Benzinga | Apr 8, 2021 07:08AM EDT

uniQure Highlights Publications Of Preclinical Data For AMT-130 In Huntington's Disease

uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that three manuscripts on preclinical data from its gene therapy candidate AMT-130 in Huntington's disease have been accepted for publication, in the journals Science Translational Medicine, Brain Science, and Brain Communications. The publications show the safety and efficacy of AMT-130 in the deep brain structures of a large animal model and outline a promising novel efficacy biomarker for AMT-130.



"Taken together, these publications demonstrate widespread biodistribution and strong, durable efficiency of AMT-130 in disease-relevant regions in a large brain," stated Ricardo Dolmetsch, Ph.D., president of research and development at uniQure. "The data provide further support for the potential therapeutic value of AMT-130, and we remain enthusiastic about our Phase I/II clinical trial of AMT-130 in patients with Huntington's disease."

Widespread and Sustained Target Engagement in Huntington Disease Minipigs

The paper published this week in Science Translational Medicine examines the translatability and long-term durability of AMT-130 in transgenic Huntington's disease minipigs, which were used to assess the biodistribution and target engagement in a larger brain. The minipig model is the largest diseased animal model available, generally weighing up to 300 pounds.

AMT-130 was administered by MRI-guided convention-enhanced delivery (CED) at a single dose, bilaterally in the caudate and putamen. Vector DNA distribution and transgene expression in minipig brains demonstrated extensive brain coverage comparable at the interim sacrifice timepoints of 6- and 12-months post administration, leading to significant lowering of mutant huntingtin (mHTT) protein in the brain.

At 12 months, the most pronounced mHTT protein lowering was observed in the putamen (85%), caudate (80%) and amygdala (78%), followed by thalamus (56%) and cerebral cortex (44%).

The publication, "Widespread and Sustained Target Engagement in Huntington Disease Minipigs upon Intrastriatal MicroRNA-based Gene Therapy," is available online in the journal Science Translational Medicine (DOI: 10.1126/scitranslmed.abb8920).