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Cerebral cavernous malformations (CCMs) are an orphan and rare disease characterized by abnormally enlarged capillary cavities, most commonly found in the cerebral cortex, brainstem, and the spinal cord. CCMs can present as a single lesion or as multiple lesions. Depending on lesion size and location, CCMs can be "clinically silent" or can present with clinical symptoms ranging from headaches to focal neurological deficits, seizures, and cerebral hemorrhage. People afflicted with CCMs have limited treatment options, which include management of seizures through anti-epileptic drugs and surgical intervention to remove lesions (eg, microsurgical resection, stereotactic radiosurgery).


There are currently no FDA-approved treatments for CCMs. Most cavernous malformations are conservatively managed by observing for changes in appearance, recent hemorrhages, or other clinical symptoms.


In June 2021, we licensed NRL-1049 from BioAxone BioSciences, Inc., which is an investigational Phase 1 stage small molecule, Rho kinase (ROCK) inhibitor designed to potentially reduce the accumulation of new lesions, as well as alleviate neurological symptoms associated with CCMs.* We believe that NRL-1049, if successfully developed and approved, has the potential to address people suffering from CCMs.

*NRL-1049 is a product candidate that has not been approved by the U.S. Food and Drug Administration for any indication.