uniQure N.V. QURE

uniQure said AMT-130 continued to slow Huntington’s disease in ongoing early- and mid-stage studies after four years.

Uniqure stock plummeted Tuesday on a disappointing four-year update for its experimental gene therapy in patients with Huntington's disease.

uniQure N.V. (NASDAQ:QURE) stock is trading lower on Tuesday after the company shared mixed long-term data from ongoing Phase 1/2 clinical studies evaluating ifezuntirgene inilparvovec for Huntington’s disease. 36-Month Clinical Results And BLA Pathway In the 36-month analysis, high-dose patients demonstrated an 80% slowing of disease progression on the Composite Unified Huntington’s Disease Rating Scale (cUHDRS) and a 67% slowing on Total Functional Capacity (TFC). cUHDRS is a specialized scori

UniQure says it still sees a path to regulatory approval for the drug candidate, AMT-130, even as the experimental gene therapy for Huntington’s disease falls hort in a recent study.

uniQure N. V. (NASDAQ:QURE) shares fell 64% on Tuesday after the gene therapy developer reported updated 48-month data for ifezuntirgene inilparvovec, or AMT-130, its experimental treatment for Huntington’s disease.

ClearPoint Neuro (NASDAQ:CLPT) shares fell 24% on Tuesday following updated clinical trial results from partner uniQure N. V.

uniQure’s Ifezuntirgene slowed disease progression by 80% on a measure combining movement, thinking and daily function.

Investing.com -- Shares of gene therapy developer uniQure N.V. (NASDAQ: QURE) cratered 64% Tuesday morning following an updated data readout for its experimental Huntington’s disease treatment, ifezuntirgene inilparvovec (AMT-130). While the treatment continued to show a dose-dependent slowing of disease progression, a failure to hit statistical significance on a primary clinical measure at 48 months triggered a massive sell-off.

~ In 12 high-dose patients at 48 months, both cUHDRS and TFC continued to demonstrate meaningful slowing of disease progression and clear dose-dependent response; 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) ~ ~ Updated data reflecting all 15 high-dose patients showed substantial treatment effect on both cUHDRS and TFC at 36 months, the timepoint that is the regulatory anch

Novo has 44 abstracts at EASD, including CagriSema analyses and Phase 2b Zenagamtide results.