News brief­ing: Io­n­is reads out pos­i­tive da­ta for prekallikrein-tar­get­ing drug; PPD col­lab­o­rates on lu­pus re­search

In a com­pas­sion­ate-use pi­lot study for hered­i­tary an­gioede­ma (HAE), Io­n­is’ PKKRx and PKK-LRx were shown to re­duce the rate of break­through at­tacks in two pa­tients.

PKK-LRx is cur­rent­ly in Phase II de­vel­op­ment to treat the rare ge­net­ic dis­ease, which caus­es rapid and painful in­flam­ma­tion at­tacks in the hands, feet, limbs, face, ab­domen, lar­ynx and tra­chea. The lig­and-con­ju­gat­ed an­ti­sense (LI­CA) oligonu­cleotide is de­signed to re­duce the pro­duc­tion of prekallikrein (PKK).

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