The Generative Brain

Regeneron Pharmaceuticals

US biopharma company sponsoring ALN-6457, the PRNP-targeting program from its 2019 collaboration with Alnylam on RNAi therapeutics for eye and CNS targets; the Phase 1/2 prion trial is registered but not yet recruiting.

Key facts

  • Phase 1/2 open-label trial with extended follow-up of multiple doses of ALN-6457 in symptomatic prion disease; sponsor Regeneron Pharmaceuticals; status not yet recruiting; estimated start 2026-10-23; estimated primary completion 2031-02-27; also registered in the EU CTIS (2026-526727-21-00); no expanded access.
  • Sponsor of NCT07832370, a Phase 1/2 open-label trial with extended follow-up of ALN-6457 (other name ALN-PRNP) in symptomatic prion disease, not yet recruiting with an estimated start of 23 October 2026.
  • The 2019 global collaboration with Alnylam covers RNAi therapeutics for targets in the eye and central nervous system and carried a USD 400 million upfront payment from Regeneron.
  • Under the collaboration terms a licensee pays development and commercial milestones totaling up to USD 150 million for each collaboration product, per Regeneron's FY2019 annual report.
  • A 2019 trade-press account named prion disease among the neurodegenerative targets considered suitable for siRNA therapy in the alliance.
  • An aggregator lists planned enrollment of 60 for the ALN-6457 trial and labels the drug a small molecule, which conflicts with the Alnylam program prefix and is unconfirmed.

Timeline

  1. 2019Alnylam collaboration begins.
  2. 2026Trial first registered.
  3. 2026Estimated start of the ALN-6457 trial.
Sources for this page