Application of RAMP Drug Innovation Engine

The first product candidates we have generated using our 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 best-in-class potential for two CNS indications and two autonomic nervous system indications:

    • A disease-modifying therapy that we believe reverses the course of Parkinson’s disease
    • A companion therapeutic that we believe removes risk of PML (Progressive Multifocal Leukoencephalopathy), a rare but rapidly fatal brain infection
    • A disease-modifying therapy that we believe reverses the course of dysphagia that arises in Parkinson’s patients
    • A disease-modifying therapy that we believe reverses the course of neurogenic constipation in Parkinson’s patients

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 36-fold enhanced potency
  • 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 pre-clinical models with our lead product candidate for Parkinson’s, IkT-148009, suggests that we can reverse neurodegeneration in the GI tract and restore normal function.

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