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

~ Patients treated with AMT-130 continue to show evidence of preserved neurological function with potential dose-dependent clinical benefits relative to an inclusion criteria-matched natural history of the disease ~

~ Mean CSF NfL continue to demonstrate favorable trends with low-dose patients below baseline at 30 months and high-dose patients near baseline at 18 months ~

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

~ Data support continuing clinical development of AMT-130 and pursuing regulatory interactions to discuss potential strategies for ongoing development ~

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

LEXINGTON, Mass. and AMSTERDAM, Dec. 19, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced updated interim data including up to 30 months of follow-up from 39 patients enrolled in the ongoing U.S. and European Phase I/II clinical trials of AMT-130 for the treatment of Huntington’s disease.

“The clinical assessment trends in the ongoing studies of AMT-130 look very promising and continue to show disease stability in Huntington’s disease patients treated with this one-time administered gene therapy, several of whom have now been followed more than two years,” stated Walid Abi-Saab, M.D., chief medical officer of uniQure. “We are observing favorable trends in evaluation of motor skills, functional independence, and composite rating scores as compared to a non-concurrent criteria-matched natural history cohort.”  

“We also are pleased to observe further declines in levels of NfL, a measurement of neuronal degradation and disease progression, with low-dose patients below baseline at 30 months of follow-up and high-dose patients near baseline at 18 months,” he continued. “Importantly, AMT-130 continues to be generally well-tolerated with a manageable safety profile at both its low and high doses. We will continue to follow these patients and look forward to initiating regulatory interactions next year.”

“The results from these Phase I/II trials continue to be very encouraging as they show positive-trending, potentially dose-dependent signals across multiple key clinical and functional measures, in conjunction with further declines in NfL,” stated Edward Wild, Ph.D., FRCP, professor of neurology at University College London (UCL) Queen Square Institute of Neurology, consultant neurologist at National Hospital for Neurology & Neurosurgery, and associate director of UCL Huntington’s Disease Centre. “While there are well-known complexities associated with analyzing and interpreting other biomarkers in Huntington’s disease, these NfL data are consistent with the clinical data suggesting possible disease stability and support the continued development of AMT-130. The Huntington’s disease community has endured a prolonged and challenging wait for disease-modifying treatment options, and we enthusiastically embrace this potentially important advancement for this devastating disease.”