Abstract
Objective
- To report the safety and clinical outcomes of bemdaneprocel in participants with PD through 3 years post transplantation.
Methods
- Participants with PD, with motor symptoms inadequately controlled by standard treatments, and who were >=50 to <=78 years of age in Canada or >=60 to <=78 years of age in the United States were eligible to enroll in
exPDite. - A total of 12 participants enrolled sequentially.
- Bemdaneprocel was administered stereotactically into the posterior putamen bilaterally through a single burr hole on each side in a single session.
- Immunosuppression was initiated during transplantation and continued for 1 year post transplantation.
- Secondary and exploratory outcomes included intracranial MRI, Unified Dyskinesia Rating Scale (UDysRS), adjusted PD diary ON time without troublesome dyskinesia and OFF time, and MDS-UPDRS Part III ON and OFF at baseline and at 6, 12, 18, and 24 months post transplantation.
Additional Findings
- None of the 8 treatment-emergent serious adverse events were related to transplanted cells or immunosuppression.
- There were no graft-induced dyskinesias and no intracerebral hemorrhages observed; no treatment-emergent adverse events (TEAEs) were related to transplanted cells.
- TEAEs in Nervous system disorders and Infections and infestations System Organ Classes were reported for 9/12 (75.0%) participants each.
- Antiparkinsonian medication use was generally stable for most participants when compared with baseline.
Conclusions
- Through 3 years post transplantation, bemdaneprocel had a favorable safety profile; additionally, there were no deaths, study discontinuations, graft-induced dyskinesias, or intracerebral hemorrhages.
- None of the treatment-emergent serious adverse events were related to transplanted cells or immunosuppression.
- Participants who received bemdaneprocel, particularly those in the high-dose cohort, trended toward improvement or stability across most motor outcomes through 3 years post transplantation, 2 years post discontinuation of immunosuppression.
- These results support the continued development of high-dose bemdaneprocel for the treatment of people with PD.
- Enrollment in
exPDite-2, a Phase 3 randomized, sham-controlled, double-blind trial (NCT06944522) to evaluate the efficacy and safety of bemdaneprocel, is ongoing.
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