BioAxone BioSciences Announces License of Cerebral Cavernous Malformations Drug Candidate BA-1049 and Portfolio of Rho Kinase Inhibitors to Neurelis

Continued Development of BioAxone Pipeline of Spinal Cord Treatment Drug Candidates

BOSTON--()--BioAxone BioSciences, Inc., a biotechnology company committed to discovering and developing drugs for unmet needs of neurological conditions today announced the license of BA-1049, its innovative orally-available ROCK2 inhibitor that has promise as a therapeutic agent for the treatment of cerebral cavernous malformations (CCMs), to Neurelis, Inc. BioAxone CEO Dr. Lisa McKerracher noted, “Neurelis is the ideal partner to develop BA-1049 because they are a commercial-stage neuroscience company focused on the development and commercialization of therapeutics for the treatment of epilepsy and orphan neurological diseases of high unmet medical need.”

Cerebral cavernous malformations, also called cavernous angiomas, are benign vascular lesions that arise randomly in the brain and spinal cord that tend to be leaky and bleed. There are both spontaneous and genetic forms of the disease, and in the genetic form patients develop multiple lesions over their lifetime. CCM is an unmet need with no pharmacological drugs to arrest lesion genesis or prevent bleeding, and there is no current treatment available other than surgical removal of the brain lesions. CCMs may cause stroke, seizures, paralysis, or other neurological symptoms dependent on their location in the brain. The standard of care is watchful waiting until neurosurgical removal of a leaky lesion is deemed un-avoidable. The progression of individual lesions is variable and unpredictable, leaving patients unable to prepare for or prevent re-occurrence of symptoms following diagnosis.

Dr. Guy Rouleau, MD, PhD., Recipient of the Order of Canada, Director of Montreal Neurological Institute McGill University, and a member of the BioAxone Scientific Advisory Board said “I am proud to have contributed to the development of BA-1049 since the early days of the BioAxone program. I have followed Dr. McKerracher’s work on Rho/ROCK signaling since the 1990s when both of us were new faculty in the Department of Neurology and Neurosurgery at McGill University. Many patients are eagerly awaiting the development of a disease modifying drug.”

Dr. Connie Lee, Founding President and CEO of the Angioma Alliance, the leading patient advocacy group stated “We are so pleased to learn that BioAxone Biosciences has partnered with Neurelis, a private commercial stage biotech company, who will advance BA-1049 development forward to provide hope for patients with CCM. BA-1049 has shown great promise in preclinical experiments completed in collaboration with our research community. It has been our dream to see BA-1049 and other pre-clinical stage assets advance to clinical trials, an important milestone in the path for potential regulatory approvals and the marketplace for patients who are waiting for life altering therapies.” Dr. Lee is the mother of a daughter with multiple CCM lesions.

BioAxone will continue to develop its pipeline including a clinical stage drug (BA-210) and a preclinical program (BA-434) targeting axon regeneration for treatment of spinal cord injury.

Research for development of BA-1049 was supported by NIH Small Business Innovation Research grants. “ We are grateful to the NIH SBIR program,” said Dr. McKerracher. “The financial support and critical comments contributed greatly to the success of our BA-1049 program.”

About BioAxone BioSciences, Inc.

BioAxone BioSciences is a biotechnology company based in Boston, MA, committed to applying its deep understanding of axon regeneration and neuronal signaling pathways to transform the lives of patients afflicted with neurotrauma or neurovascular disorders. Leveraging 20 years of pioneering research in axon regeneration and diseases involving Rho/ROCK signaling, BioAxone’s lead drug candidate is an orally-available ROCK2 inhibitor restoring endothelial cell barriers in cerebral cavernous malformations (CCMs), also known as cavernous angioma, and stroke. The Company’s pipeline also includes programs to develop drug candidates for the treatment of spinal cord injury. For more information, visit

About Neurelis, Inc.

Neurelis is a neuroscience-based company with a unique portfolio of product candidates and innovative platform technologies. Along with strategic partners, they are dedicated to enhancing therapeutic benefits and addressing significant unmet needs in patient care. Neurelis was founded to license, develop, and commercialize product candidates for the broader central nervous system, including epilepsy and psychiatry. The emphasis is on highly differentiated therapies that empower patients, care partners, and healthcare providers. The benefits of their unique platform technology allow them to form strategic partnerships to address significant unmet needs in other therapeutic areas as well. For more information on Neurelis, please visit

About the Angioma Alliance

The Angioma Alliance has a mission to inform, support and mobilize people affected by cavernous angioma and drive research for treatments and a cure. They facilitate and participate in cavernous angioma research through collaboration with researchers, research institutions and the biotechnology industry. Founded in 2002, Angioma Alliance represents patients in the United States and works closely with its 21 sister organizations around the world to improve the quality of care for cavernous angioma patients and their families. For more information on the Angioma Alliance, please visit


Beth Kurth
Conway Communications

Release Summary

BioAxone BioSciences today announced the license of BA-1049, its innovative orally-available ROCK2 inhibitor targeting CCMs, to Neurelis, Inc.


Beth Kurth
Conway Communications