our science
Eikonizo’s Platform Targets HDAC6
Eikonizo is developing oral, brain-penetrant, highly selective small molecule histone deacetylase 6 (HDAC6) inhibitors as disease-modifying therapeutics for neurodegenerative disease, including amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), Parkinson’s disease (PD) and Alzheimer’s disease (AD). Eikonizo is also developing similarly differentiated peripheral HDAC6 inhibitors for cardiorenal and other indications, such as ADPKD, Diabetic Nephropathy and heart failure.
Our lead CNS program, EKZ-102, is a potential first-in-class, oral, small molecule HDAC6 inhibitor with the distinctive combination of high potency, selectivity and CNS penetrance necessary to achieve neuroprotection while minimizing potential off-target side effects to achieve a favorable safety and tolerability profile. In vivo PET imaging using a second-generation PET agent paired with EKZ-102 has the potential to confirm CNS penetrance and target engagement in the clinic. EKZ-102 is in IND-enabling development for the treatment of ALS with planned expansion to other CNS disorders.
Therapeutic Hypothesis
Eikonizo’s unique approach to HDAC6 inhibition is designed to synergistically target the mechanisms of intracellular transport, intracellular signaling and proteostasis (protein homeostasis), key drivers of biology of multiple neurodegenerative and cardiorenal diseases.
Correcting these cellular mechanisms via HDAC6 inhibition may also improve other common pathological disease processes including chronic inflammation, mitochondrial dysfunction and oxidative/nitrosative stress and fibrosis, leading to overall improvements in cellular health and function.
Inhibiting HDAC6 in the brain and peripheral nervous system will preserve axonal transport and reduce aberrant protein aggregation, which together may slow or stop the progression of neurodegenerative disease.
HDAC6 inhibition may also impact the course of peripheral indications by improving the inflammation, oxidative stress, and fibrosis that underly the pathological defects in heart failure, chronic kidney disease and other cardiorenal disorders.
Supporting
Literature
Our platform is supported by preclinical and clinical literature validating Eikonizo’s novel HDAC6 inhibitor approach.