FDA Grants Fast Track Designation to ANPD001, Autologous Investigational Cell Therapy for the Treatment of Parkinson’s Disease

SAN DIEGO, Oct. 19, 2023 /PRNewswire/ — Aspen Neuroscience today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for ANPD001 for the treatment of Parkinson’s disease (PD) to improve motor function. ANPD001, is a personalized (autologous) cell therapy under investigation to treat PD by replacing lost dopamine neurons.

The purpose of Fast Track Designation is to get important new drugs to patients earlier. FDA grants Fast Track Designation (FTD) to facilitate development, and to expedite the review of medicines to treat serious conditions and fill an unmet medical need. Benefits of FTD include early and frequent interactions with FDA during the clinical development process, as well as eligibility for accelerated approval and priority review.

“We are pleased that FDA has granted Fast Track Designation, underscoring the potential of an autologous dopamine replacement therapy such as ANDP001 to serve as a meaningful treatment option. People with Parkinson’s disease have a significant unmet medical need and have been waiting for many years for more advanced treatment options,” said Damien McDevitt, PhD, Aspen president and CEO.

“Achieving Fast Track Designation is an important milestone that furthers our ability to collaborate with FDA and facilitate the development of ANPD001,” said Ana Sousa, MSJ, Aspen Senior Vice President, Regulatory Affairs & Quality. “Our goal is to bring this treatment to patients as safely and expeditiously as possible.” 

Aspen plans to initiate a first-in-patient Phase 1/2a clinical trial of ANPD001 for participants with moderate to severe 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 personalized, 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. 

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 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 . 

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 pluripotent-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.

Aspen Neuroscience does not make determination on patient eligibility for clinical trials and all trial related enrollment and eligibility decisions are made by clinical trial sites under the direction of a Principal Investigator. ANPD001 is investigational and is not yet approved by the FDA. At this time, Aspen Neuroscience does not offer or participate in compassionate use or expanded access programs for ANPD001.