UniQure is progressing with its investigational gene therapy AMT-260 for chronic epilepsy, reporting on June 22 that early trial results show reductions in seizure frequency among patients with refractory mesial temporal lobe epilepsy. The findings come as the company also prepares to submit a biologics license application for its Huntington’s disease gene therapy, AMT-130.
In the Phase 1/2a GenTLE trial, six patients in the low-dose cohort received AMT-260. Three of these patients experienced a decline in disabling seizures ranging from 79% to 100% over four to six months of follow-up. The remaining three saw variable changes, including a decrease of up to 33% or an increase of up to 36%. Stifel said Friday that while these data are from “a small open-label dataset,” they represent “a promising start” and suggested efficacy may improve in higher dose groups. The GenTLE study is enrolling more participants and aims for a total recruitment of twelve, with primary completion expected by November 30.
Stifel also said, “We note that these refractory patients are unlikely to show a response by chance,” but acknowledged risks remain due to the small size and early stage of the program. The analysts added, “this program should be on investors’ radar after UCB’s acquisition of Neurona.” In April, UCB acquired Neurona Therapeutics—developer of NRTX-1001, another cell therapy for drug-resistant mesial temporal lobe epilepsy—for $650 million upfront after NRTX-1001 showed an 89% median reduction in debilitating seizures seven to twelve months post-dosing.
Despite progress with AMT-260, Stifel said investor focus remains on uniQure’s planned submission for AMT-130 targeting Huntington’s disease. UniQure announced last week it intends to file its application in the third quarter following an FDA decision allowing three-year data from its Phase 1/2 trial as sufficient support for accelerated approval.
The path toward this submission has included regulatory challenges. In November last year, the FDA indicated it no longer agreed that Phase 1/2 data would suffice as primary evidence for approval; however, recent leadership changes at the agency have led regulators to reverse course again. The FDA now says it will not require sham surgery controls but instead seeks alignment on confirmatory trial design before accepting uniQure's application.
