ProQR Therapeutics N.V. PRQR
ProQR Therapeutics N.V. (Nasdaq: PRQR) is a GI/hepatology drug developer. Its lead program, AX-0810, is in Phase 1 for cholestatic diseases (NTCP target).
Upcoming catalysts
No catalyst is dated to the next 30 days or to an FDA action date.
Pipeline
| Program | Indication | Modality | Stage |
|---|---|---|---|
| AX-0810 | cholestatic diseases | RNA (siRNA/ASO/mRNA) | Phase 1 |
| AX-2402 | Rett syndrome (MECP2) | RNA (siRNA/ASO/mRNA) | Pre-clinical |
Recent results and decisions
- Sep 9, 2026started First participant dosed in the Phase 1 healthy-volunteer study of AX-0811 (Phase 1 start, so not a Phase 2+ trial_start).
today announced that the first participant has been dosed in the Phase 1 clinical trial of AX-0811 in healthy volunteers.
Filing - Apr 8, 2026positive Initial AX-0810 Phase 1 data show no safety signals and pharmacokinetics consistent with non-clinical models; target engagement data still pending.
Initial AX-0810 data demonstrate no safety signals and pharmacokinetics consistent with non-clinical models
Filing
For subscribers
- Cash runway against each catalyst, with the company's own runway statement
- Track record: every Phase 3 readout, FDA decision and guidance slip since 2014
- Expected move for each dated catalyst, from comparable past events
- Phase 2 evidence behind each Phase 3 readout
- Companies on the same target or class
- Full catalyst list with filing sentences, deals, registered trials, Excel export
Other phase 1 GI/hepatology companies
The free Monday brief
Last week's readouts and FDA decisions and the dated catalysts in the week ahead, each with its filing sentence.