Abstract Details

Title Non-motor Effects 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 3-001

Objective Explore the impact of bemdaneprocel on non-motor symptoms in participants with Parkinson’s disease.

Background Bemdaneprocel is an investigational cellular therapy composed of pluripotent stem cell–derived dopaminergic neuron precursor cells under development for the treatment of Parkinson’s disease. At 1 year post transplantation, bemdaneprocel was generally safe and well tolerated in all 12 participants in a phase 1 study.

Design/Methods In this open-label, 24-month, non-controlled study, 12 participants received 1 of 2 different doses of bemdaneprocel bilaterally to the post-commissural putamen and a 1-year immunosuppression regimen. Exploratory outcomes assessed at 12 months included the impact of bemdaneprocel on non-motor symptoms. Assessments will be repeated at 18 months, 6 months post discontinuation of immunosuppression.

Results At 12 months, participants who received the higher dose of bemdaneprocel showed an improvement in non-motor symptoms, including a median (Q1, Q3) change from baseline of −28.7% (−58.5%, −1.2%) on the Non-Motor Symptom Scale total score and a median (Q1, Q3) change from baseline of −83.3% (−100%, −50.0%) on the Neuropsychiatric Inventory Questionnaire. Measures of activities of daily living and quality of life remained stable during this time, with a median (Q1, Q3) change from baseline of −9.1% (−42.9%, 11.1%) in part II of the Movement Disorders Society-Unified Parkinson’s Disease Rating Scale and a median (Q1, Q3) change from baseline of −9.9% (−55.1%, 47.4%) on the 39-item Parkinson’s Disease Questionnaire-39 (PDQ-39) summary index. Participants in the low-dose cohort had a median (Q1, Q3) change from baseline of −21.9% (−46.7%, 12.5%) on the PDQ-39 summary index.

Conclusions Overall, participants who received the higher dose of bemdaneprocel had improvement or no worsening from baseline in non-motor outcomes. Data from additional cognitive assessments and 18-month follow-up (6 months post discontinuation of immunosuppression) will be presented.

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.
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.
Michele K. York, PhD, ABPP-CN (Baylor College of Medicine)	Dr. York has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for RAD-PD. The institution of Dr. York has received research support from Michael J. Fox Foundation. The institution of Dr. York has received research support from NIH. The institution of Dr. York has received research support from Takeda.
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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