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Amt 130

All articles tagged with #amt 130

uniQure Stock Plunges 41% as Huntington’s Gene Therapy Misses Key 48-Month Endpoint
biotechnology10 days ago

uniQure Stock Plunges 41% as Huntington’s Gene Therapy Misses Key 48-Month Endpoint

uniQure shares dropped 41% after its Huntington’s disease gene therapy, AMT-130, failed to reach statistical significance on its primary endpoint at 48 months. While the composite Unified Huntington’s Disease Rating Scale (cUHDRS) showed only a 44% slowing of progression, the secondary Total Functional Capacity (TFC) endpoint remained strong at 61%. Analysts and executives argue that methodological flaws in the control group and the stability of functional outcomes support the drug’s path to accelerated FDA approval, despite the initial market sell-off.

via Fierce Biotech|
#amt-130#biotechnology#fda-approval
FDA orders phase 3 for Huntington's gene therapy, delaying uniQure's path to approval
health7 months ago

FDA orders phase 3 for Huntington's gene therapy, delaying uniQure's path to approval

The FDA rejected uniQure's plan to file AMT-130 using phase 1/2 data with an external control and demanded a prospective, randomized, sham-controlled phase 3 trial, dampening near-term approval hopes and triggering a strategic review by the company; uniQure will discuss trial designs with the FDA and add a four-year durability analysis to the phase 1/2 data.

via Fierce Biotech|
#amt-130#fda#health
Promising Gene Therapy Hints at Slowing Huntington's Disease Progression
science8 months ago

Promising Gene Therapy Hints at Slowing Huntington's Disease Progression

A small trial of uniQure’s AMT-130 gene therapy in 29 Huntington’s disease patients showed reduced production of the mutant huntingtin protein, slower cognitive decline, and lower levels of neurofilament light in cerebrospinal fluid over three years, suggesting a potential disease-modifying effect. Results were reported in a press release and have not yet undergone peer review, but they point to a possible treatment that could be used earlier in the disease course, while researchers continue to identify early biomarkers and expand the treatment window before motor symptoms appear.

via ScienceAlert|
#amt-130#clinical-trial#gene-therapy
uniQure's AMT-130 Regulatory Uncertainty Sparks Hope for Huntington’s Disease
healthcare11 months ago

uniQure's AMT-130 Regulatory Uncertainty Sparks Hope for Huntington’s Disease

uniQure announced that the FDA no longer considers Phase I/II data versus an external control sufficient as primary evidence for a BLA submission for its Huntington's disease gene therapy AMT-130, creating uncertainty about the approval timeline. The company plans urgent discussions with the FDA and continues regulatory engagement in the EU and UK, despite holding breakthrough therapy and RMAT designations.

via Stock Titan|
#amt-130#fda#healthcare