Aspen plans to initiate a first-in-patient Phase 1/2a clinical trial of ANPD001 for participants with moderate to severe PD
ANPD001 is an investigational cell therapy product being studied as an autologous neuron replacement for Parkinson’s disease. (Credit: Annick Vanblaere from Pixabay)
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.
Source: Company Press Release