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Abstract Details

Continued Evaluation of Safety, Tolerability, and Clinical Outcomes in Participants with Parkinson’s Disease Throughout Three Years After Bemdaneprocel Administration
Movement Disorders
S26 - Movement Disorders: Clinical Trials and Therapeutics (1:24 PM-1:36 PM)
003
To report safety and clinical outcomes in participants with Parkinson’s disease (PD) 3 years after receiving bemdaneprocel.
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; trends toward improvement or stability in clinical assessments were observed through 2 years post transplantation.
In exPDite, 12 participants 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 for assessment through 5 years post transplantation (NCT05897957).
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 3 years post transplantation, 12 participants experienced 112 treatment-emergent adverse events, mostly mild or moderate in severity; none were related to transplanted cells. Eight treatment-emergent serious adverse events were reported, all unrelated to transplanted cells or immunosuppression. 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 3 years post transplantation, mean (SD) changes from baseline in patient-reported ON times without troublesome dyskinesia (+1.0 hours [3.1]) and OFF times (−0.9 hours [3.1]), and MDS-UPDRS Part III ON scores (−5.7 points [7.5]) and OFF scores (−17.9 points [8.5]) showed maintained trends toward improvement. PDQ-39 Summary index scores were similar at baseline through 3 years, with variable changes across domains and cohorts. Overall, outcomes in the low-dose cohort were stable.
These results support ongoing development of bemdaneprocel for the potential treatment of PD.
Authors/Disclosures
Harini Sarva, MD, FAAN (Weill Cornell Medical Center)
PRESENTER 		Dr. Sarva has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novo Nordisk. Dr. Sarva has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Neuroderm. Dr. Sarva has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Insightec. The institution of Dr. Sarva has received research support from Insightec. The institution of Dr. Sarva has received research support from NeuroNext. The institution of Dr. Sarva has received research support from Neuroderm. The institution of Dr. Sarva has received research support from Sun Pharma. The institution of Dr. Sarva has received research support from Prevail. The institution of Dr. Sarva has received research support from Bluerock Therapeutics. The institution of Dr. Sarva has received research support from Biogen. The institution of Dr. Sarva has received research support from Roche. The institution of Dr. Sarva has received research support from National Institute of Aging. The institution of Dr. Sarva has received research support from Michael J Fox Foundation. The institution of Dr. Sarva has received research support from Novo Nordisk. The institution of Dr. Sarva has received research support from Bukwang. The institution of Dr. Sarva has received research support from Bial. The institution of Dr. Sarva has received research support from Cerevance. The institution of Dr. Sarva has received research support from UCB. The institution of Dr. Sarva has received research support from MeiraGTX. The institution of Dr. Sarva has received research support from Biohaven. The institution of Dr. Sarva has received research support from Vima.
Claire Henchcliffe, MD, PhD, FAAN (University of California, Irvine) Dr. Henchcliffe has received personal compensation in the range of $500-$4,999 for serving as a Consultant for AskBio. Dr. Henchcliffe has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Abbvie. Dr. Henchcliffe has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Johnson & Johnson. Dr. Henchcliffe has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Tolmar. Dr. Henchcliffe has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Savanna. Dr. Henchcliffe has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Meira GTx. Dr. Henchcliffe has or had stock in Axent Biosciences Inc. The institution of Dr. Henchcliffe has received research support from Weston Brain Institute. The institution of Dr. Henchcliffe has received research support from Blue Rock Therapeutics. Dr. Henchcliffe has received personal compensation in the range of $500-$4,999 for serving as a Speaker with Parkinson Study Group. Dr. Henchcliffe has received personal compensation in the range of $500-$4,999 for serving as a Speaker/Course director with AAN. Dr. Henchcliffe has received personal compensation in the range of $500-$4,999 for serving as a Speaker with Cleveland Clinic Foundation. Dr. Henchcliffe has received personal compensation in the range of $500-$4,999 for serving as a Speaker with Vertex. Dr. Henchcliffe has received personal compensation in the range of $500-$4,999 for serving as a Speaker with ESGCT.
Andres M. Lozano, MD (Toronto Western Hosp) Dr. Lozano has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Abbott. Dr. Lozano has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Insightec. Dr. Lozano has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Boston Scientific. Dr. Lozano has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Medtronic. Dr. Lozano has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Functional Neuromodulation.
Alfonso Fasano, MD, PhD, FAAN (Toronto Western Hospital - U of Toronto) Dr. Fasano has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Abbott. Dr. Fasano has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Abbvie. Dr. Fasano has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ipsen. Dr. Fasano has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Medtronic. Dr. Fasano has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Boston Scientific. Dr. Fasano has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Abbott. Dr. Fasano has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Medtronic. Dr. Fasano has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Ceregate. Dr. Fasano has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Inbrain Neuroelectronics. Dr. Fasano has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Syneos Health. Dr. Fasano has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Annovis. Dr. Fasano has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for CADTH. Dr. Fasano has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for International Parkinson and Movement Disorders Society. The institution of Dr. Fasano has received research support from Boston Scientific. The institution of Dr. Fasano has received research support from Medtronic. The institution of Dr. Fasano has received research support from Abbvie. The institution of Dr. Fasano has received research support from Canadian Institutes of Health Research (CIHR). The institution of Dr. Fasano has received research support from Michael J Fox Foundation. The institution of Dr. Fasano has received research support from Bluerock Therapeutics. Dr. Fasano has received publishing royalties from a publication relating to health care. Dr. Fasano has a non-compensated relationship as a Medical Advisory Committee with CenteR for Advancing Neurological Innovation to Application (CRANIA) that is relevant to AAN interests or activities. Dr. Fasano has a non-compensated relationship as a Medical Advisory Committee with HopeNET that is relevant to AAN interests or activities. Dr. Fasano has a non-compensated relationship as a Medical Advisory Committee with International Essential Tremor Foundation that is relevant to AAN interests or activities. Dr. Fasano has a non-compensated relationship as a Member with Tremor Research Group that is relevant to AAN interests or activities. Dr. Fasano has a non-compensated relationship as a Co-Chair with Tremor Study Group of the IPMDS that is relevant to AAN interests or activities. Dr. Fasano has a non-compensated relationship as a Vice-Chair with NPH Study Group of the IPMDS that is relevant to AAN interests or activities. Dr. Fasano has a non-compensated relationship as a Vice-Chair with Industry Committee of the IPMDS that is relevant to AAN interests or activities.
Suneil Kalia Suneil Kalia has received personal compensation in the range of $0-$499 for serving as a Consultant for Abbott. Suneil Kalia has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Boston. Suneil Kalia has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Medtronic. Suneil Kalia has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novonordisk. The institution of Suneil Kalia has received research support from CIHR. Suneil Kalia has received research support from MJFF. The institution of Suneil Kalia has received research support from Parkinson Canada.
Kenny Yu (Memorial Sloan Kettering Cancer Center) Kenny Yu has nothing to disclose.
Cameron Brennan Cameron Brennan has nothing to disclose.
Melinda Louie-Gao, PhD Dr. Louie-Gao has received personal compensation for serving as an employee of Bluerock Therapeutics. Dr. Louie-Gao has received personal compensation for serving as an employee of Wave Life Science.
Nicole Floro Ms. Floro has nothing to disclose.
Nauman Abid, MD (BlueRock Therapeutics) Dr. Abid has received personal compensation for serving as an employee of BlueRock Therapeutics.
Viviane Tabar Viviane Tabar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for BlueRock. An immediate family member of Viviane Tabar has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for BlueRock. The institution of Viviane Tabar has received research support from BlueRock. The institution of an immediate family member of Viviane Tabar has received research support from BlueRock. Viviane Tabar has received intellectual property interests from a discovery or technology relating to health care. An immediate family member of Viviane Tabar has received intellectual property interests from a discovery or technology relating to health care.