uniQure Announces Positive Recommendation From Data Safety Monitoring Board of Phase 1/2 Clinical Trial

uniQure N.V. recently announced that 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 the 6-month safety data from the first two enrolled patients and the 90-day safety data from the next two enrolled patients in the study. No significant safety concerns were noted to prevent further dosing, and the final six patients in the first cohort are now cleared for enrollment. uniQure expects to achieve full patient enrollment in cohort one by mid-year 2021. The Phase 1/2 study is a double-blind, randomized clinical trial being conducted in the US. To date, two patients have been treated with AMT-130, and two patients received the imitation surgery.

“We are encouraged with the positive recommendation to proceed with patient enrollment from this second DSMB meeting,” said Ricardo Dolmetsch, President of Research and Development at uniQure. “We will now focus on enrolling the last six additional patients in the first cohort and look forward to sharing initial biomarker and imaging data towards the end of the year.”

The 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 5-year, multi-center trial consists of a blinded 12-month core study period followed by unblinded long-term follow-up. 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).

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.