BlueRock Therapeutics progresses bemdaneprocel for Parkinson’s to Phase III trial

Bayer AG and its subsidiary, BlueRock Therapeutics LP, have announced their plans to launch a significant Phase III clinical trial for bemdaneprocel, an investigational cell therapy aimed at treating Parkinson’s disease. This trial, termed exPDite-2, marks a pivotal point in the advancement of cell-based therapies for neurodegenerative diseases, especially Parkinson’s. It is anticipated to commence in the first half of 2025.

Bemdaneprocel is a cell therapy developed to replace dopamine-producing neurons that deteriorate in patients with Parkinson’s disease. These neurons are crucial as their loss leads to the motor function decline associated with the disease. In this novel therapy, dopaminergic neuron precursors derived from pluripotent stem cells are implanted into the patient's brain. The goal is for these precursors to mature into functional dopamine neurons, thereby potentially restoring lost neural connections and improving motor and non-motor functions in affected individuals.

The planned exPDite-2 trial is designed to be a randomized, double-blind study involving sham surgery as a control to rigorously evaluate the efficacy and safety of bemdaneprocel. Approximately 102 individuals with moderate Parkinson’s disease will participate. The trial aims to measure changes in motor function, specifically tracking the duration of "ON-time" without troublesome dyskinesia, a common side effect of Parkinson's treatments. This will be assessed over a period extending to 78 weeks. Secondary objectives include assessing movement metrics, safety, tolerability, and impacts on daily living activities and quality of life.

The initiation of this Phase III trial follows the successful completion of a Phase I study, which involved 12 participants. The earlier trial indicated that bemdaneprocel was well-tolerated, showing no serious adverse events related to the therapy two years post-surgery. Encouraging signs related to improvements in motor function were also observed.

The development of bemdaneprocel has progressed under the guidance of the U.S. Food and Drug Administration (FDA), which granted it the Regenerative Medicine Advanced Therapy (RMAT) designation in 2024. This designation underscores the potential of this therapy to fulfill unmet medical needs in serious conditions such as Parkinson’s disease.

Christian Rommel, a key executive at Bayer Pharmaceuticals, highlighted the importance of this upcoming trial. He emphasized that initiating the Phase III trial is a significant step towards making bemdaneprocel available to patients, thus furthering Bayer’s commitment to cell and gene therapy innovations.

Parkinson’s disease remains a significant global health challenge with no current cure. It is characterized by the progressive degeneration of dopamine-producing neurons, leading to symptoms like tremors, rigidity, and slowed movement. This neurodegenerative disorder also affects non-motor functions, causing cognitive and emotional difficulties. Over 10 million people worldwide are currently living with Parkinson’s, making it the second most prevalent neurodegenerative disease after Alzheimer’s.

BlueRock Therapeutics, founded in 2016 and fully acquired by Bayer in 2019, focuses on developing cellular therapies for various severe diseases. Bemdaneprocel and another investigational therapy, OpCT-001 for photoreceptor conditions, are at the forefront of their clinical endeavors. BlueRock operates under the philosophy of leveraging scientific innovation to address the urgent medical needs of patients, supported by Bayer’s extensive research and development resources.

As Bayer and BlueRock advance bemdaneprocel into this critical phase of clinical testing, the medical community and patients alike are closely monitoring its potential to become a transformative treatment for Parkinson’s disease.