Spinal Cord Injury
Spinal Cord Injury is a devastating condition for which very few medical treatment options exist. Typically there is surgical intervention to reduce spinal cord compression and to stabilize the bone structure around the cord. Cethrin is used in conjunction with surgery in a hospital setting by the neurosurgeon treating the acutely injured patient. In preclinical studies Cethrin shows strong pro-regenerative activity and reduces tissue damage. Having completed a Phase 1/2a clinical study, Cethrin is positioned to be the first drug to reach the market that offers significant functional improvement for patients with spinal cord injury (SCI).
Optic nerve ischemia and traumatic optic neuropathy
Permanent loss of vision can result when the optic nerve is injured, which can happen in neurotrauma (traumatic optic neuropathy), ischemic injury (optic nerve stroke) or autoimmune disease (autoimmune optic neuropathy). A single injection of Cethrin can significantly reduce retinal ganglion cell death in rat models of optic nerve trauma and retinal disease. There are no effective drugs available to treat traumatic and ischemic optic neuropathies. With strong scientific proof of concept, BioAxone is preparing for a Phase 1/2a clinical study to investigate Cethrin’s safety and tolerability in optic neuropathy.
Spinal surgery and disc repair
The vast majority of spinal surgeries are for diseases or pathologies that include herniated discs, tumors, vascular abnormalities, scoliosis, congenital deformities, and inflammation of the spinal column (ankylosing spondylitis). The prevalence of herniated disc in the US is approximately 200,000 cases a year. There currently is no adjunct therapy for regeneration or protection of neurons following spinal cord surgery involving laminectomy. Preclinical studies provide strong evidence that Cethrin used in spinal surgery may aid bone repair. Consequently, demonstrating more widespread benefits of Cethrin for spinal surgery would represent a significant potential for helping patients recover faster and with fewer complications.
Glaucoma affects over 70M people worldwide. In America there are 2.4M glaucoma patients, and glaucoma is the second leading cause of blindness worldwide. The most common types of glaucoma are associated with high intraocular pressure (IOP) and most medical treatments are aimed at reducing IOP. Approximately 30% of patients in North America suffer from “normal tension glaucoma” in which there is visual loss and degeneration in the absence of high intraocular pressure. Thus, current therapeutic strategies for glaucoma are insufficient and new medicines that slow disease progression are urgently needed. To develop a drug to treat glaucoma, BioAxone has produced approximately 50 small molecule inhibitors of Rho kinase. Rho kinase (ROCK) is a signaling kinase downstream of Rho and the kinase is activated by active Rho. BA-1049, the lead drug candidate is specific for Rho kinase II (ROCK II) has shown potential efficacy in reducing IOP, and also reduces death of retinal ganglion cells that die progressively in glaucoma.