BlueRock Advances Bemdaneprocel Cell Therapy to Phase III Trial for Parkinson’s Disease

BlueRock Therapeutics, a subsidiary of Bayer AG, is making waves in the treatment of Parkinson’s disease by advancing its investigational cell therapy, bemdaneprocel, to a Phase III clinical trial.

Called exPDite-2, the registrational trial — meaning a trial designed to gather the necessary data for potential regulatory approval — is slated to begin in the first half of 2025.

The clinical advancement follows a thorough review of Phase I trial data under the FDA’s Regenerative Medicine Advanced Therapy (RMAT) designation. The RMAT pathway, tailored for therapies with promising potential, played a key role in accelerating bemdaneprocel’s progression.

Bemdaneprocel is a cell therapy that uses pluripotent stem cells, cells capable of becoming any type of cell in the body. These stem cells are engineered to develop into dopamine-producing neurons, which are progressively lost in Parkinson’s disease.

In a surgical procedure, the neuron precursors are implanted into the brain, where they aim to rebuild the damaged dopamine network, improving motor control and potentially alleviating other symptoms.

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In the Phase I trial, 12 participants received the therapy and were observed for two years. The treatment demonstrated good tolerability, with no serious adverse events linked to it. Notably, secondary endpoints suggested improvements in motor function, paving the way for the larger exPDite-2 trial.

The upcoming trial will enroll approximately 102 participants with moderate Parkinson’s disease. As a double-blind, randomized study with a sham surgery control, it is designed to rigorously evaluate bemdaneprocel’s safety and efficacy. The primary goal will be to measure changes in “on-time” — periods of improved motor function without troublesome dyskinesia — over 78 weeks. Additional metrics will assess movement, quality of life and overall safety.

With the Phase III trial on the horizon, BlueRock Therapeutics and Bayer AG are working to refine their approach to ensure participant engagement and seamless execution.

As of 2023, there were approximately 2.7 million diagnosed cases of Parkinson’s disease in the major markets, with the US accounting for about 45% of these cases.

This year has started strong for Parkinson’s disease research with progress in several clinical trials and medical devices.

BIAL’s Phase II ACTIVATE trial of BIA 28-6156, which targets GBA1 gene mutations, is ongoing, with results expected in 2026 to determine its potential to modify disease progression.

Aspen Neuroscience is advancing its ASPIRO Phase I/IIa trial of ANPD001, a personalized dopamine neuronal therapy derived from patients’ own cells, which has demonstrated early safety and tolerability.

Meanwhile, Medtronic’s BrainSense Adaptive DBS, newly approved in Europe, could help advance neuromodulation therapy for Parkinson’s with real-time brain stimulation adjustments as it becomes available in 2025.

With its Phase III trial on the horizon, bemdaneprocel exemplifies the growing potential of cell and gene therapies for Parkinson’s disease.