Neurelis, Inc., announced the successful filing of its investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for NRL-1049, a Rho kinase (ROCK) inhibitor with a potential treatment for people living with cerebral cavernous malformations (CCM), a disease characterized by abnormally enlarged capillary cavities in the brain and spinal cord.
“This is a significant milestone for Neurelis and represents an opportunity to further explore the clinical utility of NRL-1049 for the treatment of cerebral cavernous malformations,” said Adrian L. Rabinowicz, M.D., Neurelis SVP Clinical Development and Medical Affairs. “Following favorable preclinical study results, we are excited to advance our mission to introduce new treatment options for patients with critical unmet medical needs.”
Following the IND filing, the Company plans to initiate a clinical study evaluating the safety of NRL-1049 in early 2023.
An analysis of NRL-1049 preclinical data was presented at the Neuroscience 2022 Conference (San Diego, CA, November 12- 16, 2022), hosted by the Society for Neuroscience (SfN). The analysis showed that in two different animal models of brain injury, NRL-1049 protected the brain, possibly through maintaining the integrity of the blood-brain barrier through a decrease in ROCK2 activation. This protection was associated with lower rates of brain edema in both animal models. A lower seizure rate in the cold injury animals and decreased intracerebral hemorrhage in the model of stroke were also observed.
“We are excited that the preclinical studies have shown that NRL-1049 is safe and efficacious in animal models,” said Enrique Carrazana, M.D., Neurelis Chief Medical Officer. “Based on the significance of these data, Neurelis is developing further studies in humans to assess the safety of NRL-1049 in the treatment of chronic and acute conditions that affect the functioning of the blood-brain barrier. If successfully developed and approved, NRL-1049 has the potential to provide relief to people suffering from CCMs.”