Small-molecule Medications with World-class Potential

Inhibikase Therapeutics’ first product candidates were generated using our Re-engineering Approach with Metabolism Preserved (RAMP™) drug innovation engine use the same targeting strategy—blocking the activation of Abl (c-Abl) kinases—as the basis for small-molecule medications with potential for two CNS indications and two autonomic nervous system indications:

  • A disease-modifying therapy that we believe halts the progression and reverses the functional loss arising from Parkinson’s disease in the brain and gastrointestinal (GI) tract while simultaneously nearly clearing the toxic form of alpha-synuclein believed to be the cause of the disease.
  • A treatment for stable-phase Chronic Myelogenous Leukemia (CML) with a prodrug of the anticancer agent, imatinib, that we believe will have a superior safety profile; improve absorption; lower the dose required for efficacy; and suppress ongoing nausea, diarrhea, and vomiting associated with imatinib therapy.
  • A companion therapeutic that we believe may remove the risk of Progressive Multifocal Leukoencephalopathy (PML), a rare but rapidly fatal brain infection, by blocking the production of John Cunningham virus (JCV), the cause of PML. Development of JCV has been linked to treatment with more than 15 medications that are used to treat cancer and autoimmune diseases, such as multiple sclerosis.
  • We are also exploring development opportunities for the treatment of Multiple System Atrophy (MSA), a neurodegenerative movement disorder which shares the hallmarks of c-Abl activation and alpha-synculein chemical modification with Parkinson’s Disease, and Dementia with Lewy Body (DLB), a Parkinson’s-like disease characterized by alpha-synuclein aggregates.

Abl Kinases and CNS Diseases

Abl kinases have been shown to play a critical role in monitoring insults to brain neurons and regulating biological pathways that are associated with neuroprotection. In addition, recent research has demonstrated that Abl kinases are essential checkpoint regulators that play a central role in Parkinson’s disease initiation and progression.

The breakthrough cancer treatment, imatinib (Gleevec®), was the first FDA-approved Abl kinase inhibitor. Because of the way imatinib is broken down in the body, imatinib’s safety is correlated with dose. Applying our RAMP technology to imatinib, Inhibikase has generated novel chemical entities that have:

  • Up to 18-fold enhanced potency against wildtype c-Abl
  • Nearly identical route of metabolism to imatinib, thereby preserving the relationship between administered dose, and drug safety and frequency of adverse events

We believe these molecules have the potential to enable dose reduction with a concomitantly improved safety profile, resulting in a safer Abl kinase inhibitor that may be chronically and systemically administered for the treatment of our initial targeted CNS indications.

Abl Kinases and Autonomic Nervous System Diseases

Parkinson’s patients have a series of disorders that may represent the earliest manifestations of the disease. These include difficulty in swallowing (dysphagia) and difficulty in passing solid waste (neurogenic constipation), both due to a loss of the neurons necessary for these functions to occur on their own. We believe that our recent analysis of GI functional disorders in preclinical models with our lead product candidate for Parkinson’s, IkT-148009, suggests that we can halt the progression of neurodegeneration in the GI tract and restore functional loss. 

RAMP™ Drug Innovation Engine

We are advancing a new class of small-molecule product candidates using our Re-engineering Approach with Metabolism Preserved (RAMPTM) drug innovation engine that target specific protein kinases for the treatment of neurodegenerative diseases.

About RAMP™ Drug Innovation Engine

Our Scientific Publications

We have a number of publications accessible regarding various topics, including progressive multifocal leukoencephalopathy (PML), Parkinson’s disease, and other applications in infectious diseases.

View Publications