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Technology

Novel Product Candidates for the Treatment of Neurological Diseases

Inhibikase Therapeutics pursues the discovery and characterization of the biochemistry of disease inside and outside of the brain. With a deep commitment to scientific discovery and translation, we pursue any path necessary to target key biochemical checkpoints to disrupt the disease process. Our current focus is the analysis and disruption of neurodegenerative pathways in the brain and gastrointestinal tract that lead to Parkinson’s and related diseases. 

Key technologies include:

  • RAMP™: Re-engineering Approach with Metabolism Preserved, a method of designing novel medications by learning from clinically evaluated and/or marketed drug products to build in desirable, clinically validated safety or pharmacology characteristics from the template into new drug substances.  
  • Prodrug delivery methods: Novel prodrug linkers that enhance drug absorption, delivery and/or suppress undesired on-dosing side effects.
View Application of RAMP

RAMP™ Drug Innovation Engine

Delivering First-in-class Small-molecule Product Candidates

Our Re-engineering Approach with Metabolism Preserved (RAMP™) drug innovation engine enables us to design and develop novel small-molecule product candidates, leveraging the clinical experience gained from currently marketed or clinically evaluated small molecule drugs that target CNS or non-CNS diseases. Using preexisting chemical entities as a template, we pursue clinically validated targets to create first-in-class small-molecule product candidates with predicted human safety profiles to treat neurodegenerative diseases.

RAMP Technology Enables:

  • Safety enhancements achieved through chemical modifications that could lead. to dose reduction, enabling chronic administration with minimized side effects.
  • Preservation of the favorable side-effect profile of the “template” medication, achieved through medicinal chemistry changes to preserve how the drug is metabolized in the body.
  • The ability to generate multiple novel/improved drug molecules that address a range of therapeutic areas from the same clinically validated kinase inhibitor.
  • Medicinal chemistry insights spanning protein kinase families.
  • Medicinal chemistry and structural engineering techniques for generating kinase inhibitors with greater selectivity and optimized pharmacological properties for drugs to treat neurodegeneration.
  • Discovery workflow attuned to novel pharmacological outcomes.
The drug innovation engine starts with a kinase inhibitor that has a safe profile. Then we re-engineer it with specific criteria to enable the same or better efficacy at lower dose. After that, we generate a family of novel drug molecules with improved potency that retains its safety profile.

RAMP™ Enables the Discovery of Novel Candidates

Treating Parkinson’s and Related Diseases with Small-Molecule Kinase Inhibitors

We are utilizing our RAMP™ discovery engine to develop a new class of protein kinase inhibitors for the potential treatment of kinase-sensitive CNS diseases. These candidates could be more potent and safer than commercially marketed kinase inhibitors and be administered chronically and systemically.

IkT-148009

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. IkT-148009 is the first oral medication shown to correlate clearance of alpha-synuclein toxicity with functional recovery in a living organism. Alpha-synuclein is a non-essential protein in the body that plays a role in neurotransmission inside and outside of the brain. In the disease state, alpha-synuclein is ‘misfolded,’ resulting in the formation of fibrillary alpha-synuclein aggregates that ultimately become pathologic and initiate the disease process in specific areas of the brain and gastrointestinal tract.

View IkT-148009

IkT-01427

An antiviral companion therapeutic to be taken alongside a primary, immunosuppressive therapy that can lead to Progressive Multifocal Leukoencephalopathy (PML). PML is a rare, but rapidly fatal brain infection, caused by the migration of the John Cunningham virus (JCV) from reservoirs in the body into the brain. JCV migration is known to be stimulated by more than 15 currently marketed therapies that are used to treat cancer and autoimmune diseases, such as multiple sclerosis.

View IkT-01427

Additional Compounds

We are actively developing novel kinase inhibitor treatments for additional Parkinson’s-related disorders, including multiple system atrophy (MSA), a neurodegenerative disorder that shares the hallmarks of c-Abl activation and alpha-synuclein pathology with Parkinson’s disease. Future programs will include Dementia with Lewy Body (DLB), a Parkinson’s-like disease characterized by alpha-synuclein aggregates that primarily lead to cognitive decline without a significant movement disorder component.

Intellectual Property

Our intellectual property includes patents issued or pending, as well as know-how related to the composition of matter and broad use of our technologies across neurodegenerative diseases inside and outside of the brain. We have carved out a broad and deep intellectual property position that enables the development of the Company’s internal product pipeline and supports value-creating partnerships with biotechnology and pharmaceutical companies.

Our Prodrug Delivery Technology

Our prodrug technology platform enables potentially greater control over the non-hematological side effects common to kinase inhibitor therapies, such as nausea or diarrhea, and it may improve drug delivery into the target tissue. Using a proprietary set of linkers and attachment points, our oral prodrugs are absorbed intact and then fall apart into the active ingredient and the linker. We believe this approach could lead to kinase inhibitors that are safer and better tolerated, with the potential to treat a variety of indications.

Prodrug process steps

Application of RAMP™ Drug Innovation Engine

Using our drug innovation engine is the basis for discovery of our product candidates and we have successfully developed several small-molecule medications with the potential to treat multiple different diseases through the same target approach.

Explore Our Pipeline