NEW HAVEN, Conn., June 19, 2019 /PRNewswire/ -- ReNetX Bio, Inc., a leading biotechnology company committed to reversing disease and damage for patients suffering from central nervous system disorders, has announced that the U.S. Food and Drug Administration (FDA) has allowed the Company's Investigational New Drug (IND) application for its lead drug candidate, fusion protein AXER-204.
Recruitment will begin immediately for the Phase 1 first-in-human trial of AXER-204 for the treatment of chronic spinal cord injury (SCI). The "RESET" Trial will assess safety, tolerability, pharmacokinetics, and efficacy in patients at leading SCI treatment centers across the U.S. An estimated 300,000 people are currently living with chronic SCI in the U.S., and there is currently no approved therapeutic to restore sensory or motor function after injury. An effective therapeutic to promote recovery of function after damage would be the first of its kind to address a significant and completely unmet medical need.
Axonal loss is a common, yet unaddressed pathology, in a broad range of chronic and acute diseases of the central and ocular nervous systems. The Company's technology relies on blocking inhibitory proteins to promote regrowth of neurons and was developed by ReNetX Founder and Scientific Advisor Stephen Strittmatter, MD, PhD, Vincent Coates Professor of Neurology at Yale University. Three proteins were identified as key inhibitors preventing neural regrowth due to binding with NgR1 (Nogo Pathway). In preclinical studies with AXER-204, these inhibitors are blocked and neural plasticity as well as new axonal growth is triggered. These new neural connections enable rewiring and reversal of damage in the central nervous system.
AXER-204 has generated positive results in numerous preclinical disease models which have been independently validated in leading academic and industrial labs. Studies have indicated that treatment with ReNetX's proprietary therapeutic AXER-204 resulted in significant regrowth of nerve fibers and increased functionality– including axonal regeneration, axonal sprouting, and synaptic plasticity – even many months after injury or damage. In addition, the therapy has been shown to be safe with no toxicological findings.
The company has received major support from leading organizations across the world, including in a collaboration with the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH ) through the BrIDGs Program (Bridging Interventional Development Gaps), and from the Wings for Life Spinal Cord Research Foundation to advance the program into the clinic. "We recognize the unmet need, we are encouraged by the success our therapeutic has shown to date, and we hope that AXER-204 will deliver a much-needed therapeutic option for patients," says Dr. Strittmatter.
"We are grateful for the partners that have supported our efforts to achieve this major milestone and are excited to advance an entirely new approach for patients to restore function to damaged nerve fibers in the central nervous system," says ReNetX CEO, Erika R. Smith. "This breakthrough therapy has the potential to reverse conditions that, until now, were considered untreatable," adds the company's lead investor Gregory P. Ho, President, Spring Mountain Capital.