Ionis reports 95% fewer HAE attacks after two years. Can DAWNZERA defend its dosing advantage?
Ionis reports sustained DAWNZERA HAE attack reductions over two years. See what the data reveal about dosing, safety and commercial growth.
Pharma & Biotech
Pharma & Biotech
Novartis lowered a major inherited heart-risk marker by 78%. Why did patients not benefit?
Novartis cut Lp(a) by 78% with pelacarsen but failed to reduce cardiovascular events, raising the stakes for Amgen and Eli Lilly.
Pharma & Biotech
Roche and Ionis report 43.1% proteinuria reduction with sefaxersen in Phase 3 IgA nephropathy trial
Roche and Ionis report a 43.1% Phase 3 proteinuria reduction with sefaxersen. Two-year kidney function results remain the next major test.
Pharma & Biotech
Ionis TRYNGOLZA wins EU recommendation after 85% reduction in pancreatitis events
European regulators have recommended extending TRYNGOLZA to adults with fasting triglycerides of at least 880 mg/dL. The decision follows two Phase 3 trials demonstrating substantial triglyceride reductions and 85% fewer adjudicated acute pancreatitis events in a pooled analysis.
Pharma & Biotech
A simple injection cut a key Alzheimer’s protein by 73%. Can Arrowhead turn that into cognitive benefit?
Arrowhead Pharmaceuticals has reported up to 73% mean reduction in cerebrospinal-fluid tau at Day 105 after subcutaneous ARO-MAPT treatment in healthy volunteers. The findings support central nervous system target engagement and a potential quarterly dosing schedule, but cognitive benefit and effects on brain tau pathology remain unproven.
Pharma & Biotech
Roche reports positive Phase III sefaxersen results as factor B competition intensifies in IgA nephropathy
Roche’s sefaxersen hits its Phase III IgAN endpoint with significant proteinuria reduction. See what the result means for regulators and competitors.
Features & Analysis
Ionis priced ZANVASTRO at $285,000 a dose. Can the first Alexander disease drug build a viable ultra-rare franchise?
FDA approval has transformed zilganersen from an experimental antisense programme into the first disease-modifying medicine for Alexander disease. For Ionis Pharmaceuticals, however, ZANVASTRO is also a test of whether years of RNA-targeted neurology research can translate into a sustainable independently commercialized franchise.
Pharma & Biotech
Ionis wins first FDA approval for Alexander disease. Can Zanvastro make an ultra-rare launch work?
Zanvastro finally gives Alexander disease a targeted therapy. Its next challenge is finding patients and funding a $1.14 million annual regimen.
Pharma & Biotech
Novartis pelacarsen fails 8,323-patient Phase 3 Lp(a)HORIZON cardiovascular trial
Novartis and Ionis say pelacarsen substantially reduced lipoprotein(a) but failed to reduce major cardiovascular events in the 8,323-patient Phase 3 Lp(a)HORIZON trial.
Pharma & Biotech
FDA approves Ionis ZANVASTRO as first disease-modifying treatment for Alexander disease
The FDA has approved Ionis Pharmaceuticals’ ZANVASTRO for pediatric and adult Alexander disease after a 54-patient study showed significant stabilization of gait at 61 weeks.