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Drug Development.

Sharon Cohen1, Simon Ducharme2, Jared R Brosch3

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Mivelsiran, an RNA interference therapeutic, showed good safety and reduced amyloid precursor protein in early Alzheimer's patients. Multiple doses further lowered sAPPβ, supporting its potential for Alzheimer's disease and cerebral amyloid angiopathy treatment.

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Area of Science:

  • Neuroscience
  • Pharmacology
  • Genetics

Background:

  • Mivelsiran is an investigational RNA interference (RNAi) therapeutic targeting the central nervous system (CNS).
  • Previous studies showed single mivelsiran doses effectively lowered amyloid-beta precursor protein (APP).
  • This study reports interim safety and pharmacodynamic data from a Phase 1 trial in early-onset Alzheimer's disease (EOAD) patients.

Purpose of the Study:

  • To evaluate the safety and tolerability of single ascending doses (SAD) and multiple ascending doses (MAD) of mivelsiran in EOAD patients.
  • To assess the pharmacodynamic effects of mivelsiran on cerebrospinal fluid (CSF) soluble APP beta (sAPPβ) levels.
  • To provide initial clinical data on a CNS-targeting RNAi therapeutic for Alzheimer's disease.

Main Methods:

  • Patients with EOAD were randomized to receive single intrathecal doses of mivelsiran (25-100mg) or placebo.
  • A subset of patients received open-label, multiple ascending doses of mivelsiran (50mg every 6 months) after washout.
  • Safety was assessed by adverse event (AE) frequency, and pharmacodynamics by changes in CSF sAPPβ levels.

Main Results:

  • In SAD cohorts, most AEs were mild/moderate; peak CSF sAPPβ reduction of -84.5% at Month 1 was sustained through Month 10 (-61.1%).
  • In the MAD cohort, no serious or severe AEs were reported.
  • After the first mivelsiran dose in MAD, CSF sAPPβ decreased by -63.7%; a second dose led to further reduction (-83.8%).

Conclusions:

  • Single and multiple doses of mivelsiran were generally well tolerated in EOAD patients.
  • Mivelsiran demonstrated robust, durable, and dose-dependent reductions in CSF sAPPβ.
  • Results support further investigation of mivelsiran for Alzheimer's disease and cerebral amyloid angiopathy.