Tag: Ionis Pharmaceuticals
-
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.
-
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.
-
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.
-
AstraZeneca Keeps Its $80 Billion Goal Despite a Major Trial Setback
AstraZeneca reaffirmed its $80 billion revenue ambition after reporting solid second-quarter results. Strong oncology and rare disease sales helped restore confidence following the failure of a major Wainua trial.