uniQure Reports Data from Two P-I/II Studies of AMT-130 for Treating Huntington’s Disease
Shots:
- The P-I/II studies assessed AMT-130 in the US (N=26) & EU/UK (N=13) for Huntington’s disease. Of these 29 received high (n=12) & low doses (n=17), 10 had imitation surgery. Data (as of Mar 2024) from 21 of them was compared with a propensity-weighted external control group (n=154) at 24mos.
- Studies showed -0.2 vs -1.0 (high) & -0.7 vs -1.0 (low) mean changes in cUHDRS with 80% & 30% slowing of disease progression, respectively, plus 11% mean reduction in CSF NfL
- The US FDA Type B RMAT meeting for AMT-130’s accelerated pathways & enrollment conclusion for the US P-I/II trial’s 3rd arm of AMT-130 + immunosuppression is planned in H2’24 with its safety results in H1’25; interim analysis of 36mos. data vs external controls in mid-2025
Ref: uniQure | Image: uniQure
Related News:- uniQure Provides Update on Low-Dose Cohort in P-I/II Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease
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Disha is a content writer at PharmaShots. She is passionate and curious about recent updates and developments in MedTech and Pharma industry. She covers news related to clinical trial results and updates. She can be contacted at connect@pharmashots.com.
