About Inhibikase

Inhibikase is a company focused on developing and commercializing small-molecule kinase inhibitor therapeutics for the safe and effective treatment of neurodegeneration inside and outside of the brain.

Our proprietary RAMP™ (Re-engineering Approach with Metabolism Preserved) drug innovation engine enables us to design and develop novel small-molecule product candidates that leverage the clinical experience gained from currently marketed kinase inhibitor drugs and imprints the properties we wish to preserve onto new molecular entities. Using this established knowledge as a starting point, we pursue the clinically validated kinase inhibitors as targets for the creation of novel drugs with enhanced efficacy and improved safety that may be administered chronically and systemically to treat kinase inhibitor-sensitive neurological and non-neurological diseases.

Kinase inhibitors that block the abnormal activation of protein kinases are a well-established drug class for cancer treatment. Inhibikase is leveraging established knowledge about clinically validated kinase inhibitor drugs that target non-CNS diseases to establish a new class of targeted therapies for neurodegenerative and other diseases.

Our initial focus is on the development of product candidates we have generated using RAMP that use the same targeting strategy—blocking the activation of Abelson tyrosine kinases (Abl kinases)—to address two distinct unmet needs for life-threatening infectious and neurodegenerative CNS diseases. Our product candidates include:

  • A disease-modifying therapy that we believe reverses the course of Parkinson’s disease in the brain and gastrointestinal (GI) tract
  • A near-term commercial opportunity in stable-phase CML (Chronic Myelogenous Leukemia) 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 the ongoing nausea, diarrhea, and vomiting associated with imatinib therapy
  • A companion therapeutic that we believe removes risk of PML (Progressive Multifocal Leukoencephalopathy), a rare but rapidly fatal brain infection induced by more than 15 medications that are used to treat cancer and autoimmune diseases, such as multiple sclerosis

Our lead small-molecule product candidate, IkT-148009, is an Abl kinase inhibitor that targets underlying disease mechanisms to reverse the course of Parkinson’s disease and the GI complications of Parkinson’s disease. We believe that IkT-148009 is the only disease-modifying drug in development for Parkinson’s disease that is focused on a clinically validated target and that IkT-148009 has the potential to be the first disease-modifying therapy for Parkinson’s disease that blocks a checkpoint on the pathway that drives Parkinson’s disease progression.

Our second small-molecule product candidate, IkT-001Pro, is an Abl kinase inhibitor prodrug of the anticancer agent, imatinib (marketed as Gleevec®). IkT-001Pro alters the way imatinib is absorbed in the GI tract, which we believe suppresses GI distress experienced by up to one-half of Gleevec® patients daily.  IkT-001Pro also delivers more imatinib into the tissues due to its altered absorption characteristics, thereby potentially reducing the dose required to achieve efficacy for imatinib-sensitive cancers.  By lowering the dose, we believe IkT-001Pro may have an improved safety profile, which may reduce the daily burden of therapy for Gleevec® patients.

Our third small-molecule product candidate, IkT-01427, is an Abl kinase inhibitor that we believe suppresses the reproduction of John Cunningham (JC) virus in the body, thereby removing the sole risk factor for the cause of PML (Progressive Multifocal Leukoencephalopathy). PML is a rare disease that causes a rapidly fatal brain infection. IkT-01427 is positioned as first-in-class companion therapeutic for any drug that carries a PML black box warning, including drugs for multiple sclerosis, Crohn’s disease, ulcerative colitis, hematological cancers, and organ transplantation.

The development of new therapies for neurodegenerative diseases has lagged behind other drug development areas, despite a growing global need for novel therapeutic approaches. A burgeoning body of research is revealing the critical roles protein kinases play in the molecular mechanisms underlying neurodegenerative diseases—yet the development of kinase-targeted therapies for neurodegenerative diseases remains a challenge due to specific issues associated with developing and commercializing drugs that treat neurodegeneration inside and outside of the brain. We are taking a risk-reducing approach to neurodegenerative drug development by leveraging established understanding of marketed kinase inhibitor drugs to develop a new generation of targeted therapies that may be administered chronically and systemically to patients with infectious or neurodegenerative diseases.

We believe that we are at the forefront of improving treatment outcomes in kinase-sensitive neurodegenerative diseases.

Back to Top