uniQure Announces Latest Positive Recommendation From Data Safety Monitoring Board in Phase 1/2 Clinical Trial of AMT-130 for the Treatment of Huntington’s Disease

uniQure N.V. recently announced the positive recommendation by the independent Data Safety Monitoring Board (DSMB) following a review of safety data from the four patient procedures in the higher-dose cohort of the Phase 1/2 clinical trial of AMT-130 for the treatment of Huntington’s disease. With the positive recommendation, the final 12 patients in this second cohort are now cleared for enrollment.

A total of 14 blinded administration procedures have been completed as of August 2021. In the study to date, eight patients have been treated with AMT-130, and six patients have received imitation surgery. Full enrollment of the second, higher-dose cohort is expected to be completed in mid-2022.

“The AMT-130 program continues to advance at a steady pace, and with this positive recommendation from the DSMB, we are eager to enroll the final 12 patients in the higher-dose cohort,” said David Cooper, MD, Vice President of Clinical Development at uniQure. “We also look forward to expanding our efforts with the initiation of a separate open-label study of AMT-130 in Europe and to sharing preliminary imaging and biomarker data from the initial four patients in 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 is exploring the safety, tolerability, and efficacy signals in a planned 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; patients will be 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 5 years. A total of 16 patients in the clinical trial 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.