Io Therapeutics, Inc., presented today results from studies of IRX4204, the company’s phase II clinical development stage, highly selective RXR nuclear receptor agonist compound, supporting its potential use for treatment of amyotrophic lateral sclerosis (ALS)

SPRING, Texas, July 16, 2024 (GLOBE NEWSWIRE) -- Io Therapeutics, Inc., presented today results from studies of IRX4204, the company’s phase II clinical development stage, highly selective RXR nuclear receptor agonist compound, supporting its potential use for treatment of amyotrophic lateral sclerosis (ALS).

The presentation titled “The RXR Nuclear Receptor Agonist Compound IRX4204 is a Potential New Treatment for Amyotrophic Lateral Sclerosis” was delivered at the ALS Nexus conference, sponsored by the ALS Association, being held in Dallas, Texas. The presentation was authored by Vidyasagar Vuligonda, Ph.D., Chief Science Officer of the company and inventor of IRX4204, and Martin E. Sanders, M.D., the company’s Chief Executive Officer.

ALS is a progressively debilitating and ultimately fatal form of neurodegeneration which has incompletely understood causes and disease mechanisms. Autoimmune imbalance in the central nervous systems (CNS) of ALS patients, manifested by too few immunosuppressive Treg cells countered by overabundance of pro-autoimmune Th17 cells, is a pathologic finding well documented in ALS patients. ALS also has documented microglia-mediated neuroinflammation, and demyelination (loss of myelin sheaths surrounding neurons) as components of its pathology.

In support of the potential for IRX4204 as a new treatment for ALS, the company reported that IRX4204 promotes growth of human Treg cells and inhibits growth of human Th17 cells, restoring their balance while concomitantly inhibiting neurodegeneration. This effect has been demonstrated in multiple animal models of neuro-autoimmunity. IRX4204 is 100% effective in inhibiting transfer of neuro-autoimmunity in a model of transfer of autoimmune Th17 cells into non-diseased mice. In this model the autoimmune neuroinflammation in the recipient mice is solely dependent on the disease-inducing effects of the transferred autoimmune Th17 cells. The observed effects of IRX4204 in this model clearly demonstrate its inhibitory effects on Th17-mediated neuro-autoimmunity. IRX4204 inhibits production of the pro-inflammatory cytokine IL-17 by Th17 cells. It also inhibits production of IL-6, another important pro-inflammatory cytokine, by CNS microglia. IRX4204 promotes maturation and growth of oligodendrocyte precursor cells into myelin-producing oligodendrocytes which repair damaged myelin. IRX4204 is neuroprotective, myelin protective, and myelin reparative in mouse models of demyelination. IRX4204 is effective on functional and histopathologic outcomes in animal models of multiple sclerosis, Parkinson’s disease (PD), and Alzheimer’s disease, in which it has protective activities on dopaminergic and cortical neurons.

IRX4204 has already demonstrated safety and tolerability of oral dosing in phase I and II clinical trials in 85 patients with various cancers and 15 patients with PD for up to 20 months of continuous treatment. In PD patients, IRX4204 demonstrated brain penetrance, and improvement of motor functions in open label assessments.

Based on its balance-restoring anti-autoimmune effects on Treg and Th17 cells, suppression of pro-autoimmune production of IL-17 by Th17 cells, suppression of pro-inflammatory production of IL-6 by microglia cells, effects promoting oligodendrocyte maturation, myelin protection, and, myelin repair, neuroprotective effects in multiple animal models of neuro-autoimmunity and neurodegeneration, and demonstration of safety of dosing in 100 humans, IRX4204 warrants clinical testing in ALS patients. The company is in the planning stages for conduct of a phase II clinical trial of IRX4204 in ALS patients.

Dr. Vuligonda stated, “Our results identify a new approach to potentially treating ALS by inhibiting multiple pathophysiologic processes with IRX4204, including autoimmune neuroinflammation, demyelination, and neuronal death.”

Dr. Sanders stated, “IRX4204 has already been demonstrated to be safe and well tolerated when administered to humans. It has potential to be an effective treatment for slowing the progression of disability and delaying mortality of ALS patients. The company plans to advance IRX4204 into clinical trials in ALS patients, to evaluate its potential to be a safe and effective treatment for this currently inadequately treated, highly burdensome, and uniformly fatal disease.”

About Io Therapeutics: Io Therapeutics, Inc. is a privately held company headquartered in Spring, Texas. More information on Io Therapeutics and its product development programs is available on the company’s web site: www.io-therapeutics.com

Forward Looking Statements: This news release contains “forward-looking statements” within the meaning of the safe harbor provisions of the United States Private Securities Litigation Reform Act of 1995.

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