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Mivelsiran (ALN-APP)

by Alnylam PharmaceuticalsUS

Stop amyloid at its source by switching off the gene that makes it, rather than clearing plaques after they form, first in cerebral amyloid angiopathy, where amyloid in brain vessels causes bleeds and dementia.

ProtoStage 2 of 5

Phase 2 in cerebral amyloid angiopathy fully enrolled (data 2028); Down syndrome Alzheimer's trial started.

Updated 15 Jul 2026Checked 11 Oct0 updates this week

Milestones

Next · cAPPricorn-1 placebo-controlled portion completes
  1. First-in-human phase 1 startsFeb 2022Complete.
  2. FDA lifts partial hold: multiple doses up to 180 mg every 6 monthsFeb 2024Complete.
  3. cAPPricorn-1 phase 2 in CAA startsMay 2024Complete.
  4. cAPPricorn-1 enrollment complete; Down syndrome trial startsJul 2026Complete.
  5. cAPPricorn-1 placebo-controlled portion completesTarget Aug 2027Current milestone.
  6. cAPPricorn-1 initial resultsTarget 2028Not yet reached.

Most important, last 3 months

Upcoming

  1. Aug 2027cAPPricorn-1 placebo-controlled portion completesin about 42 weeks (next)
  2. 2028cAPPricorn-1 initial results
Primer