AxoSim Licenses Revolutionary Mini-Brain Technology from Johns Hopkins University Enabling the Use of Human Data to Speed New Drug Research

NEW ORLEANS, May 30, 2019 /PRNewswire/ -- AxoSim, Inc., the premier provider of neuroscience discovery platforms that mimic the structure and function of the human nervous system, today announced an exclusive license from Johns Hopkins University to intellectual property underlying the "Mini-Brain" technology, which uses induced pluripotent stem cells (iPSCs) to create functional models of the human brain. Mini-Brain technology enables scientists to study key brain functions, test new therapies and screen for toxic substances in human rather than animal models at an early stage of research.

Almost 90% of drugs that look promising in animal models fail once they are tested in humans, driving up the average cost and time to develop a new drug to an estimated $2.6 billion and more than 10 years. The problem is especially acute for neurological disorders such as Alzheimer's disease, amyotrophic lateral sclerosis and multiple sclerosis, where animals are notoriously poor predictors of human outcomes and better therapies are urgently needed. The recent costly clinical trial failures of new Alzheimer's drugs highlight the need for better ways to test drug candidates much earlier in development.

The Mini-Brain technology was developed to replace inaccurate animal testing with accurate, predictive models of the human brain. It has the potential to reduce the time and cost of new drug R&D for disorders such as multiple sclerosis, glioblastoma, Alzheimer's disease, infectious diseases, and others, in addition to its applications in toxicology. Related Nerve-on-a-Chip^® technology developed at AxoSim has been shown to achieve research milestones at a fraction of the time and cost of conventional animal testing.

"Acquiring rights to the intellectual property underlying the Mini-Brain technology is a major step forward in our drive to transform neurology drug research," said Lowry Curley, PhD, Chief Executive Officer of AxoSim. "Mini-Brains are live organoids that mimic multiple brain functions, allowing for a wide variety of applications in neurotoxicity and neurological diseases with the potential to substantially speed-up and increase the effectiveness of new drug R&D. The Mini-Brain technology complements our innovative Nerve-on-a-Chip platform and accelerates our strategy of providing the broadest and most scientifically robust neuroscience models to our growing roster of biopharmaceutical partners."

In a related development, AxoSim announced that Dr. Thomas Hartung, who led the team that invented the Mini-Brain intellectual property, will serve as a Consulting Vice President of Scientific Affairs. Dr. Hartung is the Doerenkamp-Zbinden Chair for Evidence-based Toxicology and Director of the Center for Alternatives to Animal Testing at the Johns Hopkins Bloomberg School of Public Health. The company also reported that it has acquired all the assets of Organome, Inc., a company founded by Dr. Hartung to commercialize the Mini-Brain technology and other functional organ equivalents.

Dr. Curley added, "We are thrilled that Dr. Thomas Hartung has chosen to serve as a scientific consultant to AxoSim. Dr. Hartung's experience in applying advanced tissue engineering, stem cell and high content data technologies to empower early stage neuroscience and toxicology research will be very valuable as we continue to refine the Mini-Brain technology."

"AxoSim now provides a suite of models across the central nervous system and peripheral nervous system and has the only two commercially available platforms with 3D human-relevant myelination," said Benjamin Cappiello, Chief Business Officer of AxoSim. "Our world-class team strives to add significant value as an extension of preclinical discovery and toxicology at our biopharmaceutical partners, providing early human data that significantly accelerates research programs while substantially reducing costs."

AxoSim's Mini-Brain platform creates tiny brain-like organoids for neuroscience research using human induced pluripotent stem cells. These are adult cells (typically skin cells) that have been genetically reprogrammed to return to an embryonic stem cell-like state. The cells then are stimulated in the laboratory to grow into brain cells, which are engineered to reproducibly form tiny, uniform spheres barely visible to the human eye. After several months, the Mini-Brains develop different types of neurons and support cells, as well as the essential neuron insulator myelin. Importantly, the Mini-Brain components interact with each other and with their environment, and they can be replicated on a large-scale. The cells used to create iPSCs can be harvested from healthy individuals or from volunteers with certain genetic traits or diseases, allowing creation of Mini-Brains designed to study specific conditions, including Alzheimer's disease, Parkinson's disease, multiple sclerosis, and other neurodegenerative, functional, trauma-related and infectious disorders.

For more information on how AxoSim's Mini-Brain and Nerve-on-a-Chip platforms can accelerate your neuroscience research, contact Ben Cappiello at [email protected].

AxoSim at BIO 2019
Visit AxoSim at BIO 2019 at Booth #3458 and attend the BIO Session featuring AxoSim's CEO Dr. Lowry Curley and other leaders in the development and commercialization of biomimetic human R&D models.

• Next Generation Biotherapeutics, Humans-in-a-Dish: Hope or Hype?
• June 3, 2019, 4:00-5:00 pm, Room 105AB, Level 100

AxoSim at GES 2019
AxoSim CBO, Benjamin Cappiello, has been selected to participate in the Global Entrepreneurship Summit 2019, hosted by the U.S. Department of State and the Ministry of Foreign Affairs of the Netherlands, June 3-5, 2019 in the Hague, Netherlands, as part of a U.S. delegation of leading technology entrepreneurs who will engage with investors, potential partners and global thought leaders.

About AxoSim
AxoSim is the premier provider of biomimetic neuroscience discovery platforms. The company's proprietary Nerve-on-a-Chip^® and Mini-Brain technologies provide accurate, predictive models of the human nervous system, rapidly delivering clinically actionable human data early in the drug development process. AxoSim's platforms have the potential to reduce the time and cost of new drug R&D for disorders such as multiple sclerosis, glioblastoma, Alzheimer's disease, amyotrophic lateral sclerosis, infectious diseases, and others, in addition to its applications in toxicology. The Nerve-on-a-Chip technology has been shown to achieve research milestones at a fraction of the time and cost of conventional animal testing, allowing AxoSim's biopharma partners to develop more effective and safer drugs with greater speed and efficiency. For more information, visit AxoSim.com.

SOURCE AxoSim, Inc.

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