uniQure Announces First Two Patients Treated in Phase 1/2 Clinical Trial

uniQure NV recently announced that the first two patients in the Phase 1/2 clinical trial of AMT-130 for the treatment of Huntington’s disease have been treated. The Phase 1/2 study is a double-blind, randomized clinical trial being conducted in the US, with now one patient treated with AMT-130, and one patient who received the imitation surgery.

“For years, uniQure has had an unwavering commitment to advance this first-in-human AAV gene therapy for Huntington’s disease into clinical testing, and this moment marks an important milestone for our company now that we have two AAV gene therapy candidates in clinical development,” said Matt Kapusta, Chief Executive Officer of uniQure. “With the first two patients treated in this trial, we have taken a significant step forward in advancing AMT-130 closer to our goal of developing a therapy that inhibits the production of the mutant huntingtin protein. We are delighted to be working with leading experts in the field to evaluate this promising candidate.”

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 patients with early manifest Huntington’s disease randomized to treatment with AMT-130 or an imitation (sham) surgery. The 5-year, multi-center trial consists of a blinded 18-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.clinicaltrails.gov (NCT04120493).

The first two patients will be observed for an initial period of 90 days, followed by a meeting of the Data Safety Monitoring Board (DSMB). The DSMB will review the data on the first two patients and make a determination about continued dosing of the next patients.

AMT-130 is uniQure’s first clinical program focusing on the central nervous system (CNS) incorporating its proprietary miQURE platform.

“There is an urgent need for disease-modifying options to treat Huntington’s disease, and we’re excited to have an investigational gene therapy now available for HD patients,” stated George Yohrling, Chief Scientific Officer and Chief Mission Officer at Huntington’s Disease Society of America. “Based on the promising preclinical data presented on AMT-130 over the years, we are optimistic about its potential to alter the course of this devastating 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, hemophilia A, Huntington’s disease, Fabry disease, spinocerebellar ataxia Type 3, and other diseases. For more information, visit www.uniQure.com.