ProQR Announces Positive Phase 1 Target Engagement Data for AX-0810, Establishing First Clinical Validation of the Axiomer RNA Editing Platform
ProQR reported positive Phase 1 data for AX-0810, showing dose-dependent target engagement with up to an 8-fold increase in serum bile acids at the 6 mg/kg dose, significantly exceeding the 2-fold threshold. The drug demonstrated a favorable safety profile with no serious adverse events and a half-life of eight weeks, supporting the expected pharmacologic profile for sustained engagement.
Key figures
- Drug Name
- AX-0810
- Phase Of Trial
- Phase 1
- Half life weeks
- 8
- Total participants
- 33
- Active participants
- 24
- Placebo participants
- 9
- Doses evaluated mg kg
- [3,6]
- Max bile acid increase
- 8-fold
- Target engagement threshold
- 2-fold
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