Trial watch 16 -Factor D complement inhibitor.

One of the ways to find out which drugs have a potential for aHUS treatment is to look at trials for PNH treatment. One such trial is listed on Clinical Trials database is for BCX9930. It has been developed by the Pharmaceutical Company BioCryst,

This technology is a two tablets a day option. Full details about the trial can be seen at this link.

This is a novel complement inhibitor as it stops little known Complement Factor D from working. Factor D is needed to “cleave” ( slice) Factor B in two, and one of those bits called , “Bb” goes on to “cleave”to (attach to) C3b to create the C3 convertase C3bBb, which when another portion of C3b is added creates a C5 convertase, C3bBbC3b,which can on go to trigger the C5b-9 membrane attack complex. And for those susceptibl results in aHUS.

The trial is in two parts. Part 1 is double blind trial where half of patients will receive Factor D and half a placebo for 12 weeks. In Part 2 all patients in the trial will receive BCX9930.

Recruitment to the trial has been paused while BioCryst investigate a “creatinine” adverse event reported in some of the recruited patients. More information can be found at this link.

Article No. 525

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