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 drugs with best-in-class potential for two CNS indications:

  • 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

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 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:

  • 40-fold enhanced potency
  • 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.

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