Aspen Neuroscience Announces FDA Clearance of Investigational New Drug Application for ANPD001, Autologous Cell Therapy for the Treatment of Parkinson's Disease

Aspen Will Initiate First Multicenter Phase 1/2a Clinical Trial of an Autologous iPSC Based Therapy in the U.S.

SAN DIEGO, Aug. 8, 2023 /PRNewswire/ -- Aspen Neuroscience today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application, enabling the company to proceed with a clinical trial for ANPD001, a personalized (autologous) cell therapy to treat Parkinson's Disease by replacing lost dopamine neurons.

Next Aspen plans to initiate a first-in-patient Phase 1/2a clinical trial for people with moderate to severe Parkinson's Disease (PD). This follows the company's 2022 Trial-Ready Screening Cohort Study to screen, enroll and begin manufacturing cells for potential patient candidates for the clinical trial. This study will be the first multicenter Phase 1/2a trial of an autologous iPSC-derived therapy in the U.S.

Aspen's autologous approach uses induced pluripotent stem cells (iPSCs) developed from the patient's own skin cells, to manufacture dopamine neuronal precursor cells (DANPCs). These cells are then evaluated for potential activity using robust quality control assays, including Aspen's proprietary artificial intelligence-based genomics tests, before being implanted.

"This is a major milestone in Aspen's mission to develop and deliver personalized iPSC-derived cell replacement therapies for people with unmet medical needs, starting with Parkinson's disease," said Faheem Hasnain, chairman of the Aspen board of directors. "This is an exciting time for the Aspen team and the patients who have been so instrumental in enabling the company's development."

"The IND clearance of ANPD001 sets in motion a path toward a new treatment for the more than one million Americans and 10 million people worldwide with Parkinson's disease," said Damien McDevitt, PhD, Aspen president and CEO. "Our visionary team is working to make personalized regenerative medicine a reality, and we look forward to advancing this cell therapy for patients who are waiting."

The autologous cell therapy approach was pioneered by Aspen's co-founders, Jeanne Loring, PhD, Professor Emeritus and Director of The Center for Regenerative Medicine, Department of Molecular Medicine at the Scripps Research Institute, and Andres Bratt-Leal, PhD, Aspen senior vice president, research and development.

About ANPD001

ANPD001 is an investigational cell therapy product being studied as an autologous neuron replacement for Parkinson's disease. Aspen's personalized 3-step manufacturing approach starts from a small sample of the patient's own skin cells, followed by reprogramming to iPSCs and then differentiation into DANPCs. The DANPCs are then provided to the patient via surgery to replace their 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 artificial intelligence-based genomics tests.  

About the Phase 1/2a Clinical Trial
The ANPD001 clinical trial is a Phase 1/2a, single-arm, open-label study. The planned dose escalation clinical trial will evaluate the safety, tolerability and preliminary efficacy of ANPD001 in patients with moderate to severe PD.   

About Parkinson's Disease
Parkinson's disease is the world's second most common neurodegenerative disease. Patients can experience non-motor symptoms such as mental and behavioral changes, sleep problems, depression, memory difficulties and fatigue; and motor symptoms caused by the loss of dopamine neurons in the brain, including unintended or uncontrollable movements (dyskinesia), shaking (tremor), stiffness, and difficulty with balance and coordination, which worsen over time.

By the time of diagnosis, it is common for people to have lost the majority of cells that make dopamine in the brain. To date, there is no disease-modifying therapy that can stop, replace or prevent the loss of dopamine neurons or slow the progression of PD.

About Aspen Neuroscience
Headquartered in San Diego, Aspen Neuroscience, Inc. is a clinical development-stage, private company focused on personalized (autologous) regenerative medicine. The company is developing patient-derived iPSCs to create personalized cell therapies that address diseases with high unmet medical needs, beginning with autologous neuron replacement for Parkinson's disease and extending across the brain and affected organs.

A leading iPSC platform company, Aspen combines stem cell biology with the latest artificial intelligence and genomic approaches to investigate patient-specific, restorative treatments. The company has developed a best-in-class platform to create and optimize iPSC-derived cell therapies, which includes in-house bioinformatics, manufacturing and QC. For more information and important updates, please visit https://www.aspenneuroscience.com.    

SOURCE Aspen Neuroscience, Inc.