Primate Study Demonstrates Safety and Feasibility of Cell Transplantation Approach
Currently in Phase 1/2a First-in-Human Parkinson's Disease Clinical Trial

SAN DIEGO, July 29, 2024 /PRNewswire/ -- The Journal of Neurosurgery has published online a study by the Wisconsin National Primate Research Center at the University of Wisconsin, Madison regarding a novel cell transplantation approach being used for delivery of ANPD001, an autologous, dopaminergic neuronal cell replacement under investigation by Aspen Neuroscience as a potential treatment for Parkinson's Disease.

The study by the Wisconsin National Primate Research Center demonstrated the safety and feasibility of the treatment approach for ANPD001 in non-human primates. Aspen is currently investigating ANPD001 in the ASPIRO trial, a first-in-human, open-label Phase 1/2a clinical trial in people with moderate to severe Parkinson's disease.

The study was also incorporated into Aspen's Investigational New Drug (IND) application with the U.S. Food & Drug Administration (FDA), enabling the company to proceed with the clinical trial of ANPD001.

University of Wisconsin researcher Marina Emborg, M.D, PhD, professor, Department of Medical Physics, led the study. A previous study by Dr. Emborg, and her colleagues, published in 2021, demonstrated that an autologous neuronal replacement therapy reversed Parkinsons-like symptoms in primates.

"The results of the study established that this approach can be used to safely target neuronal replacement therapy with precision to the brain and supports its clinical investigation as a procedure for cell replacement therapy in neurodegenerative diseases," concluded Dr. Emborg.

"The study was an important step in our work to bring the promise of a cell-replacement therapy to people with Parkinson's disease," said Andrés Bratt-Leal, PhD, study co-author, Aspen Neuroscience co-founder and senior vice president of research and development. "The results were instrumental in opening our first-in-human trial and informing how we deliver patients' own cells."

ANPD001 begins with a small sample of the patient's own skin cells, followed by reprogramming to induced pluripotent stem cells (iPSCs) which can become any type of cell. The iPSCs are then differentiated into dopaminergic neuron precursor cells (DANPCs), which are transplanted under real-time MRI guidance into the putamen region of the brain, replacing cells that were lost or damaged due to disease.

"By the time of diagnosis, it is common for people with Parkinson's to have lost the majority of dopaminergic neurons, leading to progressive loss of motor and neurological function," explained Edward Wirth III, MD, PhD, study co-author and Aspen's chief medical officer. "To replace these lost cells, we must target a very specific area of the brain with a high degree of surgical precision. Utilizing the latest advances in intraoperative MRI guided techniques, the patient's new cells are transplanted, a few microliters at a time, to the exact area where they are most needed."

Affecting more than one million Americans, PD is a neurodegenerative disorder that causes walking and motor problems, as well as impaired balance and coordination. Existing therapies alleviate symptoms but do not treat the underlying disease process, leading to a significant unmet medical need for those suffering from this chronic condition.

About the ASPIRO Trial

The Autologous-derived Study of a Parkinson's Investigational Regenerative therapy in an Open-label trial (ASPIRO) is a Phase 1/2a clinical trial to assess the safety, tolerability, and potential efficacy of ANPD001 in patients with moderate to severe Parkinson's disease. The primary study endpoint is safety and tolerability of ANPD001. Secondary endpoints include improvement in "on" time, when patients experience periods of symptom control, and improvements in motor symptoms and quality of life based on standard Parkinson's disease rating scales.

About Aspen Neuroscience

Aspen Neuroscience, Inc., headquartered in San Diego, is dedicated to autologous regenerative medicine. The company's patient-derived iPSC platform is used to create personalized therapies, a crucial step in addressing diseases with high unmet medical needs, starting with autologous neuron replacement for Parkinson's disease.

Aspen combines cell biology with the latest machine learning and genomic approaches to investigate patient-specific, restorative cell treatments. The company's unwavering commitment to quality is evident in its best-in-class platform to create and optimize pluripotent-derived cell therapies, which includes in-house bioinformatics, manufacturing, and quality control.

SOURCE Aspen Neuroscience, Inc.