uniQure  N.V. (NASDAQ: QURE) Doses First Two Subjects In the EU Clinical Study of AMT-130 in Huntington Disease Treatment 

uniQure N.V. (NASDAQ: QURE) has announced the dosing of the first two subjects in the European phase Ib/II clinical; study of AMT-130  in Europe. AMT-130 is a potential one-time gene therapy for Huntington’s disease treatment. The company is conducting the clinical study in several sites across the UK, Germany and Poland.

uniQure expands clinical development of AMT-130

The research and development president at the company, Ricardo Dolmetsch, said, We are very pleased to expand the clinical development of AMT-130 and to build on our ongoing experience in Phase I/II clinical trial in the United States. We expect to complete patient enrollment in this European study by the end of the year and to provide safety and target-engagement data from the full 10-patient, low-dose cohort in the U.S. trial in the second quarter of this year.”

Currently, the Interventional Neurotherapy Center (INC) at Mazowiecki Szpital Bródnowski Hospital is Europe’s first and so far only facility for MRI-aided gene therapy injections.

Neurosurgery department  and INC chairperson Miroslaw Zabek commented, “Our team is extremely excited to participate in this important Huntington’s disease scientific research alongside our colleagues in the U.S. and to dose the first patients in the European clinical trial of AMT-130.”

Study to evaluate AMT-130’s safety and dosage in Huntington’s disease 

The proof of concept, safety, and dosing of AMT-130 for Huntington’s disease treatment will be investigated in 15 individuals with early apparent Huntington’s disease, divided into a five-subject low-dose open-label group and a nine-person higher-dose open-label group. AMT-130 will be given to all of the patients. Given recent setbacks in the treatment of Huntington’s disease, the dosing of the first patients in the EU gene therapy study is a massive milestone.

The multi-centre trial includes a six-month post-treatment trial phase followed by a five-year long-term follow-up period. Patients will get a single dose of AMT-130 delivered directly into the striatum using MRI-aided, convection-enhanced stereotactic neurosurgery administration (caudate and putamen). Recruitment for the study is presently underway at IPiN and INC in Poland, with plans to extend to surgical and referral locations in the UK and Germany.