uniQure’s Huntington’s disease gene therapy has failed to significantly slow disease progression after 48 months, sending the biotech’s stock down 41% when trading began. But mitigating circumstances and the ongoing strength of a secondary endpoint kept analysts optimistic about AMT-130’s path to approval.

One year ago, uniQure linked AMT-130 to a 75% slowing of disease progression, as measured by the composite Unified Huntington’s Disease Rating Scale (cUHDRS), at Month 36 of a phase 1/2 trial. Tuesday, the biotech shared updated data on the 12 patients. At month 48, uniQure linked AMT-130 to a 44% slowing of disease progression on cUHDRS. The result fell short of statistical significance. 

Investors…