Aspen Neuroscience Completes ASPIRO Cohort 3 and 4 Dosing, Advancing Sasineprocel Toward Phase 3

Aspen Neuroscience announced the completion of dosing in Cohorts 3 and 4 of its ASPIRO phase 1/2a trial of sasineprocel (ANPD001), bringing the total number of patients dosed in the program to 15 as of June 30, 2026.¹ The milestone represents one of the largest reported clinical experiences for an autologous cell therapy in Parkinson's disease (PD) and advances the program's commercial-ready cryopreserved formulation into broader patient testing ahead of a planned phase 3 transition.

Sasineprocel is an autologous induced pluripotent stem cell (iPSC)-derived dopaminergic neuron precursor cell (DANPC) therapy. It is created from a patient's own skin biopsy, reprogrammed to iPSCs, and differentiated into DANPCs, which are then delivered bilaterally to the posterior putamen via MRI-guided surgery. Because it uses the patient's own cells, no immunosuppression is required, a meaningful differentiator from allogeneic cell therapy approaches currently in development.

Cohorts 3 and 4 mark the first use of Aspen's commercial-ready formulation, a cryopreserved, thaw-and-inject drug product designed for scalable manufacturing and streamlined clinical delivery. Unlike the fresh cell preparation used in Cohorts 1 and 2, the new formulation allows cells to be administered immediately upon arrival at the clinical site, reducing procedural complexity and dependence on on-site cell processing infrastructure. The formulation has been deemed preclinically comparable to earlier cohorts, maintaining continuity of the therapeutic product across the program.

"We are proud to reach this important milestone, which reflects meaningful progress in the sasineprocel program and underscores the growing clinical experience with our personalized, autologous approach," said Damien McDevitt, PhD, President and CEO of Aspen Neuroscience. "Importantly, the use of our commercial formulation in these cohorts represents a critical step toward scalable manufacturing and commercial readiness as we prepare for phase 3 initiation."¹

The cohort 3 and 4 dosing follows a series of positive data readouts from the earlier ASPIRO cohorts. At the AD/PD 2026 International Conference in March in Copenhagen, Aspen presented 12-month data from the first eight treated patients across Cohorts 1 and 2, reporting numerical improvements in motor function, activities of daily living, patient-reported outcomes, and quality of life in both dose groups.² Among four patients receiving the low dose (5 million cells per hemisphere) and four receiving the higher dose (5 to 10 million cells per hemisphere), the company reported mean increases in Good ON time of 2.1 and 2.4 hours, respectively.² Imaging evidence of graft survival and engraftment was also reported. All eight patients tolerated the procedure without serious adverse events, severe graft-induced dyskinesia, or hemorrhage, and no immunosuppression was required in any participant.²

The 12-month findings built on 6-month data reported in 2025, which showed a 45% mean improvement in MDS-UPDRS Part III OFF scores and a 71% improvement in MDS-UPDRS Part II scores in the first three treated patients, alongside reductions in OFF time.³

"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, MD, Professor of Neurology at the University of California San Francisco.²

No disease-modifying therapy is currently approved for Parkinson's disease, and existing treatments address symptoms without slowing the underlying neurodegeneration. Sasineprocel is designed to replace lost dopaminergic neurons and reconstruct the neural circuitry of the putamen, with the goal of providing durable functional benefit rather than symptomatic management alone.

Aspen continues to progress manufacturing scale-out and process optimization in support of pivotal trial requirements. The company has indicated plans to advance sasineprocel into phase 3 testing later this year, following the trajectory set by the phase 1/2a safety and early efficacy data.¹

REFERENCES
1. Aspen Neuroscience announces completion of Cohort 3 and 4 dosing in ASPIRO phase 1/2a trial of sasineprocel for Parkinson's disease. News release. Aspen Neuroscience, Inc. June 30, 2026. Accessed June 30, 2026. https://www.prnewswire.com/news-releases/aspen-neuroscience-announces-completion-of-cohort-3-and-4-dosing-in-aspiro-phase-12a-trial-of-sasineprocel-for-parkinsons-disease-302813731.html
2. 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. News release. Aspen Neuroscience, Inc. March 18, 2026. Accessed March 19, 2026. https://aspenneuroscience.com/aspen-neuroscience-announces-positive-12-month-data-from-its-aspiro-clinical-trial-in-a-late-breaking-oral-presentation-at-t/
3. Aspen Neuroscience initiates Cohort 3 in ASPIRO phase 1/2a trial, highlighting commercial formulation of ANPD001 and its transformative potential for Parkinson's disease. News release. Aspen Neuroscience, Inc. September 25, 2025. Accessed September 25, 2025. https://www.prnewswire.com/news-releases/aspen-neuroscience-initiates-cohort-3-in-aspiro-phase-12a-trial-highlighting-commercial-formulation-of-anpd001-and-its-transformative-potential-for-parkinsons-disease-302566431.html