Aspen Neuroscience Announces First Patient Dosed in First-in-Human Phase 1/2a Clinical Trial of Autologous Neuronal Cell Replacement Therapy for Parkinson's Disease

First U.S. Multi-center, Multi-patient Phase 1/2a Trial of an Autologous Neurological Therapy

SAN DIEGO, April 17, 2024 /PRNewswire/ -- Aspen Neuroscience, Inc. announced that the first patient has been dosed in the ASPIRO trial, a Phase 1/2a open label clinical trial to assess safety and tolerability of ANPD001, an autologous, dopaminergic neuron cell replacement therapy for participants with moderate to severe Parkinson's disease (PD). Aspen Neuroscience is a private biotechnology company developing personalized regenerative therapies.

The first transplantation procedure in the ASPIRO study was performed by Banner – University Medical Center Tucson neurosurgeon Paul Larson, MD, FAANS, professor of neurosurgery at the University of Arizona College of Medicine – Tucson. The hospital is one of five clinical sites for the Phase 1/2a trial.  Dr. Larson is the lead neurosurgeon and Banner – University Medicine Tucson is the lead dosing site for the ASPIRO study.

Dr. Larson is a pioneer in the field of interventional MRI-guided stereotaxy, and has performed over 1,000 iMRI procedures. His clinical research team has been the solo or lead group in 12 cell and gene therapy clinical research trials since 2004, and is one of the top teams in intracranial delivery of novel therapeutics for neurodegenerative disorders. 

"Parkinson's disease is the most common neurodegenerative movement disorder, primarily affecting the depletion of dopamine neurons in the midbrain.  By the time of diagnosis, it is common for people with Parkinson's to have lost the majority of dopaminergic (DA) neurons in the nigrostriatal pathway, which leads to progressive loss of motor and neurological function," explained Dr. Larson. "This is the first use of the autologous approach in a formal clinical trial, and it is an honor to be part of this important study."

"The initiation of this clinical trial is a major milestone in Aspen's mission to develop and deliver personalized, regenerative neurologic therapies for people with unmet medical needs, starting with Parkinson's disease," said Damien McDevitt, PhD, Aspen Neuroscience President and Chief Executive Officer. "To date, there is no disease-modifying therapy that can stop, replace or prevent the loss of dopamine neurons or slow the progression of Parkinson's."

"This first-in-human trial holds significant promise to investigate the ability of ANPD001 to improve the lives of people with moderate to advanced Parkinson's disease," said Edward Wirth III, MD, PhD, Chief Medical Officer of Aspen Neuroscience.  "Our 2022 Trial Ready Cohort Screening Study has completed enrollment, and we plan to dose patients in the ASPIRO Phase 1/2a study this year."

About the ASPIRO Trial

ASPIRO is an open-label Phase 1/2a clinical trial to assess the safety and tolerability of ANPD001 in patients with moderate to severe Parkinson's disease. The trial includes patients 50–70 years of age, and excludes patients with cognitive impairment and other comorbidities that could preclude treatment. All enrolled patients are under the care of a movement disorder specialist.

The primary study endpoint is safety and tolerability of two sequential escalating doses of ANPD001.  Secondary endpoints include improvement in "on" time, when patients experience periods of good symptom control, and improvements in motor symptoms and quality of life based on standard Parkinson's disease rating scales.

About ANPD001

ANPD001 is an investigational autologous neuronal replacement therapy being studied as a regenerative therapy for PD. Aspen's personalized approach means that patients do not require  immunosuppressive drugs to counteract the body's immune response against foreign tissue.

Aspen's manufacturing process starts from a small sample of the patient's own skin cells, followed by reprogramming to induced Pluripotent Stem Cells (iPSCs) and then differentiation of the iPSCs into dopaminergic neuron precursors (DANPCs). These DANPCs are transplanted into the putamen, replacing cells that were lost or damaged due to disease. The quality of each person's cells is assessed at every manufacturing stage using Aspen's proprietary machine learning-based genomics tests.  

About the University of Arizona College of Medicine – Tucson

The University of Arizona College of Medicine – Tucson is shaping the future of medicine through state-of-the-art medical education programs, groundbreaking research and advancements in patient care in Arizona and beyond. Founded in 1967, the college boasts more than 55 years of innovation, ranking among the top medical schools in the nation for research and primary care. Through the university's partnership with Banner Health, one of the largest nonprofit health care systems in the country, the college is leading the way in academic medicine. For more information: medicine.arizona.edu (or follow on: Facebook | Twitter | LinkedIn | Instagram).

About Banner – University Medical Center Tucson and South

Banner – University Medical Center Tucson and Banner – University Medical Center South are part of Banner – University Medicine, a premier academic medical network. These institutions are academic medical centers for the University of Arizona College of Medicine – Tucson. Included on the two campuses are Diamond Children's Medical Center and many specialty clinics. The two academic medical centers are part of Arizona-based Banner Health, one of the largest nonprofit health care systems in the country. Banner Health is in six states: Arizona, California, Colorado, Nebraska, Nevada and Wyoming. For more information, visit www.bannerhealth.com/universitytucson  or www.bannerhealth.com/universitysouth.

About Aspen Neuroscience

Headquartered in San Diego, Aspen Neuroscience, Inc. is a clinical development-stage, private company focused on autologous regenerative medicine. The company's patient-derived iPSC platform is used to create personalized therapies to address diseases with high unmet medical needs, beginning 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 has developed a best-in-class platform to create and optimize pluripotent-derived cell therapies, which includes in-house bioinformatics, manufacturing and quality control. For more information and important updates, please visit https://www.aspenneuroscience.com.

SOURCE Aspen Neuroscience, Inc.