Aspen Neuroscience Reports Positive 6-Month Data for ANPD001 in Parkinson’s Disease Ahead of IAPRD 2025 Presentation

08 May 2025 | Thursday | News

First-of-its-kind autologous cell therapy shows early clinical and patient-reported improvements with favorable safety in Phase 1/2a trial for moderate to advanced Parkinson’s disease
Autologous iPSC-derived neurons (PRNewsfoto/Aspen Neuroscience, Inc.)

Autologous iPSC-derived neurons (PRNewsfoto/Aspen Neuroscience, Inc.)

  • ANPD001 was well tolerated with no major safety issues in low-dose cohort
  • Patients showed early improvements in patient-reported and clinician-reported outcomes
  • Detailed Phase 1/2a trial data will be presented this week at IAPRD 2025

Aspen Neuroscience has released 6-month data from the first three patients dosed in the ASPIRO Phase 1/2a trial of ANPD001, an investigational autologous dopaminergic neuronal precursor cell (DANPC) therapy being studied in patients with moderate to advanced Parkinson's Disease (PD).

After recent publication in Parkinsonism and Related Disorders, detailed 6-month data on the first three patients will be presented this week at the 30th World Congress on Parkinson's Disease and Related Disorders (IAPRD 2025), taking place in New York from May 7-10.  Aspen Neuroscience Medical Director Avram Fraint, M.D., M.S. will present "Safety, tolerability, and efficacy of intracranial delivery of autologous iPSC-derived dopaminergic precursors in moderate to advanced Parkinson's disease" on Saturday, May 10 during the IAPRD Guided Poster session.

"Parkinson's disease is the second most common neurodegenerative disorder. By the time of diagnosis, most people with Parkinson's have lost the majority of their dopaminergic neurons, leading to progressive loss of motor and non-motor function," said Dr. Fraint. "To date, data from the first three patients in the ASPIRO study show that precision delivery of personalized DANPCs is safe and well-tolerated."

"In this first-of-its-kind study, we are seeing clinician-reported and patient-reported improvements as well as a strong safety and tolerability profile at six months. Importantly, ANPD001 has the unique advantage of not requiring immunosuppression," said Edward Wirth III, MD, PhD, Chief Medical Officer of Aspen Neuroscience.

"We are very encouraged by the ASPIRO study results to date. With more than one million people currently living with Parkinson's disease in the U.S. alone, these data are promising for the Parkinson's community, and for the autologous cell therapy field," said Damien McDevitt, PhD, president and CEO of Aspen Neuroscience.

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