Abstract Details

Title Safety, Tolerability, and Clinical Assessment of Bemdaneprocel for Parkinson’s Disease: 18-month Results from a Phase 1 Study

Topic Movement Disorders

Presentation(s) P9 - Poster Session 9 (8:00 AM-9:00 AM)

Poster/Presentation
Number 3-007

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.

Authors/Disclosures
Claire Henchcliffe, MD, PhD, FAAN (University of California, Irvine) PRESENTER	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.
Harini Sarva, MD, FAAN (Weill Cornell Medical Center)	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.
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.
Whitney Stemple (BlueRock Therapeutics)	Whitney Stemple has received personal compensation for serving as an employee of BlueRock Therapeutics.
Nauman Abid, MD (BlueRock Therapeutics)	Dr. Abid has received personal compensation for serving as an employee of BlueRock Therapeutics.
Marcus Yountz, MD, FAAN (BlueRock Therapeutics)	Dr. Yountz has received personal compensation for serving as an employee of Alexion, AstraZeneca Rare Disease. Dr. Yountz has stock in AstraZeneca.
Ahmed Enayetallah, PhD, MBBCh (BlueRock Therapeutics)	Dr. Enayetallah has received personal compensation for serving as an employee of Alexion. Dr. Enayetallah has received stock or an ownership interest from Alexion. Dr. Enayetallah has received stock or an ownership interest from AstraZeneca.
Antoine Lampron, PhD (Bluerock Therapeutics)	Mr. Lampron has received personal compensation for serving as an employee of Bluerock Therapeutics. Mr. Lampron has received personal compensation for serving as an employee of Alexion Inc..
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.

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