CAMBRIDGE, Mass.--(BUSINESS WIRE)--Eikonizo Therapeutics, Inc., a biotechnology company developing novel, first-in-class brain-penetrant HDAC6 inhibitors to slow or stop progression of neurodegenerative disorders, has published results from the First-in-Human Study of [18F]EKZ-001 in healthy volunteers in the European Journal of Nuclear Medicine and Molecular Imaging. The article, entitled “Clinical validation of the novel HDAC6 radiotracer [18F]EKZ-001 in the human brain” is available online at: http://dx.doi.org/10.1007/s00259-020-04891-y. Funding for the study was provided by the Alzheimer’s Drug Discovery Foundation (ADDF). “Thanks to early investment from ADDF, we were able to initiate this trial in Eikonizo’s first year,” said Janice Kranz, PhD, Eikonizo Co-Founder, President and CEO.
[18F]EKZ-001 is a radiolabeled imaging agent that binds the enzyme histone deacetylase 6 (HDAC6) and, together with positron emission tomography (PET), enables quantitative imaging of HDAC6 distribution in the brain. This study represents the first evaluation of an HDAC6-specific PET tracer in humans, enabling use of [18F]EKZ-001 for target engagement studies in future trials of HDAC6-targeting therapeutics as well as in other research on HDAC6 in brain disorders. Inhibiting HDAC6 has shown beneficial effects in animal models of neurodegenerative diseases, including amyotrophic lateral sclerosis and Alzheimer’s disease, making [18F]EKZ-001 a valuable research tool.
The study was performed at University Hospitals Leuven in Belgium and co-led by Koen Van Laere, MD, PhD, Jan de Hoon, MD, PhD, and Michel Koole, PhD. [18F]EKZ-001 was found to be safe and well tolerated by adult volunteers. The study identified the optimal PET imaging approach for [18F]EKZ-001 and characterized regional HDAC6 distribution in the living human brain. Previously, this could only be achieved post-mortem. HDAC6 was widely expressed, with the highest [18F]EKZ-001 uptake observed in the hippocampus and entorhinal cortex, regions particularly relevant in neurodegeneration.
“We are greatly encouraged by these results and plan to use [18F]EKZ-001 PET to confirm and quantify brain HDAC6 target engagement by Eikonizo’s drug candidates in therapeutic clinical trials,” said corresponding author Tonya Gilbert, PhD, Director of Translational Research at Eikonizo. “This capability gives Eikonizo a unique competitive advantage by enabling optimal dose selection and thus more likely successful efficacy trials,” explained Dr. Kranz. “HDAC6 is a promising target for neurotherapeutic development, and this clinical validation of [18F]EKZ-001, a molecule born in my MGH (Massachusetts General Hospital) lab, is a key step in the development of HDAC6-based therapies.” said Jacob Hooker, PhD, Co-Founder of Eikonizo, Professor of Radiology at Harvard Medical School, and Director of Radiochemistry at the Athinoula A. Martinos Center.
“We are very proud to have provided funding for this study, and are enthusiastic about the potential role of HDAC6-targeting therapies for Alzheimer’s disease,” said Howard Fillit, MD, Founding Executive Director and Chief Science Officer of the Alzheimer's Drug Discovery Foundation. “At ADDF we have long supported the concept that a drug ‘cocktail’ targeting multiple pathways may be needed and Eikonizo’s small molecule drug is a promising part of that approach, as well as possibly monotherapy.”
Eikonizo also announced two executive appointments: Richard Margolin, MD, as Chief Medical Officer and Dean Hickman, D.Phil., as Director of Translational Pharmacology and Drug Development. Dr. Margolin, who has led clinical neuroscience efforts at companies including Pfizer, CereSpir, Schering-Plough/Merck and i3 Research, brings decades of experience in clinical pharmacology, CNS clinical trial design and execution, molecular imaging and fluid biomarkers. Dr. Hickman has supported drug discovery, development, and post marketing efforts in multiple therapeutic areas, including neuroscience, during his 20+ years at Abbott Laboratories and Amgen. “He brings sophisticated pharmacokinetic, drug metabolism and translational pharmacology expertise to Eikonizo,” said Dr. Kranz. “We are delighted to welcome Rick and Dean to our team. Their deep industry experience will serve us well in advancing our compounds to the clinic.”
About Eikonizo Therapeutics, Inc.
Eikonizo is a biotechnology company committed to creating life-changing therapies by discovering brain-penetrant small molecules to slow or stop neurodegeneration and leveraging companion target engagement technology to de-risk and accelerate development. Eikonizo is funded by Kevin Kinsella, founder of Avalon Ventures, and ADDF, among other investors. For information, www.eikonizo.com.
About the Alzheimer’s Drug Discovery Foundation
Founded in 1998 by Leonard A. and Ronald S. Lauder, the Alzheimer's Drug Discovery Foundation is dedicated to rapidly accelerating the discovery of drugs to prevent, treat and cure Alzheimer's disease. The ADDF is the only public charity solely focused on funding the development of drugs for Alzheimer's, employing a venture philanthropy model to support research in academia and the biotech industry. Through the generosity of its donors, the ADDF has awarded more than $150 million to fund over 626 Alzheimer's drug discovery programs and clinical trials in 19 countries. To learn more, please visit: http://www.alzdiscovery.org/.