Bayer AG and its subsidiary BlueRock Therapeutics are set to advance bemdaneprocel, an investigational cell therapy for Parkinson's disease, into a Phase 3 clinical trial. The registrational trial, named exPDite-2, is slated to begin in the first half of 2025, marking a significant step in developing allogeneic cell-based therapies for neurodegenerative disorders. This move follows productive discussions with the U.S. Food and Drug Administration (FDA) after bemdaneprocel secured Regenerative Medicine Advanced Therapy (RMAT) designation.
Trial Design and Endpoints
The exPDite-2 trial is designed as a randomized, sham surgery-controlled, double-blind study. It aims to enroll approximately 102 participants with moderate Parkinson's disease. The primary endpoint is the change from baseline to week 78 in the Hauser PD Diary measure of 'on' time without troublesome dyskinesia, adjusted for a 16-hour waking day. Secondary endpoints will capture objective measures of movement, safety, tolerability, activities of daily living, and quality of life.
Bemdaneprocel: A Novel Cell Therapy Approach
Bemdaneprocel (BRT-DA01) is an investigational cell therapy intended to replace the dopamine-producing neurons lost in Parkinson's disease. The therapy involves the surgical transplantation of dopaminergic neuron precursors, derived from pluripotent stem cells, into the putamen region of the brain. Once transplanted, these cells have the potential to re-form neural networks affected by Parkinson's disease and restore motor and non-motor function.
Phase 1 Results and RMAT Designation
The advancement to Phase 3 is supported by positive 24-month data from the Phase 1 exPDite study, which demonstrated the therapy's tolerability and showed that the transplanted cells survived and grew in the brain. The Phase 1 trial, involving 12 participants, reported no serious adverse events related to the drug product at 24 months post-surgery. Furthermore, encouraging trends were observed in secondary endpoints related to motor impairments. In May 2024, the FDA granted bemdaneprocel a RMAT designation, which can accelerate the development of new treatments by offering increased flexibility in clinical trials, early FDA engagement, and priority review.
Parkinson's Disease: An Unmet Need
Parkinson's disease is a progressive neurodegenerative disorder affecting more than 10 million people worldwide. It is characterized by the loss of dopamine-producing nerve cells in the brain, leading to motor symptoms such as tremors, muscle rigidity, and slowed movement, as well as non-motor symptoms like fatigue, cognitive issues, and depression. Current treatments primarily address symptoms, and there is no cure, highlighting the need for new therapies that can holistically manage the disease.
Leadership Perspective
"We are thrilled to take this critical step in our development program towards further investigating a potential new therapeutic option for people living with Parkinson's disease," said Amit Rakhit, MD, MBA, Chief Development and Medical Officer at BlueRock Therapeutics. Christian Rommel, Member of the Executive Committee of Bayer’s Pharmaceuticals Division, and Global Head of Research and Development, added, "With the planned initiation of the Phase III clinical trial, we are committed to bringing bemdaneprocel faster to patients in need."
