uniQure Announces Positive Recommendation to Advance Phase 1/2 Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease

uniQure N.V. recently announced the independent Data Safety Monitoring Board (DSMB) overseeing the Phase 1/2 clinical trial of AMT-130 for the treatment of Huntington’s disease has met and reviewed safety data for the fully enrolled first cohort of ten patients. This data set included 9-month safety data from the first two enrolled patients, 6-month safety data from the next two enrolled patients, and 30-day safety data on remaining six patients in the trial. The DSMB recommended continued dosing in the study, and uniQure will now begin to enroll patients in the higher-dose cohort of the trial. The Phase 1/2 study is a double-blind, randomized clinical trial being conducted in the US. To date, six patients have been treated with AMT-130, and four patients received the imitation surgery.

“We are very pleased with the progress of the study and with the recommendations of the DSMB. We look forward to proceeding with dose-escalation in the second cohort of patients,” said Ricardo Dolmetsch, PhD, President of Research and Development at uniQure. “We remain highly focused on progressing this therapy given the significant unmet need in the Huntington’s disease community and are committed to initiating clinical development of AMT-130 in Europe in the second half of this year and to sharing initial imaging and biomarker data from the US clinical trial before the end of the year.”

The US Phase 1/2 clinical trial of AMT-130 for the treatment of Huntington’s disease will explore the safety, tolerability, and efficacy signals in 26 total patients with early manifest Huntington’s disease split into a 10-patient, low-dose cohort followed by a 16-patient, higher-dose cohort randomized to treatment with AMT-130 or an imitation (sham) surgery. The multi-center trial consists of a blinded 12-month core study period followed by unblinded 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). Additional details are available on www.clinicaltrials.gov (NCT04120493).

The European, open-label Phase 1b/2 study of AMT-130 will enroll 15 patients with early manifest Huntington’s disease across two dose cohorts. Together with the US study, the European study is intended to establish safety, proof of concept, and the optimal dose of AMT-130 to take forward into Phase 3 development or into a confirmatory study should an accelerated registration pathway be feasible. AMT-130 is uniQure’s first clinical program focusing on the central nervous system (CNS) incorporating its proprietary miQURE platform.

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 and other diseases. For more information, visit www.uniQure.com