RARE Daily

Neurelis Acquires Rights to Portfolio of Compounds Targeting Rare CNS Disorder

June 22, 2021

Neurelis said it has acquired a portfolio of novel ROCK-2 inhibitors that target cerebral cavernous malformations, a rare disorder of the central nervous system that currently has no pharmacologic treatments available, from Bioaxone Biosciences.

Photo: Craig Chambliss, president and CEO of Neurelis

The companies did not disclose financial terms of the transaction.

Cavernous malformations (CCMs) are characterized by abnormally enlarged capillary cavities most commonly found in the cerebral cortex, brainstem, and spinal cord. CCMs can present as a single or as multiple lesions and, depending on size and location, can be clinically silent or can present with clinical symptoms ranging from headaches to focal neurological deficits, seizures, and cerebral hemorrhage. CCMs can arise sporadically or may be inherited.

Familial CCMs account for at least 20 percent of all cases. Mutations in the CCM1-CCM3 genes all cause hyperactivation of the Rho/ROCK signaling pathway in brain vascular endothelial cells (ROCK). Hyperactivation of ROCK then contributes to the loss of endothelial integrity, leading to capillary malformation and eventually to the potential for lesions.

Patients afflicted with CCMs have limited treatment options, which include management of seizures through antiepileptic drugs and surgical intervention to remove lesions. There are currently no approved treatments for CCMs, and most cavernous malformations are conservatively managed by observing for change in appearance, recent hemorrhage, or clinical symptoms.

BioAxone’s lead product, BA-1049, offers an opportunity to treat CCMs by stabilizing lesions, but also by possibly preventing new lesions from emerging.

“We understand the high unmet need faced by patients diagnosed with cerebral cavernous malformations and the overlap with our focus in epilepsy, given the prevalence of seizure activity resulting from CCMs,” said Craig Chambliss, president and CEO of Neurelis. “Adding BA-1049 to our pipeline highlights our commitment to developing new therapies for patients and strengthens our position as an innovative neuroscience company.”

Author: Rare Daily Staff

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