Merck KGaA has agreed to acquire Silence Therapeutics for approximately $1.3 billion in equity value, offering $20 per ADS. The transaction centers on divesiran, an RNA interference therapy that achieved an 88% response rate in Phase 2 polycythemia vera testing.
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.
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
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Plozasiran clears CHMP but the real test is reimbursement. Europe's HTA bodies will now decide whether clinical FCS diagnosis is enough.