uniQure's Huntington's gene therapy shows 44% slowing at 48 months but misses statistical significance; shares fall ~70%

uniQure reported 48-month data from Phase I/II studies of ifezuntirgene inilparvovec in Huntington's disease: 12 high-dose patients showed 44% slowing of disease progression on the primary cUHDRS endpoint, but the result did not reach statistical significance, and 17% of high-dose patients developed serious brain inflammation. Shares fell about 70%.

Detected & updated continuously · Source: Nebula