uniQure N.V. QURE
uniQure N.V. (Nasdaq: QURE) is a CNS/neurology/psychiatry drug developer. Its lead program, AMT-130, is in Phase 2 for Huntington's disease.
Upcoming catalysts
No catalyst is dated to the next 30 days or to an FDA action date.
Pipeline
| Program | Indication | Modality | Stage |
|---|---|---|---|
| AMT-130 | Huntington's disease | gene therapy | Phase 2 |
| AMT-260 | Refractory mesial temporal lobe epilepsy | gene therapy | Phase 1 |
| AMT-191 | Fabry disease | gene therapy | Phase 1 |
| AMT-162 | SOD1-ALS | gene therapy | Phase 1 |
Recent results and decisions
- Sep 29, 2026mixed 48-month high-dose (n=12) vs updated Enroll-HD B external control: primary endpoint cUHDRS showed 44% slowing but not statistically significant (p=0.144); TFC 61% slowing (nominal p=0.008).
the primary endpoint of cUHDRS showed 44% slowing of disease progression (non-significant p=0.144) and TFC showed a 61% slowing of disease progression (nominal p=0.008)
Filing - Sep 29, 2026positive Updated 36-month analysis of all 15 high-dose patients vs updated external control: 80% slowing on cUHDRS (nominal p=0.005) and 67% on TFC (nominal p=0.011); not part of the submitted BLA.
cUHDRS showed 80% slowing of disease progression compared to the updated external control (nominal p=0.005).
Filing - Sep 2, 2026submitted BLA for accelerated approval of AMT-130 submitted to FDA, supported by the 3-year Phase I/II analysis; priority review requested.
today announced the submission of a Biologics License Application (BLA) to the United States (U.S.) Food and Drug Administration (FDA) for the accelerated approval of ifezuntirgene inilparvovec (AMT-130), an investigational gene therapy for the treatment of Huntington's disease.
Filing
10 more on record.
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
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Other phase 2 CNS/neurology/psychiatry 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.