Tag: antisense oligonucleotide
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Novartis’ Pelacarsen Fails Phase 3 Lp(a)HORIZON Despite Lowering Lp(a)
Novartis and Ionis reported that pelacarsen lowered Lp(a) but failed to significantly reduce 4-point MACE in the Phase 3 Lp(a)HORIZON trial, raising new questions about the clinical validity of Lp(a) lowering.
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Why Lowering Lp(a) Wasn’t Enough: What Pelacarsen’s Phase 3 Failure Means
Pelacarsen targets LPA mRNA to reduce apo(a) production and circulating Lp(a), but the Phase 3 Lp(a)HORIZON result showed that molecular target engagement did not translate into fewer major cardiovascular events.
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FDA Approves Ionis’ Zanvastro as the First Treatment for Alexander Disease
The FDA approved Ionis’ Zanvastro as the first treatment for Alexander disease, validating GFAP-targeted antisense therapy in an ultra-rare neurological disorder.
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How Zilganersen Targets Alexander Disease by Reducing GFAP RNA
Zilganersen is a GFAP-targeting antisense oligonucleotide that reduces GFAP production upstream of protein accumulation, astrocyte stress and white-matter injury in Alexander disease.