uniQure Announces Update on U.S. Phase I/II Clinical Trial of AMT-130 Gene Therapy for the Treatment of Huntington’s Disease

~ AMT-130 continues to be generally well-tolerated across both dose cohorts ~

~ Patients treated with AMT-130 show preserved function compared to baseline and clinical benefits relative to natural history of the disease ~

~ Neurofilament Light Chain (NfL) in cerebrospinal fluid (CSF) was below baseline at 24 months in patients treated with the low-dose of AMT-130 and declining towards baseline at 12 months in patients treated with the high-dose of AMT-130 ~

~ Suppression of CSF mHTT in low-dose cohort supports AMT-130 target engagement; Greater variability observed in high-dose cohort ~

~ Promising data support continuing clinical development of AMT-130 and pursuing regulatory interactions to discuss late-stage development ~

~ Investor conference call and webcast today at 8:30 a.m. ET ~

LEXINGTON, Mass. and AMSTERDAM, June 21, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced promising interim data, including up to 24 months of follow-up, from 26 patients enrolled in the ongoing U.S. Phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease.

“We are very pleased with the data from the interim analysis of our U.S. Phase I/II clinical trial of AMT-130, a one-time administered investigational gene therapy for Huntington’s Disease,” stated Ricardo Dolmetsch, Ph.D., president of research and development at uniQure. “AMT-130 continues to be generally well-tolerated with a manageable safety profile at both doses. Importantly, both doses show preliminary evidence of clinical and functional benefits, including favorable trends in Total Motor Score, Total Functional Capacity and the composite Unified Huntington’s Disease Rating Scale compared to natural history. We plan to engage with regulators to advance this promising clinical program as we collect more data from these patients and from our European study.”   

“Today’s encouraging interim update shows early signs of a potential clinical benefit of AMT-130 and supportive trends in neurofilament light chain, a key marker of neuronal damage that has proven useful across multiple neurodegenerative disorders,” stated Sarah Tabrizi, M.D., FRCP, Ph.D., professor of clinical neurology, director of the University College London (UCL) Huntington’s Disease Center and joint head of the department of neurodegenerative disease at UCL. “Despite the small patient numbers, I am encouraged to see that patients treated with either dose of AMT-130 appear to have largely preserved function and are trending favorably to natural disease course at up to 24 months. These interim results provide early hope for patients suffering from this devastating disease, and I look forward to additional clinical updates and the further investigation of AMT-130 as a potentially important treatment option for patients with Huntington’s disease.”