36-Month Phase I Results Highlight Bemdaneprocel Stem Cell Therapy Promise in Parkinson’s

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Written By: Shreya Bendsure, BPharm

Reviewed By: Pharmacally Editorial Team

Bemdaneprocel (BRT-DA01) is an investigational pluripotent stem cell-derived cell therapy aimed at replacing dopamine-producing neurons lost in Parkinson’s disease (PD). Recent 36-month data from the exPDite Phase I clinical trial, presented at the prestigious International Congress of Parkinson’s Disease and Movement Disorders Society (MDS), held in October 5-10 at Hawaii show a favorable safety profile and encouraging motor function improvements, highlighting its potential as a transformative therapeutic approach for PD. 

Background of Parkinson’s disease

Parkinson’s disease is a progressive neurodegenerative disorder characterized by the loss of dopaminergic neurons in the brain, primarily in the substantia nigra. This leads to dopamine deficiency, causing motor symptoms such as bradykinesia, tremors, rigidity, and postural instability, as well as non-motor symptoms that impact quality of life. Current treatments mainly focus on symptom management rather than halting or reversing neuronal loss.

About Bemdaneprocel (BRT-DA01)

Bemdaneprocel is an investigational cell therapy derived from human embryonic stem cells. It is designed to replace the lost dopaminergic neurons by implanting neuron precursors directly into the patient’s brain via a surgical procedure, aiming to restore dopamine production and improve motor and non-motor functions. The therapy received FDA Fast Track designation in 2021 and Regenerative Medicine Advanced Therapy (RMAT) designation in 2024, reflecting its potential to meet significant unmet medical needs.

Bemdaneprocel is developed and sponsored by BlueRock Therapeutics LP, a wholly owned subsidiary of Bayer AG, a global pharmaceutical company recognized for its commitment to innovative therapies.

exPDite Phase I Clinical Trial Overview

The exPDite Phase I trial (NCT04802733) was a first-in-human study designed to assess the safety, tolerability, and preliminary efficacy of Bemdaneprocel in patients with moderate Parkinson’s disease. Twelve participants were enrolled and received either a low or high dose of the investigational therapy.

This open-label, dose-escalation trial involved a single surgical implantation of the dopaminergic neuron precursors into the putamen region of the brain. The primary endpoint focused on safety and tolerability over a 36-month follow-up period. Secondary and exploratory endpoints assessed motor symptom changes using the Movement Disorder Society Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Parts II and III and Parkinson’s disease Diary measures related to daily motor function.

Results and Motor Outcomes

At 36 months post-implantation, Bemdaneprocel demonstrated a strong safety profile with no adverse events attributed to the therapy or surgery. Imaging studies using F-Dopa confirmed transplanted cell survival and engraftment even after discontinuation of immunosuppressant at 12 months.

Motor outcomes showed clinically meaningful improvements:

  • High-dose group (n=7): Mean reduction of 17.9 points on MDS-UPDRS Part III (“OFF” medication state), and a 4.3-point improvement in Part II related to activities of daily living.
  • Low-dose group (n=4): Mean reduction of 13.5 points on MDS-UPDRS Part III with modest changes in Part II.
  • Parkinson’s disease Diary measures indicated increased “Good ON” time without troublesome dyskinesia and decreased “OFF” time, particularly in the high-dose cohort.

What it means

The 36-month results from the exPDite Phase I trial underscore the potential of Bemdaneprocel as a durable and transformative treatment for Parkinson’s disease. The clinically significant reductions in motor symptom severity, especially in the high-dose cohort, reflect a substantial improvement in patients’ motor function and daily living activities. The increase in “Good ON” time without troublesome dyskinesia and the decrease in “OFF” time indicate that patients experience better symptom control and an improved quality of life.

The sustained survival and engraftment of transplanted dopaminergic neurons beyond the 12-month immunosuppression period demonstrate the therapy’s ability to restore dopamine production long-term, addressing a core physiological deficit in PD. The lack of therapy- or surgery-related adverse events over three years strengthens the confidence in Bemdaneprocel’s safety and tolerability.

These findings suggest that Bemdaneprocel could shift the current PD treatment standard from symptomatic management toward a regenerative approach that replaces lost neurons and potentially slows disease progression. As the therapy progresses to Phase III evaluation, the results provide a hopeful outlook for a significant clinical impact in Parkinson’s disease management.

The pivotal Phase III trial, exPDite-2, recently dosed its first Parkinson’s disease patient, marking a critical advancement in clinical development. This multicenter, double-blind, sham-controlled study will enroll approximately 102 moderate PD patients to rigorously evaluate the efficacy, safety, and overall impact of Bemdaneprocel, potentially supporting future regulatory approval.

Safety Profile

No serious adverse events related to Bemdaneprocel or its surgical implantation was reported throughout the 36-month study. The discontinuation of immunosuppression at 12 months did not adversely affect graft survival or patient safety, reinforcing the therapy’s tolerability.

Key Opinions

Dr. Claire Henchcliffe, Principal Investigator, emphasized the importance of the long-term safety data and encouraging clinical signals, especially for the high-dose group. BlueRock Therapeutics’ senior leadership expressed optimism about Bemdaneprocel’s potential to become a durable, disease-modifying treatment for Parkinson’s patients.

The promising 36-month data have paved the way for the ongoing registrational Phase III trial (exPDite-2), a double-blind, sham surgery-controlled study enrolling about 102 participants. This next phase will aim to confirm efficacy and further evaluate safety to support regulatory approval.

Bemdaneprocel (BRT-DA01) shows promising long-term safety and motor function improvements at 36 months in the exPDite Phase I trial for Parkinson’s disease. This investigational stem cell-derived therapy offers hope for replacing lost dopamine neurons to restore motor abilities and improve quality of life. With no serious adverse events reported and sustained clinical benefits, Bemdaneprocel is advancing to Phase III trials, representing a potential breakthrough in PD treatment.

References

 BlueRock Therapeutics reports positive 36-month results from Phase I trial of bemdaneprocel for treating Parkinson’s disease, 07 October 2025, BlueRock Therapeutics, https://www.bluerocktx.com/bluerock-therapeutics-reports-positive-36-month-results-from-phase-i-trial-of-bemdaneprocel-for-treating-parkinsons-disease/

Cell Therapy Bemdaneprocel Continues to Show Positive Effects on Parkinson Disease at 36 Months, 08 October 2025, Neurology Live, https://www.neurologylive.com/view/cell-therapy-bemdaneprocel-continues-show-positive-effects-parkinson-disease-36-months

36-Month Data on Bemdaneprocel, Ravulizimab Shines in CHAMPION-NMOSD Trial, FDA Fast Tracks Anti-MBTR Tau Antibody, 11October 2025, Neurology Live https://www.neurologylive.com/view/36-month-data-bemdaneprocel-ravulizimab-shines-champion-nmosd-trial-fda-fast-tracks-anti-mbtr-tau-antibody

First Parkinson’s disease patient treated in BlueRock’s pivotal Phase III trial of investigational cell therapy bemdaneprocel, 22 September 2025, Bayer Global, https://www.bayer.com/media/en-us/first-parkinsons-disease-patient-treated-in-bluerocks-pivotal-phase-iii-trial-of-investigational-cell-therapy-bemdaneprocel/

Phase 1 Safety and Tolerability Study of MSK-DA01 Cell Therapy for Advanced Parkinson’s Disease, ClinicalTrials.gov ID NCT04802733, https://clinicaltrials.gov/study/NCT04802733


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