The data, which are the first data with BCX9930 in patients with C3G, are being presented in a poster session at the 18th European Meeting on Complement in Human Disease, which is being held in
'The analyses from this study add to the body of evidence that show Factor D inhibition can correct dysregulation of the alternative pathway and has the potential to be a meaningful treatment approach for multiple complement-mediated diseases. The data are consistent using both commercially available assays and sophisticated proprietary assays our team has developed, which we can also utilize with our next-generation pipeline programs to quickly assess complement activation in vivo early in development,' said Dr.
BioCryst EMCHD 2022 Presentation Highlights
BCX9930, an Oral Factor D Inhibitor, Suppresses Complement Alternative Pathway C3 Convertase Activity in vitro, and in Patients with Complement 3 Glomerulopathy (Poster #80);
The study assessed the effects of oral BCX9930 on AP activity in ex vivo activated serum from healthy volunteers (n=16) and C3G patients (n=6) who participated in a phase 1, open-label study to evaluate the pharmacokinetics and pharmacodynamics of a single dose of BCX9930.
One assay was used to assess the ability of BCX9930 to inhibit formation of AP C3 convertase in vitro (Elisa-detecting properdin (P)-bound C3 convertase), two assays were used to assess the ability of BCX9930 to inhibit AP complement activity ex vivo (AP Wieslab kit and multiplex assays), and another assay was used to assess the ability of BCX9930 to inhibit formation of AP C3 convertase ex vivo (immunofixation electrophoresis).
The analyses found that a single oral dose (600 mg) of BCX9930:
Demonstrated >99 percent (median) suppression of the AP, and that >98 percent (median) suppression was maintained for 24 hours post-dosing, in C3G patients.
Achieved rapid (within two hours) and maximal (median >99 percent relative to pre-dose levels) suppression of the generation of C3a, a product of C3 convertase activity, in both healthy volunteers and C3G patients.
These data support further development of Factor D inhibitors for the treatment of complement-mediated diseases, including C3G and other diseases driven by dysregulation of the AP.
In the study, a single dose of BCX9930 600 mg was generally safe and well-tolerated. Based on prior work with BCX9930, the proposed clinical dose of 400 mg (bid) provides a similar level of AP suppression as the 600 mg single dose used in this study in C3G patients.
Currently, BioCryst is conducting the RENEW proof-of-concept basket study evaluating BCX9930 in C3G, immunoglobulin A nephropathy (IgAN) and primary membranous nephropathy (PMN), all rare renal diseases caused by dysregulation of the alternative pathway.
BioCryst is also currently conducting the REDEEM-1 and REDEEM-2 pivotal trials evaluating BCX9930 in paroxysmal nocturnal hemoglobinuria (PNH).
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