FDA gibt Freigabe des Prüfantrags für ein neues Arzneimittel für die AMT-260-Gentherapie zur Behandlung refraktärer mesialer Temporallappenepilepsie bekannt. MK 362 Mio. $ Wird derzeit deutlich unter Cash gehandelt https://www.uniqure.com/investors-media/press-releases
Presented promising clinical update from U.S. and European Phase I/II trials of AMT-130 in Huntington’s disease; Up to three years of follow-up data to be presented in mid-2024; Regulatory interactions and clarity on potential strategies for clinical development expected in 2024
~ Announced FDA clearance of two Investigational New Drug (IND) applications; Initiation of Phase I/II clinical trials in mesial temporal lobe epilepsy (mTLE) and Fabry disease, in addition to SOD1-ALS, are expected in the first half of 2024
Announced strategic reorganization to advance multiple clinical-stage programs and deliver $180 million of cost savings over the next three years
- On track to initiate FDA interaction regarding AMT-130 in second quarter of 2024 and provide a clinical update from the Phase I/II trials in mid-2024
- Clinical trial initiation for Fabry disease on track to begin in second quarter of 2024, followed by refractory mesial temporal lobe epilepsy and SOD1-ALS in third quarter of 2024
- Comprehensive review of operations and options to reduce expenses underway and expected to be completed in mid-2024
- Type B meeting scheduled with the FDA in the fourth quarter of 2024 to initiate discussions regarding a potential expedited clinical development pathway for AMT-130 in Huntington’s disease