On 18 March, at the AD/PD 2026 International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders, Aspen Neuroscience announced new 12-month data from the ongoing Phase I/IIa ASPIRO trial (NCT06344026) of its autologous induced pluripotent stem cell (iPSC) derived dopaminergic neuron precursor cell (DANPC) therapy, sasineprocel.
The results showed the DANPC therapy was associated with continued safety and sustained clinical benefit in patients with Parkinson’s disease, as measured by safety and tolerability, change in good ON time, changes from baseline in the Movement Disorder Society – Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Part II and Part III OFF scores, and quality of life improvements as assessed by The Parkinson’s Disease Questionnaire (PDQ-39). In the first eight treated patients, investigators reported numerical improvements across motor function, activities of daily living, patient-reported outcomes, and quality of life, while also showing imaging evidence of graft survival and engraftment.
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The safety and tolerability data displayed that sasineprocel was well-tolerated by all subjects. No serious surgical adverse events (AEs) associated with the bilateral intracerebral administration were reported, and neither were graft-induced dyskinesia nor symptomatic haemorrhage or infarction. Some mild AEs reported included incision site pain and headache, but no AEs were related to the study drug itself. Of the clinical outcomes, patients in the lower dose cohort (n=4) experienced an increase of 2.1 hours in mean good ON time, whereas the higher dose group (n=4) experienced an increase of 2.4 hours.
Overall, mean MDS-UPDRS Part III OFF scores improved by 15.5 points in the lower dose group and 13.5 points in the higher dose group, respectively, while mean MDS-UPDRS Part II scores improved by 5.3 points in the low-dose group and 2.3 points in the high-dose group. Mean PDQ-39 scores improved by 51.6% in the low-dose cohort and 28.5% in the high-dose cohort, with several patients also reducing levodopa equivalent daily dose during follow-up. Investigators also reported that FDOPA PET imaging showed cell survival and successful engraftment, adding biological support to the observed clinical changes. Aspen also highlighted the accuracy of DANPC placement in the post-commissural putamen through real-time intraoperative imaging.
This clinical data indicates that sasineprocel may have a future as a monotherapy competing with dopaminergic or anti-Parkinson’s disease therapies. However, as a monotherapy, sasineprocel would face competition from well-established therapies for the treatment of core Parkinson’s disease symptoms, such as levodopa, dopamine agonists (DA), and monoamine oxidase B inhibitors (MAO-B).
According to GlobalData’s Drugs Database, six dopamine agonists are currently marketed across the seven major pharmaceutical markets (the US, France, Germany, Italy, Spain, the UK, and Japan), many of which are available as generics. Aspen’s sasineprocel could also face competition from other cell therapies being developed for Parkinson’s disease such as Bluerock Therapeutics’ bemdaneprocel, which is in Phase III development.
As an iPSC-derived cell-therapy, Aspen’s sasineprocel is likely to be far more expensive than traditional Parkinson’s disease therapies, meaning it could face challenges in receiving regulatory approval, being reimbursed, and ultimately gaining traction in the market. However, as an iPSC-derived cell-therapy, it has a novel, potentially first-in-class, non-dopaminergic mechanism, which differentiates it from competitors.
The question for clinicians would be how to integrate such a unique treatment in routine Parkinson’s disease care. Aspen will need to demonstrate that sasineprocel can show superior efficacy and safety over marketed dopaminergic therapies in large Phase III clinical trials or engage in head-to-head studies for direct comparison. Aspen has plans to start a large Phase III trial later in 2026, and this will be critical in discerning whether these results can be replicated in a larger, controlled population.
In summary, Aspen’s ASPIRO trial results suggest that sasineprocel demonstrates a strong safety profile and promising clinical benefits in patients with Parkinson’s disease, supported by both functional and imaging evidence. While these findings highlight its potential as a novel, first‑in‑class iPSC‑derived therapy, its ultimate success will depend on proving superior efficacy and cost‑effectiveness in larger Phase III trials, which will determine its viability alongside or in place of established dopaminergic treatments.
