The Generative Brain

ALN-6457 (NCT07832370)

Registry record NCT07832370: Regeneron's Phase 1/2 open-label trial of ALN-6457 in adults with symptomatic prion disease, not yet recruiting, estimated start 23 October 2026 and estimated completion 27 February 2031, also registered in the EU. Sites and eligibility are not yet public.

Status as of 2026-10-16. Please check the study registry for the latest position.

Key facts

  • Context: a 2019 trade-press account of the Regeneron-Alnylam alliance named prion disease among the neurodegenerative targets considered suitable for siRNA therapy, so a prion program from this alliance was foreseeable.
  • Origin: the sponsor and Alnylam formed a global RNAi collaboration in 2019 covering eye and CNS targets with a USD 400 million upfront payment, which is the context for the ALN-PRNP program name.
  • Official title: Phase 1/2 open-label trial with extended follow-up to evaluate safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of multiple doses of ALN-6457 in participants with symptomatic prion disease.
  • Sponsor Regeneron Pharmaceuticals; study id ALN-6457-PD-25129; EU CTIS 2026-526727-21-00; class industry.
  • Status not yet recruiting; estimated start 2026-10-23; estimated primary and study completion 2031-02-27; no expanded access.
  • Arms: dose escalation and cohort expansion, both receiving ALN-6457 per the protocol (other name ALN-PRNP).
  • Primary outcomes: adverse events through Week 156; percent change in plasma NfL through Week 130.
  • Secondary outcomes: CSF PrP and change, drug concentrations in plasma and CSF through Week 130, and overall survival at Week 52.
  • An aggregator reports 60 planned participants and calls the drug a small molecule; neither is confirmed on the registry text read.
  • Registry first submitted 2026-09-16, one month before the estimated start.

Timeline

  1. 2026Record first submitted.
  2. 2026Estimated start.
  3. 2031Estimated completion.
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