Uniqure’s Huntington’s hopes take a hit

A 27% drop in Uniqure’s share price seems a little overdone, seeing as the clinical trial pause announced yesterday concerns only the high dose of the group’s Huntington’s project, AMT-130. But the reaction illustrates current scepticism around gene therapies: when something goes wrong, investors seem to fear the worst. The halt was spurred by severe adverse reactions in three patients receiving high-dose AMT-130, 6x10¹³, who have since “fully or substantially” recovered. The cause of the problem is unknown, but analysts suggested an immune response to the project or procedure, given the events occurred soon after administration; AMT-130 is delivered directly into deep brain structures. Low-dose AMT-130, 6x10¹², is still in play, no link with serious adverse events having been found. This dose yielded promising biomarker results in June, but the big test will involve functional endpoints. Data here are due in the second quarter of 2023, when Uniqure will also report results with high-dose patients already treated in its US trial. Stifel analysts looked on the bright side, saying the high dose could still be “workable”, but that it might not be needed given the low-dose results. Less-than-convinced investors now face a nervous wait.