomerulopathy, IgA nephropathy and primary membranous nephropathy.

BCX9930 is an investigational treatment and has not been deemed safe and effective by the FDA.

Clinical Program

BCX9930 is currently being evaluated in clinical trials for the treatment of complement-mediated diseases.

REDEEM-1 is a randomized, open-label, active comparator-controlled study of the efficacy and safety of oral BCX9930 monotherapy in PNH patients with an inadequate response to a C5 inhibitor. Learn more about REDEEM-1.

REDEEM-2 is a randomized, placebo-controlled study to evaluate the efficacy and safety of oral BCX9930 as monotherapy versus placebo in PNH patients not currently receiving complement inhibitor therapy. Learn more about REDEEM-2.

BioCryst is also preparing to initiate a proof-of-concept trial of BCX9930 in renal complement-mediated diseases. The trial will be a basket study to evaluate BCX9930 for the potential to treat patients with C3 glomerulopathy, IgA nephropathy and primary membranous nephropathy.

BCX9930 is an investigational treatment and has not been deemed safe and effective by the FDA.