Safety, Tolerability, and Clinical Assessment of Bemdaneprocel for Parkinson’s Disease: 18-month Results from a Phase 1 Study
Claire Henchcliffe1, Harini Sarva2, Andres Lozano3, Alfonso Fasano4, Suneil Kalia5, Kenny Kwok Hei Yu6, Cameron Brennan6, Whitney Stemple7, Nauman Abid7, Marcus Yountz7, Ahmed Enayetallah7, Antoine Lampron7, Viviane Tabar6
1University of California, Irvine, 2Weill Cornell Medical Center, 3Toronto Western Hosp, 4Toronto Western Hospital - U of Toronto, 5University of Toronto, 6Memorial Sloan Kettering Cancer Center, 7BlueRock Therapeutics
Objective:
This first-in-human phase 1 study assesses the safety, tolerability, and clinical outcomes of bemdaneprocel in participants with Parkinson’s disease.
Background:
Bemdaneprocel is an investigational cellular therapy comprising pluripotent stem cell–derived dopaminergic neuron precursor cells under development for the treatment of Parkinson’s Disease.
Design/Methods:
In this open-label, 24-month, non-controlled study, 12 participants received 1 of 2 doses of bemdaneprocel bilaterally delivered to the post-commissural putamen and a 1-year immunosuppression regimen. The primary objective was safety and tolerability at 1 year. Exploratory clinical outcomes were also evaluated.
Results:
In the primary analysis at 1 year (previously presented), 2 serious adverse events (SAEs) unrelated to bemdaneprocel were reported, 1 in each cohort (hospitalization due to COVID-19; transient seizure 1 day after surgery). Treatment-emergent adverse events (TEAEs) reported by 11/12 participants were mostly mild or moderate in severity (65/66), and none were considered related to bemdaneprocel. Early trends toward clinical improvement (median [Q1, Q3] change from baseline) at 12 months included: Movement Disorders Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Part III OFF scores and patient-reported Good ON times of 1.0 (−26.0, 13.0) and +0.7 (−0.4, 1.0) hours, respectively, in the low-dose cohort, and −13.0 (−20.0, −4.0) and +2.2 (−0.4, 2.2) hours, respectively, in the high-dose cohort. Median (Q1, Q3) change from baseline in patient-reported OFF time was −1.6 (−1.7, −0.4) and −1.9 (−2.2, 0.4) hours in the low-dose and high-dose cohorts, respectively. We will present 18-month (6 months after discontinuation of immunosuppression) safety, tolerability, and motor data. Conclusions:
Bemdaneprocel was well tolerated, with no major safety issues in all 12 participants through 1 year. Exploratory clinical measures suggested improvements in MDS-UPDRS Part III OFF scores, and patient-reported Good ON time and OFF time. Safety and clinical outcomes with 18 months of follow-up, 6 months post discontinuation of immunosuppression, will be presented.