AXER-204

Spinal cord injuries are serious issues impacting hundreds of thousands of people in the United States. It’s estimated there are more than 17,000 new cases of spinal cord injuries each year, which can cause permanent damage in patients. With the prevalence of such injuries in the U.S., as well as the rest of the world, multiple companies are focusing their efforts on developing regenerative medicines to treat these injuries, which can result in permanent changes in strength, sensation and other body functions below the site of the injury, according to the Mayo Clinic. In addition to profound persistent neurological impairment, patients with chronic SCI suffer a variety of other health consequences including respiratory complications, cardiovascular complications, urinary and bowel complications, spasticity, pain syndromes, pressure ulcers, osteoporosis and bone fractures. According to the National Spinal Cord Injury Statistical Center, the leading cause of spinal cord injury is vehicle crashed, followed closely by falls. Other common causes of spinal cord injuries include sports injuries and acts of violence, such as gunshot wounds. Developing therapeutics to help patients regain full function are underway. BioSpace takes a look at recent work undertaken by researchers in the field: Keio University – Japanese researchers at Tokyo’s Keio University are aiming to use induced pluripotent stem (iPS) cells to treat spinal cord injuries. Until this particular study, the use of induced pluripotent stem cells had only been allowed in treating, eyes, hearts, cranial nerves and blood platelets of patients as part of regenerative medicine research. The trial will attempt to take the induced pluripotent stem cells and grow them into nerve cells. The Japanese research team will transplant two million of these new nerve cells into the injured areas of each patient via injection in hopes of inducing a response. The patients will also be given immunosuppressant drugs to prevent a rejection of the transplanted cells. InVivo Therapeutics – In February, Massachusetts-based InVivo published research data in the Journal of Neurosurgery: Spine describing the previously presented complete six-month primary endpoint results from the company's single-arm INSPIRE 1.0 study. Invivo’s biodegradable device is surgically implanted in patients who are dealing with a complete thoracic AIS A spinal cord injury. The article pointed to data that showed a probable benefit of a bioresorbable polymer scaffold for safety and neurological recovery in patients with complete thoracic spinal cord injury. InVivo has officially closed the INSPIRE 1.0 study and is actively enrolling patients into a second pivotal clinical study of the Neuro-Spinal Scaffold in patients with acute SCI, dubbed INSPIRE 2.0. Lineage Cell Therapeutics – California-based Lineage Cell Therapeutics, Inc. provided an update on its oligodendrocyte progenitor cell (OPC) therapy for the treatment of acute spinal cord injury in December. The company said it developed enhanced differentiation process, leading to major improvements in production and quality of its OPC1 cell therapy drug product. Improvements included ready-to-inject formulation of OPC1, which enables clinical use at a much larger number of spinal cord treatment centers, accelerating enrollment for a larger and potentially registrational clinical trial, the company said. The company also saw improvements in OPC1 functional activity, as assessed by cellular migration and secretion of key growth factors. OPC1 is an oligodendrocyte progenitor cell (OPC) transplant therapy designed to provide clinically meaningful improvements in motor recovery in individuals with acute spinal cord injuries. ReNetx Bio – In January 2020, Connecticut-based ReNetX Bio received Fast Track designation from the FDA for its clinical asset AXER-204 for the potential treatment of Chronic Spinal Cord Injury. ReNetX Bio is currently conducting a Phase I/II clinical trial dubbed RESET in patients with SCI. Topline results are expected to be announced this year. AXER-204 is designed to block axonal growth inhibitors in the central nervous system to promote neural repair. While Damage to axons in the CNS is permanent, ReNetx believes damaged and remaining undamaged axons provide an opportunity for restoration of function if new connections can be grown. In preclinical studies, AXER-204 has been shown to promote axonal growth and recovery of neurologic function in the central nervous system by acting as a decoy and binding the 3 key inhibitors Nogo-A, MAG, and OMgp. This interrupts their activation of NgR1 thereby promoting axonal growth. MicroCures – New York-based MicroCures’ lead product is siFi2. Preclinical research suggests that siFi2 may help overcome these natural inhibitors and trigger successful axon regeneration and reattachment at the site of injury. In preclinical studies of spinal cord injury in rats, MicroCures has demonstrated that treatment with siFi2 allowed axon growth to occur through the injury’s inhibitory barriers. Researchers did not witness similar axon growth among control animals, according to the company’s website.