Ionis Pharmaceuticals shares slide as eplontersen misses key heart disease endpoint
See why the CARDIO-TTRansform miss complicates eplontersen’s ATTR-CM path and tests gene silencing on top of stabilizer therapy.
Pharma & Biotech
Pharma & Biotech
Roche ends two Huntington’s disease programmes after separate clinical setbacks
Roche Holding AG has discontinued development of tominersen and RG6496, removing two investigational huntingtin-lowering medicines from its neurology pipeline after separate setbacks emerged almost simultaneously. The Phase 2 GENERATION HD2 trial showed that tominersen lowered important biological markers but did not meaningfully slow clinical decline, while the Phase 1 POINT-HD study of RG6496 was halted
Pharma & Biotech
Ionis fills pivotal Angelman syndrome cohort, but can obudanersen confirm its early promise?
Discover why Ionis' REVEAL enrollment turns obudanersen into a decisive Phase 3 test for Angelman syndrome treatment.
Pharma & Biotech
Ionis Pharmaceuticals moves beyond rare disease as Tryngolza enters the wider triglyceride market
Tryngolza is no longer confined to the rare familial chylomicronemia syndrome market. Its broader United States approval gives clinicians the first therapy specifically shown to reduce acute pancreatitis risk in adults with severe hypertriglyceridemia, although questions surrounding dose selection, hepatic monitoring, reimbursement and long-term use will influence how quickly the treatment changes practice.
Pharma & Biotech
Why Praxis’ latest FDA win could matter far beyond one genetic epilepsy program
Find out how Praxis’ FDA Breakthrough Therapy Designation for elsunersen could reshape SCN2A-DEE epilepsy treatment and ASO development.
Pharma & Biotech
Can salanersen help Biogen defend its rare neurology franchise beyond Spinraza?
Find out how Biogen’s salanersen could reshape spinal muscular atrophy treatment after FDA breakthrough therapy status.
Pharma & Biotech
Can Arrowhead Pharmaceuticals expand plozasiran without the Ionis patent fight becoming a commercial overhang?
Arrowhead Pharmaceuticals is advancing plozasiran fast, but can it overcome the Ionis patent dispute? Read the deeper legal and commercial analysis.
Pharma & Biotech
FDA fast track for Argo Biopharma’s BW-20805 siRNA puts HAE’s next competitive battleground in focus
Argo Biopharmaceutical Co., Ltd. (Argo Biopharma), a Shanghai-based clinical-stage RNA interference therapeutics company, has received Fast Track Designation from the U.S. Food and Drug Administration for BW-20805, its investigational small interfering RNA therapy targeting prekallikrein for the prevention of hereditary angioedema attacks. The designation comes as the company runs a global Phase II open-label study