BioAxone is a clinical-stage biopharmaceutical company that is transforming treatments for neurotrauma and neurovascular disorders. The company has developed innovative therapies based on a deep understanding of axon regeneration and neuronal signaling pathways.

BioAxone research and clinical development work is focused in the following main areas:

  • The company’s lead drug for spinal cord injuries, VX-210 (formerly Cethrin™), has been licensed to Vertex Pharmaceuticals. It is in a Phase 2b/3 SPRING clinical trial for treatment of acute cervical spinal cord injury.
  • BioAxone has an IND-ready ROCK-2 selective, oral kinase inhibitor, BA-1049, for treatment of neurovascular disorders. ROCK2 signaling is overactivated in endothelial cells in hereditary angioma, a disease where benign vascular malformations are “leaky” and can cause stroke. Similarly, in acute ischemic stroke, ROCK2 is hyperactivated in brain vascular endothelial cells causing risk of hemorrhage. BA-1049 repairs vascular permeability following stroke and reduces reperfusion injury.
  • A preclinical drug development program is focused on the intrinsic barriers to regeneration brought on by neuronal aging. BioAxone has developed a novel self-delivering RNA interference compound that reduces expression of PTEN, a protein that blocks axon regeneration in adult neurons.
  • ROCK inhibitors offer a new therapy for glaucoma, the second leading cause of blindness in the United States. BioAxone is developing novel ROCK2-selective inhibitors to improve therapies available to patients with potential to reduces progression of disease.