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
16 July 2024 - 2:26PM
UK Regulatory
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.
