BlueRock Therapeutics, a subsidiary of Bayer, has announced a significant milestone in the development of a new treatment for Parkinson’s disease. The first patient has been treated in their pivotal Phase III clinical trial, known as exPDite-2.
This trial will evaluate the safety and effectiveness of bemdaneprocel, an investigational cell therapy. The trial is the first of its kind for an allogeneic pluripotent stem cell-derived therapy for Parkinson’s disease.
The exPDite-2 Trial
The exPDite-2 trial is a multicenter, double-blind study that will compare bemdaneprocel to a sham surgery. Approximately 102 people with Parkinson’s disease will be enrolled in the trial.
The primary goal of the study is to measure the change in “ON-time” without troublesome dyskinesia, a common issue for Parkinson’s patients. This will be measured at 78 weeks from the start of the trial.
About Bemdaneprocel
Bemdaneprocel is a cell therapy that replaces dopamine-producing neurons lost in Parkinson’s disease. These neurons are grown from human embryonic pluripotent stem cells.
The therapy involves a surgical procedure to implant these neuron precursors into the brain. The goal is for these new cells to form new neural networks and restore motor and non-motor function.
Previous Trial Results
A Phase I trial of bemdaneprocel with 12 participants showed positive results. The therapy was well-tolerated, with no serious adverse events related to the treatment after 24 months.
Encouraging trends were also seen in the improvement of motor impairments. These results were presented at the 2024 International Congress of Parkinson’s Disease and Movement Disorders.
| Trial Phase | Number of Participants | Key Findings |
|---|---|---|
| Phase I | 12 | Well-tolerated, no serious adverse events, encouraging motor improvement trends |
| Phase III (exPDite-2) | ~102 | Efficacy, safety, and overall impact compared to sham surgery |
The Need for New Treatments
Parkinson’s disease is a progressive neurodegenerative disease that affects more than 10 million people worldwide. It is the second most common neurodegenerative disease.
There is currently no cure for Parkinson’s, and existing treatments do not fully manage the symptoms. This creates an urgent need for new therapies like bemdaneprocel.
Looking Ahead
The results of the exPDite-2 trial will be a key part of the data package for regulatory submissions. If successful, bemdaneprocel could become a new treatment option for people with Parkinson’s disease.
As stated by Amit Rakhit, MD, MBA, BlueRock’s Chief Development and Medical Officer, “The initiation of the exPDite-2 trial represents a major step forward toward advancing bemdaneprocel…as a potentially transformative cell therapy aimed at restoring motor and non-motor function.” [1]
References
[1] BlueRock Therapeutics. (2025, September 22). First Parkinson’s disease patient treated in BlueRock’s pivotal Phase III trial of investigational cell therapy bemdaneprocel. GlobeNewswire. https://www.globenewswire.com/news-release/2025/09/22/3153737/0/en/First-Parkinson-s-disease-patient-treated-in-BlueRock-s-pivotal-Phase-III-trial-of-investigational-cell-therapy-bemdaneprocel.html
