About Inhibikase

Inhibikase is a clinical-stage specialty pharmaceutical company focused on developing and commercializing small-molecule kinase inhibitor therapeutics for the safe and effective treatment of CNS diseases, including diseases 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 that target non-CNS diseases. Using this established knowledge as a starting point, we pursue the clinically validated kinases 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 CNS 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 CNS 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 commercialization pipeline includes:

  • A disease-modifying therapy that reverses the course of Parkinson’s disease
  • A companion therapeutic that 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. 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 and third small-molecule product candidates, IkT-001Pro and IkT-01427, are Abl kinase inhibitors that suppress 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-001Pro and IkT-01427 are positioned as first-in-class companion therapeutics 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 CNS 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 CNS diseases—yet the development of kinase-targeted therapies for CNS diseases remains a challenge due to specific issues associated with developing and commercializing drugs that treat in the brain. We are taking a risk-reducing approach to CNS drug development by leveraging established understanding of marketed kinase inhibitor drugs to develop a new generation of targeted CNS therapies that may be administered chronically and systemically to patients with infectious or neurodegenerative CNS diseases.

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

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