New Medicines

Early manifest Huntington's disease in adults


New molecular entity

Development and Regulatory status

Phase II Clinical Trials
Apr 19US FDA granted fast track status for AMT 130 for treatment of HD. [2]
Jan 18Orphan Medicinal Product Designation (OMPD) from the EMA for the treatment of Huntington´s disease. [2]
Oct 17Orphan drug designation from the US FDA for treatment of HD. [2]


Single dose gene therapy. An AAV5 vector carrying artificial micro-RNA designed to silence the huntingtin gene by targetting accumulation of the exon 1 HTT fragment (a source of abnormal protein aggregation in HD).[3]
Average prevalence in Western Europe including the UK, Australia and USA has been estimated at 9.71 per 100,000. [1]
Early manifest Huntington's disease in adults

Trial or other data

Oct 20uniQure reports that two additional patient enrolment procedures were completed in the PI/II (NCT04120493) trial. The decision to continue ongoing patient enrolment was taken, following a favourable meeting with the independent Data Safety Monitoring Board (DSMB) in Sep 20, regarding a review of the 90-day follow-up data from the first two patients. The DSMB highlighted no significant safety concerns to preclude further dosing. Further patient enrolment is contingent on another DSMB meeting and its outcome, to review 90-day follow-up data on the two new patients and 6-month data from the first two patients. The meeting is anticipated to be conducted in early 2021 [6].
Jun 20First two enrolled pts treated with AMT 130 in PI/II study. [3]
Sep 19PI/II proof-of-concept study (NCT04120493) initiated. This randomised, multicentre, dose escalation, double-blind, imitation surgery, first-in-human (FIH) study will evaluate the safety, tolerability and efficacy of AMT 130 in pts with early manifest HD. The trial will enrol ~ 26 pts in the US who will have single dose of AMT-130 via intrastriatal administration at two different dose levels or imitation surgery. The primary outcome measure is the number and type of Adverse Events (AE) and secondary outcomes include duration of persistence of AMT-130 in the brain through month 60 and changes in the Unified Huntington Disease Rating Scale (UHDRS) through month 60. Data are expected in December 2022. [3,4]