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 dual mechanism approach to HDAC6 inhibition is designed to synergistically target both intracellular transport and proteostasis defects, two key drivers of neurodegenerative and cardiorenal disease biology.
HDAC6 inhibitor treatment has the potential to preserve axonal transport and reduce pathogenic protein aggregates to stop or slow the progression of ALS and other neurodegenerative diseases. Our HDAC6 inhibitor platform also offers the potential to address other indications associated with intracellular transport and proteostasis defects, including cardiorenal diseases such as autosomal dominant polycystic kidney disease (ADPKD), Diabetic Nephropathy (DN) and Heart Failure.
Supporting
Literature
Our platform is supported by preclinical and clinical literature validating Eikonizo’s novel HDAC6 inhibitor approach.