BlueRock Therapeutics advances to Phase III trial for Parkinson’s disease iPSC therapy
BlueRock Therapeutics LP, a cell therapy company owned by Bayer AG, says it plans on advancing to Phase III clinical trials for bemdaneprocel, its investigational cell therapy for Parkinson’s disease. The registrational trial, named exPDite-2, is expected to begin in the first half of 2025 and may pave the way for allogeneic cell-based therapies for […]
- Phase III exPDite-2 trial follows completion and discussion of Phase I trial data with US FDA under Regenerative Medicine Advanced Therapy (RMAT) designation
- exPDite-2 is a randomized, sham surgery-controlled double-blind trial assessing efficacy and safety of bemdaneprocel in people living with moderate Parkinson’s disease
- Expected to be initiated in H1 2025, exPDite-2 is the first registrational Phase III clinical trial for an investigational allogeneic pluripotent stem cell derived therapy to treat Parkinson’s disease
BlueRock Therapeutics LP, a cell therapy company owned by Bayer AG, says it plans on advancing to Phase III clinical trials for bemdaneprocel, its investigational cell therapy for Parkinson’s disease. The registrational trial, named exPDite-2, is expected to begin in the first half of 2025 and may pave the way for allogeneic cell-based therapies for neurodegenerative disorders.
The company says that in their Phase I 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. Building on these results, exPDite-2 is a double-blind trial that will assess the efficacy, safety and overall impact of bemdaneprocel compared to a sham surgery control. The trial is designed to enroll approximately 102 participants with moderate Parkinson’s disease.
The primary endpoint of the study is change from baseline to week 78 in PD diary measure of ON-time without troublesome dyskinesia, adjusted for a 16-hour waking day. In addition, the trial will incorporate secondary endpoints designed to capture objective measures of movement, safety and tolerability, and instruments that capture activities of daily living and quality of life.
The advancement to this registrational trial follows discussions with the U.S. Food and Drug Administration (FDA) under the Regenerative Medicine Advanced Therapy (RMAT) designation granted by the FDA in May 2024.
Depending upon the outcome, the results from this trial are intended to be part of a robust data package to support regulatory submissions for marketing authorization.
About bemdaneprocel (BRT-DA01)
Bemdaneprocel (BRT-DA01) is an investigational cell therapy designed to replace the dopamine producing neurons that are lost in Parkinson’s disease. These dopaminergic neuron precursors are derived from pluripotent stem cells that are human embryonic stem cells developing into mature dopamine neurons after implantation.
In a surgical procedure, these neuron precursors are implanted into the brain of a person with Parkinson’s disease. When transplanted, they have the potential to re-form neural networks that have been severely affected by Parkinson’s disease and to potentially restore motor and non-motor function to patients. In 2021 bemdaneprocel received Fast Track Designation and in 2024 a Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. The company says that data from the Phase I trial’s 12 participants presented at the 2024 International Congress of Parkinson’s Disease and Movement Disorders (MDS) demonstrated good tolerability, with no serious adverse events related to drug product at 24 months post-surgery. Further, they also report encouraging trends observed in secondary endpoints related to motor impairments at 24 months post-surgery. These participants continue in the long term Continued Evaluation Study. Bemdaneprocel has not been approved for treatment of any disease or medical condition by any health authority.
