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Evaluation of the RXR Agonist Compound IRX4204

Study Rationale:
Drugs that activate the retinoid X nuclear receptor (RXR) have been reported to promote survival of dopaminergic neurons in previously published pre-clinical studies. IRX4204 is a novel, second-generation RXR activating compound, which has been demonstrated to be highly potent and selective for activating RXR. IRX4204 has already been tested in humans as a treatment for various types of cancers. Because the compound is already at a clinical testing stage, and prior data supports that a drug with RXR activating effects may be effective in Parkinson’s disease (PD), this project sought to provide data potentially supporting the conduct of clinical trials of IRX4204 in patients with PD.

The hypotheses tested are that IRX4204 is effective in treating a pre-clinical model of PD, and that it would penetrate the brains of the models following oral administration.

Study Design:
A contract research laboratory tested IRX4204 and its brain penetration in a pre-clinical model of PD.

Impact on Diagnosis/Treatment of Parkinson’s Disease:             
A drug that promotes dopaminergic neuron survival has potential for being a disease-modifying treatment for PD. To date, no drugs have been demonstrated to modify the course of PD.

Next Steps for Development:
Io Therapeutics has filed an investigational new drug application with the U.S. Food and Drug Administration to be allowed to conduct the first clinical study of IRX4204 in people with PD.


  • Rosh Chandraratna, PhD, MBA

    Santa Ana, CA United States

  • Martin E. Sanders, MD

    Santa Ana, CA United States

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