uniQure Announces Dosing of First Patients in European Open-Label Clinical Trial of AMT-130 Gene Therapy in Huntington’s Disease

uniQure N.V. recently announced the dosing of the first two patients in its European open-label Phase 1b/2 clinical trial of AMT-130, a potential one-time gene-therapy approach for the treatment of Huntington’s disease. The clinical trial is taking place at several sites in Poland, the UK, and Germany.

“We are very pleased to expand the clinical development of AMT-130 and to build on our ongoing experience in the Phase 1/2 clinical trial in the US,” said Ricardo Dolmetsch, PhD, President of Research and Development at uniQure. “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 US trial in the second quarter of this year.”

“The Interventional Neurotherapy Center (INC) at Mazowiecki Szpital Bródnowski Hospital is the first and only center in Europe currently performing MRI-guided infusions of gene therapies,” said Professor Miroslaw Zabek, MD, PhD, the chairperson of the department of neurosurgery and INC. “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.”

“Since 1995, our center at the Institute of Psychiatry and Neurology (IPiN) has offered genetic testing and clinical care to Polish patients with Huntington’s disease,” said Dr. Grzegorz Witkowski, MD, PhD, IPiN principal investigator. “Our patients have been very interested in the potential for a one-time treatment to stop progression of the disease and, given the recent setbacks in Huntington’s disease research it means a lot to the Polish HD Community to be able to enroll the first patients in this first EU gene therapy trial.”

The European Phase 1b/2 clinical trial of AMT-130 for the treatment of Huntington’s disease will explore the safety, proof of concept, and dosing in 15 total patients with early manifest Huntington’s disease split into a five person, low-dose open-label cohort, followed by a nine patient, higher-dose open-label cohort. All patients will be dosed with AMT-130.

The multi-center study consists of an initial 6-month post-treatment study period followed by long-term follow-up for five years. Patients will receive a single administration of AMT-130 through MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum (caudate and putamen). The study is currently open for recruitment at IPiN and surgery at INC in Poland and is expected to expand to referral and surgical sites in the United Kingdom and Germany. Additional details are available on www.clinicaltrialsregister.eu (EudraCT 2020-001461-36).

AMT-130 is uniQure’s first central nervous system (CNS) focused gene therapy product consisting of an AAV5 vector carrying an artificial micro-RNA specifically tailored to silence the huntingtin gene, leveraging our proprietary miQURE silencing technology. The therapeutic goal is to inhibit the production of the mutant protein (mHTT). Using AAV vectors to deliver micro-RNAs directly to the brain for non-selective knockdown of the huntingtin gene represents a highly innovative and promising approach to treating Huntington’s disease.

Huntington’s disease is a rare, inherited neurodegenerative disorder that leads to motor symptoms including chorea, and behavioral abnormalities and cognitive decline resulting in progressive physical and mental deterioration. The disease is an autosomal dominant condition with a disease-causing CAG repeat expansion in the first exon of the huntingtin gene that leads to the production and aggregation of abnormal protein in the brain. Despite the clear etiology of Huntington’s disease, there are no currently approved therapies to delay the onset or to slow the disease’s progression.

uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, Huntington’s disease, Fabry disease, spinocerebellar ataxia Type 3 temporal lobe epilepsy, Alzheimer’s, Parkinson’s, and ALS. For more information, visit www.uniQure.com.