NEURELIS ANNOUNCES FIRST HEALTHY SUBJECTS DOSED IN INVESTIGATIONAL STUDY OF NRL-1049, A RHO KINASE (ROCK) INHIBITOR WITH POTENTIAL TO TREAT CEREBRAL CAVERNOUS MALFORMATIONS
First in human study will evaluate safety, tolerability, and pharmacokinetic parameters of NRL-1049, a Rho kinase (ROCK) inhibitor
Excerpt from the Press Release:
SAN DIEGO, Calif., March 24, 2023 /PRNewswire/ — Neurelis, Inc., announced the first two cohorts dosed in the investigational clinical trial of NRL-1049, a Rho kinase (ROCK) inhibitor, 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 first-in-human study designed to establish safety, tolerability, and pharmacokinetic parameters of NRL-1049.
“We are excited to initiate this study because it will build upon the preclinical activity and tolerability observed in preclinical studies. People afflicted with CCMs have limited treatment options, which include management of seizures through anti-seizure medications and in limited selected cases surgical intervention to remove lesions,” said Founder and CEO of Neurelis, Craig Chambliss. “If successful, NRL-1049 has the potential to be the first FDA approved treatment for CCMs.”
An analysis of NRL-1049 preclinical data showed that transgenic mice showing the mutations for familial CCMs treated with NRL-1049 showed a significant dose-dependent reduction in lesion volume and a reduction in hemorrhage (iron deposition) near lesions at all doses after treatment compared with placebo.
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