Bayer’s BlueRock advances allogeneic cell therapy into phase 3 Parkinson’s trial

Bayer subsidiary BlueRock Therapeutics announced plans to initiate a phase 3 clinical trial for its investigational allogeneic cell therapy for Parkinson’s, marking the first phase 3 trial for a pluripotent stem cell derived therapy in the disease.

The registrational trial, exPDite-2, is expected to begin in the first half of 2025 and will enroll approximately 102 participants with moderate Parkinson’s disease. The double-blind trial will assess the efficacy, safety and overall impact of bemdaneprocel compared to a sham surgery control. The primary endpoint of the study is change from baseline to week 78 in Parkinson’s diary measure of “ON-time” without troublesome dyskinesia (movement disorders), adjusted for a 16-hour waking day.

In a phase 1 study with 12 participants, bemdaneprocel demonstrated tolerability, with no serious adverse events related to drug product at 24 months post-surgery. Further, encouraging trends were observed in secondary endpoints related to motor impairments at 24 months post-surgery.

Bemdaneprocel is designed to replace the dopamine producing neurons that are lost in Parkinson’s disease with dopaminergic neuron precursors derived from pluripotent stem cells. In a surgical procedure, these neuron precursors are implanted into the brain of a person with Parkinson’s disease. Once transplanted, they have the potential to re-form neural networks that have been severely affected by the disease and to potentially restore motor and non-motor function to patients.

The advancement into a phase 3 registrational trial follows discussions with the U.S. FDA under the Regenerative Medicine Advanced Therapy (RMAT) designation granted by the agency in May 2024.