Sarepta cuts its operating loss to $9m, but can new RNA drugs offset Elevidys’ decline?
Sarepta narrows its Q3 operating loss to $9 million as Elevidys sales fall 37%. Examine safety, RNA pipeline data and guidance.
Pharma & Biotech
Pharma & Biotech
Arrowhead Pharmaceuticals secures NHS funding recommendation for REDEMPLO in familial chylomicronaemia syndrome
REDEMPLO gains NHS backing for genetically confirmed FCS in England and Wales, following strong triglyceride reductions in the PALISADE trial.
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
Sarepta’s RNA therapy reaches the nucleus in myotonic dystrophy models. Can it restore muscle function?
Sarepta has reported up to 80% knockdown of DMPK RNA in both the nucleus and cytoplasm of patient-derived muscle cells, alongside correction of abnormal RNA splicing in preclinical models. The findings address a central delivery challenge in myotonic dystrophy type 1, while an ongoing Phase 1/2 trial will determine whether the mechanism translates into patient
Pharma & Biotech
Arrowhead has enrolled 70 patients in a rare cholesterol trial, but can zodasiran clear the Phase 3 bar?
Arrowhead completes Phase 3 YOSEMITE enrollment as zodasiran faces a crucial test of LDL cholesterol lowering in patients with severe HoFH.
Pharma & Biotech
SHASTA-3 and SHASTA-4 results support Arrowhead Pharmaceuticals’ planned plozasiran FDA filing
Arrowhead’s Phase 3 SHASTA data show deep triglyceride reductions and fewer pancreatitis events, supporting a planned 2026 U.S. filing.
Pharma & Biotech
Is Arrowhead’s plozasiran becoming the RNAi drug to watch in extreme triglyceride disorders?
Arrowhead’s plozasiran data ease dosing concerns in complex patients, but Phase 3 results will decide its broader triglyceride market role.
Pharma & Biotech
What Madrigal Pharmaceuticals, Inc.’s siRNA deal signals for the future of MASH treatment
Can Madrigal Pharmaceuticals, Inc. reshape MASH treatment with ARO-PNPLA3? Discover what this precision strategy means for the future.
Pharma & Biotech
Arrowhead just unlocked Australia for REDEMPLO, but the bigger RNAi test starts now
Arrowhead has regulatory momentum in rare lipid disease. The harder test is turning REDEMPLO approval into durable global uptake.
Pharma & Biotech
Arrowhead’s plozasiran clears CHMP: what a positive opinion for FCS means for siRNA’s regulatory arc in Europe
Plozasiran clears CHMP but the real test is reimbursement. Europe's HTA bodies will now decide whether clinical FCS diagnosis is enough.