BioAxone BioSciences Receives Notice of Allowance on Patent for Treatment of Cerebral Cavernous Malformations and Cerebral Aneurysms with Rho Kinase Inhibitors

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CAMBRIDGE, Mass.–(BUSINESS WIRE)–BioAxone BioSciences, Inc., a biotechnology company focused on developing innovative drugs to treat neurotrauma and neurovascular disorders, today announced that it has received a Notice of Allowance from the United States Patent and Trademark Office (USPTO). The notice refers to a key patent application (US 15/590,815), which will add to the intellectual property protection for BA-1049, BioAxone’s first in-class, selective Rho kinase 2 (ROCK2) inhibitor, for use in combating angioma (also called cerebral cavernous malformations (CCM)). When issued, this patent will cover using BA-1049 specifically to treat angioma and also the ability to combine this proprietary compound with other known inhibitors. BA-1049 acts to reverse the loss of endothelial barrier dysfunction that occurs in vascular disorders of the central nervous system.

BA-1049 is part of the neurovascular-focused pipeline of Rho kinase 2 inhibitors in development by BioAxone. Rho kinase 2 (ROCK2) is a drug target that plays a key role in propagating biological signals that regulate brain endothelial cell barrier function and this enzyme is frequently overactivated in neurovascular pathologies where barrier function is compromised. In animal models of stroke, reversing the overactivation of ROCK2 that occurs in the vasculature restores endothelial cell barrier function.

“This is important news for BioAxone and further strengthens the IP portfolio for our catalogue of ROCK2 inhibitors to treat neurovascular disorders,” said Lisa McKerracher, PhD, CEO, BioAxone BioSciences. “BA-1049 is a promising oral drug candidate for treatment of angioma to reverse endothelial cell defects that cause symptoms. It is the only new chemical entity in development to target the growth and leakiness of cavernous angioma lesions and could be a transformative treatment for this unmet need.”

About BA-1049
BA-1049 is a first-in-class oral inhibitor of Rho kinase 2 (ROCK2), a kinase that regulates endothelial cell junctions and the blood brain barrier. It is in development to treat angioma (cerebral cavernous malformation), a cerebrovascular disease where thin-walled, leaky capillaries form brain lesions susceptible to bleeding that predispose patients to a lifetime risk of seizure, hemorrhagic stroke and other neurological defects. In preclinical experiments, BA-1049 has shown promise to repair the leaky endothelial cell barrier and potentially to reverse progression of the disease.

About BioAxone BioSciences
BioAxone BioSciences is a clinical-stage biotechnology company based in Cambridge, MA, and is developing innovative drugs to restore neurological function for patients with spinal cord injuries and neurovascular disorders. The company also has preclinical programs in glaucoma and aimed at restoring epithelial barriers in gastrointestinal disorders. BioAxone is led by a team of scientists renowned for their work on axon regeneration, diseases involving Rho/ROCK signaling and cell barrier function. For more information, visit and follow the company on Twitter at @BioAxone.


Paul Sagan, 617-374-8800 Ext. 106

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