Objective: To report the safety and clinical outcomes of bemdaneprocel in participants with Parkinson’s disease (PD) through 3 years post transplantation.

Background: Bemdaneprocel is an investigational cell therapy composed of human embryonic stem cell-derived midbrain dopaminergic neuron progenitor cells. In exPDite (NCT04802733), a Phase 1 open-label 2-year study, predefined safety, tolerability, and feasibility criteria were met at 1 year, and trends toward improvement or stability in clinical assessments were observed through 2 years post transplantation.

Method: Twelve participants with PD received low-dose (n=5; 0.9 million cells/putamen) or high-dose (n=7; 2.7 million cells/putamen) bemdaneprocel injected bilaterally into the postcommissural putamen in a single surgical session. A 1-year immunosuppression regimen began immediately preoperatively. Following study completion, all participants enrolled in a continued-evaluation study through 5 years post transplantation (NCT05897957).

Results: Participants (N=12) were a median of 67.0 years of age, 75% male, and 67% White. Median time since PD diagnosis was 9.0 years. Through 2 years post transplantation, 12 participants experienced 89 treatment-emergent adverse events, mostly mild or moderate in severity [table 1]. Three treatment-emergent serious adverse events, all unrelated to bemdaneprocel, were reported: COVID-19 (low dose), gastrointestinal hemorrhage (high dose), and seizure (high dose) possibly related to surgery. There were no deaths or graft-induced dyskinesias. No intracerebral hemorrhages or mass lesions were observed by magnetic resonance imaging. In the high-dose cohort at 2 years post transplantation, mean (SD) changes from baseline in patient-reported ON times without troublesome dyskinesia (+1.8 hours [2.6]) and OFF times (−1.9 hours [2.6]) and MDS-UPDRS Part III OFF scores (−21.9 points [7.8]) showed a continuing trend toward improvement [table 2]; non-motor assessments were stable. Outcomes in the low-dose cohort were stable. Results through 3 years post transplantation will be presented.

Conclusion: Bemdaneprocel demonstrated a favorable safety profile and trended toward improvement or stability across clinical assessments through 2 years post transplantation. Results to date support the ongoing development of bemdaneprocel for the treatment of PD.

Table 1

Table 2

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MDS Abstracts - https://www.mdsabstracts.org/abstract/continued-evaluation-of-bemdaneprocel-following-a-phase-1-study-for-parkinsons-disease-outcomes-through-3-years/