Inhibikase Therapeutics, Inc.
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Dr. Milton Werner is the President and Chief Executive Officer of Inhibikase Therapeutics, a company developing novel protein kinase inhibitor therapeutics to treat neurodegenerative disease and viral infection inside and outside of the brain. Previously, Dr. Werner served as Vice President of Research at Celtaxsys, a cell-free immunotherapeutics company. From September 1996 until June 2007, Dr. Werner was a Head of the Laboratory of Molecular Biophysics at The Rockefeller University in New York City. Throughout his scientific career, Dr. Werner has been an innovator integrating chemistry, physics, and biology into a comprehensive approach to solving problems in medicine, including an explanation of the origin of “maleness” in humans, the mechanistic basis of several forms of leukemia and lymphoma and, more recently, the development of therapeutics that can halt and potentially reverse functional loss in neurodegenerative disease. Dr. Werner is the author or co-author of more than 70 research articles, reviews, and book chapters and has given lectures on his research work throughout the world. He is the recipient of numerous private and public research grants totaling more than $30 million. He is the recipient of several awards, including the Naito Memorial Foundation Prize, the Young Investigator Award from the Sidney Kimmel Cancer Foundation, the Research Chair from the Brain Tumor Society, and a $1 million Distinguished Young Scholars in Medical Research Award from the W. M. Keck Foundation. Dr. Werner received his Doctor of Philosophy in Chemistry from the University of California, Berkeley, and his Bachelor of Science in Biochemistry from the University of Southern California, and he was an NIH intramural postdoctoral fellow prior to his tenure at the Rockefeller University.

Presenter of 1 Presentation

PARKINSON’S DISEASE MODIFICATION THROUGH ABL KINASE INHIBITION

Session Type
SYMPOSIUM
Date
Fri, 18.03.2022
Session Time
05:15 PM - 07:15 PM
Room
ONSITE: 133-134
Lecture Time
05:45 PM - 06:00 PM

Abstract

Aims

The Abelson Tyrosine Kinase (c-Abl) is implicated in initiating and driving progression of neurodegeneration in Parkinson’s disease and other synucleinopathies. Recent advances in modeling Parkinson’s disease in mice suggested that c-Abl activation may be required for disease initiation and progression to occur, and therefore inhibition of c-Abl could be a strategy for disease-modification of Parkinson’s.

Methods

The novel c-Abl inhibitor IkT-148009 was dosed orally once daily at human equivalent doses in therapeutic models of inherited and sporadic Parkinson’s disease in mice. The outcome of these studies prompted clinical evaluation of IkT-148009 in older healthy subjects, ages 45-70, and in Parkinson’s patients over a wide range of doses. Safety, tolerability and pharmacokinetics were assessed in healthy subjects and assessment of clinical benefit and biomarker changes was performed in Parkinson’s patients.

Results

Animal model studies indicated that therapeutic administration of IkT-148009 was neuroprotective, drove functional recovery and cleared alpha-synuclein aggregates from the brain and GI tract. Clinical studies progressed with no clinically meaningful adverse events to date. Functional assessment of motor function in the brain and GI tract and cognitive function assessments are ongoing as of this writing. Clinical pharmacology indicates that therapeutic dosing levels consistent with animal model efficacy studies can be reached without safety concerns.

Conclusions

IkT-148009 is a promising therapeutic agent for Parkinson’s disease whose mechanism of action suggests it may act as a disease-modifying treatment with the potential for significant clinical benefit.
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