The clinical trial comes after the company's Trial-Ready Screening Cohort Study conducted in 2022, which involved screening, enrolling, and beginning the process of manufacturing cells for potential candidates for the clinical trial
US FDA approves IND application for Aspen’s Parkinson's disease treatment. (Credit: PublicDomainPictures from Pixabay)
Aspen Neuroscience has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application, which allows the company to move forward with a clinical trial involving ANPD001, a personalised cell therapy designed to treat Parkinson’s Disease by replacing dopamine neurons that have been lost.
The next step for Aspen is to initiate a Phase 1/2a clinical trial for individuals with moderate to severe Parkinson’s Disease.
This clinical trial comes after the company’s Trial-Ready Screening Cohort Study conducted in 2022, which involved screening, enrolling, and beginning the process of manufacturing cells for potential candidates for the clinical trial.
Notably, this study will mark the first multicentre Phase 1/2a trial of an autologous induced pluripotent stem cell (iPSC)-derived therapy in the US.
Aspen Neuroscience’s approach to treating Parkinson’s Disease is personalised and autologous, utilising iPSCs derived from a patient’s own skin cells to generate dopamine neuronal precursor cells (DANPCs). These DANPCs undergo thorough quality control assessments, including the application of Aspen’s proprietary artificial intelligence-based genomics tests, before they are implanted into the patient.
This approach holds promise for advancing the treatment of Parkinson’s Disease and may provide a new avenue for managing this complex condition.
Aspen chairman of the board of directors Faheem Hasnain said: “This is a major milestone in Aspen’s mission to develop and deliver personalised iPSC-derived cell replacement therapies for people with unmet medical needs, starting with Parkinson’s disease. This is an exciting time for the Aspen team and the patients who have been so instrumental in enabling the company’s development.”
Aspen president and CEO Damien McDevitt said: “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.
“Our visionary team is working to make personalised regenerative medicine a reality, and we look forward to advancing this cell therapy for patients who are waiting.”
The autologous cell therapy approach employed by Aspen Neuroscience was pioneered by its co-founders, Dr Jeanne Loring and Dr Andres Bratt-Leal. Dr Jeanne Loring, a Professor Emeritus and Director of The Center for Regenerative Medicine at the Department of Molecular Medicine at the Scripps Research Institute, played a significant role in developing this approach. Dr Andres Bratt-Leal, Aspen’s Senior Vice President of Research and Development, also contributed to the development of this innovative approach to treating Parkinson’s disease.
About ANPD001
ANPD001 is an investigational cell therapy product developed by Aspen Neuroscience for the treatment of Parkinson’s disease. The therapy involves a personalized three-step manufacturing process that aims to replace the lost or damaged dopamine neurons in patients with Parkinson’s disease.
About the Phase 1/2a Clinical Trial
The Phase 1/2a clinical trial for ANPD001 is designed as a single-arm, open-label study with the goal of evaluating the safety, tolerability, and preliminary efficacy of the investigational therapy in patients with moderate to severe Parkinson’s disease.