Eikonizo Therapeutics Announces Publication in Brain Demonstrating Disease-Modifying Potential of Selective HDAC6 Inhibitors in Preclinical Models of ALS and FTD

Increased health and survival of motor neurons and reduced plasma neurofilament light chain (NfL) observed, underscoring disease-modifying potential of CNS-penetrant HDAC6 inhibitors, including EKZ-102, the Company’s lead candidate for the treatment of ALS and other neurodegenerative diseases

Company also awarded SBIR grant to advance EKZ-102 in Parkinson’s disease (PD), supported by strong literature linking HDAC6 inhibition to PD pathology

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Eikonizo Therapeutics, a biopharmaceutical company developing disease-modifying therapies to improve the lives of people impacted by neurodegenerative and cardiorenal diseases, today announced the advance online publication of its preclinical research in the peer-reviewed journal Brain, demonstrating the therapeutic potential of the company’s highly selective, CNS-penetrant HDAC6 inhibitors in the treatment of neurodegenerative disease, including amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The company will also be presenting a subset of this data at the upcoming ALS Therapy Development Institute (TDI) Summit on October 17, 2025 in an oral presentation, titled “The evolution of HDAC6 as a potential therapeutic target for ALS”.

The article in Brain, titled “A next-generation HDAC6 inhibitor for amyotrophic lateral sclerosis and frontotemporal dementia” (James, et al., Brain, https://doi.org/10.1093/brain/awaf380) reports that selective HDAC6 inhibition mitigated broad proteostasis and axonal transport defects, oxidative stress and neuroinflammation in preclinical models of familial and sporadic ALS and in FTD. The data also show a reduction in mislocalized TDP-43—a proteostasis defect seen in most ALS cases and about half of FTD cases—while restoring some of its normal activity and increasing survival in lab-grown human motor neurons. Importantly, treatment with EKZ-438 (a tool compound and predecessor to Eikonizo’s lead candidate EKZ-102) was associated with improved motor function, as measured by performance-based tests, and reduced plasma NfL—a key marker of disease modification – in a preclinical animal model. Together, these data reinforce the therapeutic potential of Eikonizo’s lead candidate, EKZ-102, a potential first-in-class oral HDAC6 inhibitor distinguished by its high potency, selectivity and CNS penetration. EKZ-102 is on track to enter Phase 1 clinical trials in 2026.

In parallel, Eikonizo has been awarded a Phase 1 Small Business Innovation Research (SBIR) grant by the National Institute of Neurological Disorders and Stroke (NINDS) to support the advancement of its lead candidate, EKZ-102, for the treatment of Parkinson’s disease. A growing body of evidence links HDAC6 inhibition to disease pathways in PD—including mitochondrial dysfunction and α-synuclein aggregation—further underscoring the broad potential of the company’s HDAC6 inhibitors as disease-modifying therapeutics for the treatment of neurodegenerative disease.

“The findings published in Brain and presented at ALS TDI reinforce the promise of our highly selective HDAC6 inhibitors and position EKZ-102 to advance into human clinical trials as a potential disease-modifying strategy for ALS and FTD,” said Rick Lundberg, President and CEO of Eikonizo. “The SBIR award broadens that path into Parkinson’s disease, giving us the momentum to move EKZ-102 into the clinic in 2026 and generate the human data that matters for patients.”

About EKZ-102

Eikonizo’s lead development candidate, EKZ-102, is a first-in-class, oral, small-molecule HDAC6 inhibitor with the distinctive combination of high potency, selectivity and CNS penetrance necessary to protect neuronal function while minimizing potential off-target side effects to achieve a favorable safety and tolerability profile. EKZ-102 is in IND-enabling development with clinical studies planned in 2026.

About Eikonizo Therapeutics

Eikonizo is developing novel, disease-modifying small molecule therapeutics targeting histone deacetylase 6 (HDAC6) to improve the lives of people impacted by neurodegenerative and cardiorenal diseases. Our unique approach to HDAC6 inhibition is designed to synergistically modulate multiple key mechanistic drivers of neurodegenerative and cardiorenal disease biology.

Contacts

Media:

Gina Nugent, Nugent Communications

[email protected]