Market
What will be the outcome of AMT-130 Phase I/II trials by June 30, 2025?
2
Resolution / Starting Odds
Significant improvement • 25%
Moderate improvement • 25%
No improvement • 25%
Worsening condition • 25%
uniQure trial announcements and scientific publications
Story
uniQure Aligns with FDA for BLA Submission and Accelerated Approval of AMT-130 in Phase I/II Huntington's Disease Studies
Dec 10, 2024, 12:18 PM
uniQure has announced that the FDA has agreed that data from its ongoing Phase I/II studies for AMT-130, a treatment for Huntington's Disease, may serve as the primary basis for a Biologics License Application (BLA) submission under the Accelerated Approval pathway. This alignment with the FDA is seen as a significant step forward for the company, as it establishes the use of natural history external control data in the review process. The FDA's flexibility in review standards for gene therapy applications is also noted, with emphasis on the importance of biomarkers like mHTT and NfL being deprioritized in this context. The announcement has generated positive sentiment among investors, drawing comparisons to previous successful approvals in the biotech sector.
View original story
Similar markets
Worse outcomes • 25%
Moderate improvement • 25%
No improvement • 25%
Significant improvement • 25%
Conditional approval • 25%
Phase III trial initiation • 25%
BLA submission • 25%
No progress • 25%
Negative results • 25%
Positive results with MRD-negative CR • 25%
Mixed results • 25%
Trial discontinued • 25%
Positive efficacy, but safety concerns • 25%
Negative efficacy, but good safety • 25%
Negative efficacy and safety concerns • 25%
Positive efficacy and safety results • 25%
Unsuccessful trial • 33%
Successful trial • 33%
Inconclusive results • 34%
Unsuccessful • 25%
Successful with moderate efficacy • 25%
Successful with high efficacy • 25%
Trial inconclusive • 25%
Negative immunogenicity results • 25%
Study inconclusive • 25%
Positive safety and immunogenicity results • 25%
Negative safety results • 25%
Positive results • 33%
Negative results • 33%
Inconclusive results • 34%
Negative results halting development • 25%
Neutral results requiring further study • 25%
Positive results leading to Phase 2 • 25%
Other outcome • 25%