Neurelis has dosed the first two cohorts in the investigational clinical trial of NRL-1049 to treat cerebral cavernous malformations (CCM) in patients.

The first in human, double-blind, placebo-controlled, randomised, dose-escalation, single ascending dose clinical trial has been designed to establish the tolerability, pharmacokinetic parameters, and safety of NRL-1049 in healthy participants.

Its primary objectives are to collect data on tolerability and clinical safety and to determine the maximum tolerated dose (MTD) of a single oral NRL-1049 dose.

Neurelis founder and CEO Craig Chambliss said: “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.

“If successful, NRL-1049 has the potential to be the first FDA approved treatment for CCMs.”

The investigational pre-clinical stage small molecule, Rho kinase (ROCK) inhibitor NRL-1049 has been designed to potentially reduce new lesions accumulation, and alleviate neurological symptoms that are associated with CCMs, a disease which is characterised by abnormally enlarged capillary cavities in the spinal cord and brain.

The company licensed the ROCK inhibitor from BioAxone BioSciences in June 2021.

The US Food and Drug Administration (FDA) accepted the investigational new drug (IND) application for NRL-1049 earlier this year.

In preclinical data, NRL-1049 demonstrated a significant dose-dependent reduction in lesion volume as well as in haemorrhage (iron deposition) near lesions after treatment at all doses against a placebo.