Unlocking the Key to Parkinson’s Treatment: Inhibikase Therapeutics Reveals c-Abl as a Promising Target

Inhibikase Therapeutics, Inc. has made a breakthrough in the battle against Parkinson’s and related disorders. The company recently published a study in Science Translational Medicine which describes the potential of its protein kinase inhibitor therapeutic, IkT-148009, as a groundbreaking disease-modifying treatment. The study, titled “The c-Abl inhibitor IkT-148009 suppresses neurodegeneration in mouse models of heritable and sporadic Parkinson’s disease,” was published on January 18, 2023 and demonstrates the tremendous promise of this new therapy.

In a groundbreaking new study published by our collaborators, genetic deletion of c-Abl blocked neurodegeneration in animals with alpha-synuclein plaques, suggesting a critical role of c-Abl in the Parkinson’s disease process. Our research further highlighted the neurodegenerative functional screen that led to the discovery of IkT-148009, a drug that has the potential to halt disease progression, drive functional recovery, and protect neurons in the brain from degradation – all at an exposure consistent with a 50 mg oral dose in PD patients. Remarkably, this therapeutic benefit was accompanied by significant reduction of alpha-synuclein pathology in the brain – a long-awaited development in Parkinson’s treatment. These data demonstrate the promise of IkT-14809 as a disease-modifying therapy and support continued clinical development of this groundbreaking new drug.

Some of the key takeaways from the event included: deepening our understanding of the topic, discovering new strategies to tackle the problem, and gaining new insights into the issue. Attendees were able to network and share ideas, while also learning from experts in the field. The event provided a valuable opportunity to explore the issue in greater depth and build a better understanding of how to approach it.

The c-Abl family of enzymes displays a remarkable level of selectivity, as exemplified by the drug IkT-148009, which selectively targets one member of the family. This selectivity has been demonstrated through a number of studies, revealing the potential of this enzyme family to revolutionize the treatment of certain diseases.

In an effort to identify potential therapeutic agents for suppressing c-Abl activation in the brain, researchers have developed a functional neurodegenerative screen – and the results were remarkable! The screen identified IkT-148009 as a promising therapeutic agent, providing hope for patients suffering from neurological diseases.

The therapeutic efficacy of IkT-148009 as a disease-modifying treatment has been demonstrated in animal models of Parkinson’s Disease (PD) at the same doses being evaluated in the 201 trial. This promising treatment has the potential to significantly improve the lives of those suffering from PD.

Gaining an understanding of how misfolded proteins form aggregates and cause neurodegenerative diseases has long been an area of intense study. Recent research has shed light on how these misfolded proteins interact with healthy proteins, leading to the accumulation of toxic aggregates that can damage nerve cells and eventually cause progressive deterioration of the nervous system. This knowledge helps us to better understand and target the root causes of these devastating diseases.

By using IkT-148009 to inhibit c-Abl, a protein involved in the formation of aggregates, researchers have been able to make a major breakthrough in treating multiple models of human diseases – correlating the removal of protein aggregates with functional recovery. This has been a long sought-after goal, and these findings have the potential to revolutionize treatments for these diseases.

About Inhibikase

Inhibikase Therapeutics, Inc. (Nasdaq: IKT) is a pioneering, clinical-stage pharmaceutical company developing cutting-edge treatments for Parkinson’s disease and related disorders. Its multi-therapeutic pipeline is designed to address neurodegeneration, targeting both the brain and GI tract, as well as orphan indications related to Parkinson’s, such as Multiple System Atrophy. Its lead program, IkT-148009, is an Abelson Tyrosine Kinase (c-Abl) inhibitor, designed to provide a better patient experience with fewer on-dosing side-effects. In addition, the RAMP™ medicinal chemistry program has identified a number of follow-on compounds to IkT-148009 that may be applied to other cognitive and motor function diseases of the brain. With offices in Atlanta, Georgia and Boston, Massachusetts, Inhibikase is a leader in the field of innovative, life-saving treatments.

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