Brett Monia, Ionis Pharmaceuticals CEO

Io­n­is presents piv­otal rare dis­ease da­ta, builds to­ward first so­lo launch: #ACC24

As Io­n­is gears up to launch its first whol­ly-owned med­i­cine, re­searchers fleshed out its Phase 3 suc­cess in a rare ge­net­ic dis­ease Sun­day morn­ing.

The Cal­i­for­nia-based com­pa­ny ex­pand­ed on the piv­otal tri­al win for olezarsen, which hit its pri­ma­ry end­point in Sep­tem­ber. In the new da­ta pre­sent­ed at the an­nu­al Amer­i­can Col­lege of Car­di­ol­o­gy meet­ing, Io­n­is said olezarsen at the 80 mg dose low­ered triglyc­eride lev­els by a place­bo-ad­just­ed 44% af­ter six months, good for a p-val­ue of p<0.001.

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