Aspen Neuroscience reported 12-month data from the ASPIRO clinical trial evaluating sasineprocel (ANPD001), an autologous iPSC-derived dopaminergic neuron precursor cell therapy, in eight patients with Parkinson’s disease. The open-label Phase I/IIa results, presented at the AD/PD 2026 conference in Copenhagen, included numerical changes across motor, functional, and quality-of-life measures in two dose cohorts, according to the company.
Among four patients receiving a low dose (5 million cells per hemisphere) and four receiving a higher dose (5–10 million cells per hemisphere), the company reported mean increases in Good ON time of 2.1 and 2.4 hours, respectively. Mean MDS-UPDRS Part III OFF scores decreased by 15.5 points in the low-dose cohort and 13.5 points in the higher-dose cohort. MDS-UPDRS Part II scores fell by 5.3 and 2.3 points, and PDQ-39 scores improved by 51.6% and 28.5%, respectively. Aspen stated that several patients achieved these changes while also reducing levodopa equivalent daily doses, though specific LEDD figures were not disclosed. FDOPA PET imaging indicated cell survival and engraftment. On safety, the company reported no serious surgical adverse events, no severe graft-induced dyskinesia, and no symptomatic hemorrhages or infarctions. No immunosuppression was administered.
The trial (NCT06344026) is a first-in-human, open-label, multi-center study enrolling Parkinson’s disease patients aged 50–70. The primary endpoint is safety and tolerability of bilateral intracranial delivery into the post-commissural putamen. Because the study is open-label and involves only eight patients with no concurrent control arm in the reported data, the observed numerical changes cannot be attributed to treatment effect; placebo and expectation effects in surgical Parkinson’s trials are well documented. No statistical analyses or confidence intervals were reported. The company said it plans to advance sasineprocel to a Phase III study later in 2026. Sasineprocel holds US FDA Fast Track designation.
Sasineprocel is derived from a patient’s own skin fibroblasts reprogrammed into iPSCs and differentiated into dopaminergic neuron precursors, eliminating the need for immunosuppression required by allogeneic cell therapies such as bemdaneprocel (Bayer/BlueRock Therapeutics), which reported Phase I data in 12 patients in 2023. Cross-trial comparisons are limited by differences in study design, duration, and patient populations.