Bayer Advances Investigational Stem Cell Therapy Bemdaneprocel into Registrational Phase III Trial for Parkinson’s Disease

On January 14, Bayer and its independently operated clinical-stage cell therapy subsidiary BlueRock Therapeutics recently announced the launch of a Phase III clinical trial for the investigational cell therapy bemdaneprocel targeting Parkinson's disease. The press release noted that this registration clinical trial, named exPDite-2,Expected to launch in the first half of 2025, this will be an important milestone in the development of allogeneic cell therapies for neurodegenerative diseases.。 

exPDite-2 is a randomized, double-blind, controlled trial designed to evaluate the efficacy, safety, and overall impact of bemdaneprocel in Parkinson's patients. The trial aims to recruit approximately 102 participants with moderate Parkinson's disease.The primary endpoint of the study depends on the change in "PD diary" ON time duration from baseline to week 78, assuming patients remain awake for 16 hours per day without experiencing severe dyskinesia.In addition, the trial will also include secondary endpoints aimed at recording objective indicators such as movement, safety, and tolerability, as well as tools for documenting daily life and its quality.

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Pre-registration Opened

Bemdaneprocel is an investigational allogeneic pluripotent stem cell-derived therapy composed of dopaminergic neurons generated from pluripotent stem cells, which canSurgically implanted into the brains of Parkinson's disease patients. After transplantation, these cells have the potential to rebuild neural networks destroyed by Parkinson’s disease, with the hope of restoring both motor and non-motor functions in patients.。

BemdaneprocelCompleted Phase I clinical trial and received the FDA's Regenerative Medicine Advanced Therapy (RMAT) designation in the United States. In May 2024, the therapy received the FDA's Regenerative Medicine Advanced Therapy (RMAT) designation in the United States.

In September 2024, Bayer announcedbemdaneprocelThe Phase I clinical trial exPDite showed 24 months of positive data. Twelve subjects diagnosed with Parkinson's disease underwent surgical transplantation, receiving two different dose levels.bemdaneprocelInjected into the bilateral postcommissural putamen and received one year of immunosuppressive therapy.Cohort A (5 subjects) received a dose of 0.9 million cells per putamen. Cohort B (7 subjects) received a dose of 2.7 million cells per putamen. The primary endpoint was the evaluation of safety and tolerability at 12 months.

Data show:

• Using Hauser PD Diary,Compared to baseline, 7 subjects in the high-dose group were in "Good ON" at 24 months.”The time in status increased by 1.8 hours, at“OFF”The time in status decreased by an average of 1.9 hours.Five subjects in the low-dose group“Good ON”The time in status was reduced by an average of 0.8 hours.“OFF”The time in status increased by 0.4 hours;

• Using MDS-UPDRS Part III in“OFF”The results of measuring motor symptoms under the state show that, compared with the baseline,The high-dose group showed a 21.9-point reduction in motor symptom scores, while the low-dose group exhibited an average decrease of 8.3 points.

• Measured using Part II of the MDS-UPDRS for the impact on activities of daily living, the results showed that compared to baseline,The high-dose group decreased by an average of 3.4 points, while the low-dose group increased by an average of 2.0 points.

In terms of safety, the 24-month safety profile was consistent with early findings, indicating that patients...bemdaneprocelThe tolerance is still good, with no reports ofbemdaneprocelRelated adverse events.