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 (http://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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