Brett Monia, Ionis CEO

Slammed with fresh clin­i­cal fail­ure, Bio­gen and Io­n­is dis­card ear­ly-stage ALS can­di­date

Months re­moved from a late-stage set­back on the amy­otroph­ic lat­er­al scle­ro­sis front, Bio­gen and Io­n­is are scrap­ping a sep­a­rate can­di­date af­ter it, too, yield­ed dis­ap­point­ing clin­i­cal re­sults.

BI­IB078, an an­ti­sense oligonu­cleotide specif­i­cal­ly de­signed for a ge­net­ic form of the dis­ease known as C9orf72-as­so­ci­at­ed ALS, did not show clin­i­cal ben­e­fit in a Phase I study. While it seemed to pass the pri­ma­ry end­point on safe­ty and tol­er­a­bil­i­ty, the drug didn’t meet any of the sec­ondary end­points — leav­ing lit­tle to sal­vage.

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