Aspen Neuroscience Announces Positive 12‑Month Data from its ASPIRO Clinical Trial in a Late‑Breaking Oral Presentation at the AD/PD™ 2026 International Conference on Alzheimer's and Parkinson's Diseases

Results Show Continued Safety, Tolerability, and Sustained Clinical Benefit Including Improvements in Function, Physician- and Patient-Reported Outcomes, and Quality-of-Life

This Personalized Autologous Cell Therapy Aims to Replace and Restore the Damaged Neural Circuitry in Parkinson's Patients that Underpins Everyday Movement and Function, Without the Need for Immune Suppression

SAN DIEGO, March 18, 2026 /PRNewswire/ -- Aspen Neuroscience, a clinical-stage regenerative medicine company developing autologous induced-pluripotent stem cell (iPSC)-derived therapies to initially address neurodegenerative diseases with high unmet medical need, today announced data from its ongoing Phase 1/2a ASPIRO* clinical trial in a late‑breaking oral presentation at the AD/PD^™ 2026 International Conference on Alzheimer's and Parkinson's Diseases, taking place March 17–21, 2026 in Copenhagen, Denmark.

The presentation highlights 12‑month safety, tolerability, and efficacy data following delivery of Sasineprocel (ANPD001), an autologous induced pluripotent stem cell (iPSC)‑derived dopaminergic neuron precursor cell therapy (DANPC), in patients with Parkinson's disease without requiring immune suppression or permanent implantable hardware.

"These data support Aspen's differentiated autologous approach and underscore the potential for sustained clinical benefit without the need for chronic immunosuppression," said ASPIRO trial investigator Chad Christine, M.D., Professor of Neurology, University of California at San Francisco. "This regenerative cell therapy approach aims to not only restore dopamine levels in the brain, but also to replace and rebuild the neural circuitry essential for everyday movement and function, with the goal of slowing or halting disease progression."

The presentation represents data from the first eight treated patients – four of whom received a low dose and four of whom received a higher dose of the therapy. At twelve months, both dosing cohorts showed numerical improvements in function, physician- and patient-reported outcomes, and quality-of-life, including: mean Good ON time (+2.1 hours in low dose (LD), +2.4 hours in high dose (HD)), mean MDS-UPDRS III OFF scores (-15.5 points in LD, -13.5 points in HD), mean MDS-UPDRS II scores (-5.3 points in LD, -2.3 points in HD), and mean improvement in PDQ-39 scores (51.6% in LD, 28.5% in HD).

Several patients had these improvements while also demonstrating LEDD (Levodopa equivalent daily dose) reductions. Additionally, FDOPA PET imaging shows cell survival and successful engraftment of Sasineprocel.

No serious surgical adverse events and no severe graft-induced dyskinesia were observed. No symptomatic hemorrhages or infarctions were observed. Real-time intraoperative imaging confirmed accurate placement of DANPCs into the post-commissural putamen.

"We're very encouraged by these data showing improvements in function, physician- and patient-reported outcomes, and quality-of-life, and look forward to advancing Sasineprocel to a Phase 3 study later this year," said Revati Shreeniwas, M.D., Chief Medical Officer, Aspen. "These findings support our approach of reprogramming a patient's own cells to wind back the epigenetic clock to a pre‑disease state. Combined with precisely delivering our multi-cell composition to the putamen, we're starting to see the positive clinical impact of rebuilding the neural networks damaged by this debilitating disease."

Late‑Breaking ASPIRO Data at AD/PD 2026

Abstract Title: "Twelve‑Month Safety, Tolerability, and Efficacy of Intracranial Delivery of Autologous iPSC‑Derived Dopaminergic Precursors (Sasineprocel) in Parkinson's Disease"

Date/Time: March 18, 2026, 3:45-4:00 p.m. CET

Session Name: MODULATING NEUROINFLAMMATION, Α-SYNUCLEIN, LRRK2, AND DOPAMINERGIC REPAIR: EARLY HUMAN DATA

About the ASPIRO Trial

ASPIRO is a first-in-human, multi-patient, multi-center clinical trial of an autologous cell therapy for Parkinson's disease. This Phase 1/2a open-label trial is evaluating safety, tolerability and preliminary efficacy of Sasineprocel (ANPD001) in PD patients aged 50–70.

The study's primary aim is to assess safety and tolerability of bilateral intracranial administration of autologous iPSC DANPCs into the post-commissural putamen.

The study's secondary aim is to evaluate change in clinician- and patient-reported PD-specific clinical outcomes.

The product was derived from skin fibroblasts which were converted into iPSCs and then differentiated into DANPCs. Four patients received 5 million cells per hemisphere, and four patients received 5 million to 10 million cells per hemisphere, all under MRI guidance via occipital surgical approach. Clinical outcomes were assessed every six months after transplantation.

More information about the Phase 1/2a trial is available at clinicaltrials.gov (NCT06344026).

About Sasineprocel (ANPD001) 

Sasineprocel (ANPD001) is the most advanced autologous investigational cell therapy in the United States for treating Parkinson's disease. Aspen's personalized approach means that patients do not require immunosuppressive (IS) drugs to dampen the body's immune response against the foreign cells used in allogeneic cell therapy. This approach will eliminate IS-associated adverse events, IS drug-monitoring requirements and enable dosing for those with contraindications to IS therapies. In addition, this approach will not require implanted permanent hardware.

Aspen's manufacturing process starts from a small biopsy of the patient's own skin cells, followed by reprogramming to iPSCs, which rejuvenates the cells by turning back their biological clock to a pre-disease state. The iPSCs are then differentiated into DANPCs, which are transplanted into the posterior putamen, replacing 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 machine-learning-based genomics tests. 

With this novel therapeutic approach, it is Aspen's vision to address the core challenge of Parkinson's disease: to not only restore dopamine levels in the brain but to also replace and restore the damaged neural circuitry that supports everyday movement and function—without the requirement of immune suppression. Our investigational autologous cell therapy is designed to rebuild these neural networks with the goal to potentially slow or halt disease progression. 

Sasineprocel has received Fast Track designation by the U.S. Food and Drug Administration (FDA). 

For more information, visit www.aspenneuroscience.com. 

* The study was made possible by funding from the California Institute for Regenerative Medicine (CIRM), a state of California Agency that funds regenerative medicine, stem cell, and gene therapy research (Grant Number CLIN2-15547).

SOURCE Aspen Neuroscience, Inc.