Cell Therapy Bemdaneprocel Continues to Show Positive Effects on Parkinson Disease at 36 Months

Newly presented data from a phase 1/2 trial showed that bemdaneprocel (BlueRock Therapeutics), an investigational cell therapy, maintained a favorable safety profile at 36 months, with stable motor outcomes and continued positive efficacy trends from baseline in patients with Parkinson disease (PD). The agent continues to be studied in an ongoing phase 3 registrational trial, dubbed exPDite-2, that will have data read out in 2027.¹

Presented at the International Congress of Parkinson’s Disease and Movement Disorders (MDS) Congress, held October 5-10, in Hawaii, the phase 1/2 trial included 12 patients with PD who received surgical transplantation of 1 of 2 different doses of bemdaneprocel cells to the post-commissural putamen bilaterally, along with immunosuppression regimen for 1 year. Within the high-dose cohort (n = 7), findings revealed a mean reduction of 17.9 points in MDS-Unified Parkinson Disease Rating Scale (UPDRS)-Part III at 36 months. The lower dose cohort experienced slightly less decreases (13.5 points), although both were considered to be clinically meaningful.

After 36 months of treatment, the cell therapy continued to demonstrate a safe profile, with no adverse events (AEs) related to the treatment or surgical procedure. Using F-Dopa imaging, investigators reported that transplanted cells continued to survive and engraft in the brain after discontinuing immunosuppression therapy at 12 months.

"Bemdaneprocel represents a new approach to restoring the dopamine inputs that are lost in Parkinson’s, and leverages substantial advances in stem cell technology," Claire Henchcliffe, MD, chair of the UC Irvine School of Medicine’s Department of Neurology, University of California, Irvine, said in a statement.¹ "The new 3-year data is a critical next step to evaluate longer term safety. While there is a need for caution in interpreting the positive trends in clinical outcomes, initial signals are there, particularly in the higher dose cohort and the upcoming exPDite-2 clinical trial should shed further light on potential benefits."

Bemdaneprocel, a unique and innovative cell therapy approach, is designed to replace the dopamine producing neurons that are lost in PD. These dopaminergic neuron precursors, derived from human embryonic pluripotent stem cells, mature into dopamine neurons after implantation. To date, bemdaneprocel has gained fast track designation and regenerative medicine advanced therapy designation from the FDA, further supporting the cell therapy’s development.

After 36 months of treatment, those in the high dose cohort showcased mean MDS-UPDRS Part II reductions of –4.3 points relative to baseline. Once again, the lower dose cohort did not perform as well, with mean increases of 0.2 points.

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According to the latest data release, patients in the high-dose group spent roughly 1 additional hour per day in the good ON state–when symptoms were well controlled and free of troublesome dyskinesia–compared with baseline, while their OFF time decreased by about 0.9 hours. Two patients in this group did not complete the PD Diary at 36 months. In the low-dose group, good ON time rose modestly by about 0.2 hours, with a corresponding 1.1-hour reduction in OFF time.

In early January, Bayer AG and BlueRock officially unveiled its phase 3 exPDite-2 trial, a registrational trial that is expected to be a part of a robust data package for future regulatory submissions. The study, the first such phase 3 trial testing an allogenic pluripotent stem cell therapy in PD, is expected to be a randomized, sham-controlled, double-blind trial featuring approximately 102 patients with moderate levels of the disease.²

ExPDite-2 will use change in PD diary measure of ON time without troublesome dyskinesia, adjusted for a 16-hour walking day, over a 78-week period, as the primary end point. In addition, the trial will incporate secondary end points designed to capture objective measures of movement, safety and tolerability, and instruments that capture activities of daily living and quality of life.

At the 2024 MDS Congress, BlueRock presented positive 24-month data from the phase 1/2 trial, with findings showing that bemdaneprocel was safe, with positive efficacy signals. Relative to baseline, those in the high-dose cohort had a mean reduction of 21.9 points in UPDRS-Part III scale while the low dose cohort demonstrated mean increases of 8.3 points. At this time, there was a mean reduction of 3.4 points and 2.0 points in MDS-UPDRS Part II, among the high and low-dose cohorts, respectively.³

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REFERENCES
1. BlueRock Therapeutics reports positive 36-month results from Phase I trial of bemdaneprocel for treating Parkinson’s disease. News release. October 7, 2025. Accessed October 7, 2025. https://www.biospace.com/press-releases/bluerock-therapeutics-reports-positive-36-month-results-from-phase-i-trial-of-bemdaneprocel-for-treating-parkinsons-disease
2. BlueRock Therapeutics advances investigational cell therapy bemdaneprocel for treating Parkinson’s disease to registrational Phase III clinical trial. News release. BlueRock Therapeutics. January 13, 2025. Accessed October 7, 2025. https://www.bluerocktx.com/bluerock-therapeutics-advances-investigational-cell-therapy-bemdaneprocel-for-treating-parkinsons-disease-to-registrational-phase-iii-clinical-trial/
3. BlueRock Therapeutics’ investigational cell therapy bemdaneprocel for Parkinson’s disease shows positive data at 24-months. News release. BlueRock Therapeutics. September 27, 2024. Accessed October 7, 2025. https://www.bayer.com/media/en-us/bluerock-therapeutics-investigational-cell-therapy-bemdaneprocel-for-parkinsons-disease-shows-positive-data-at-24-months/