BlueRock Therapeutics, a subsidiary of Bayer AG, has announced that its investigational cell therapy, bemdaneprocel, for the treatment of Parkinson's disease, has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA). This designation aims to expedite the development and review of regenerative medicine therapies intended to treat serious conditions.
Significance of RMAT Designation
The RMAT designation, managed by the FDA's Center for Biologics Evaluation and Research (CBER), is reserved for investigational regenerative medicines, including cell therapies, that have the potential to address unmet medical needs in serious diseases. Bemdaneprocel's RMAT designation provides BlueRock Therapeutics with opportunities for early and frequent interactions with the FDA, including guidance on clinical trial design, surrogate endpoints, and potential pathways for accelerated approval.
Christian Rommel, PhD, Head of Research and Development at Bayer’s Pharmaceuticals Division, stated, “The RMAT designation for bemdaneprocel underscores the potential of this candidate to fundamentally change the way we think about Parkinson’s disease care.”
Bemdaneprocel: A Novel Cell Therapy for Parkinson's Disease
Bemdaneprocel (BRT-DA01) is an investigational cell therapy designed to replace the dopamine-producing neurons that are lost in Parkinson's disease. The therapy involves the surgical implantation of dopaminergic neuron precursors, derived from human embryonic stem cells, into the brain. The goal is to reform neural networks affected by Parkinson's and restore motor and non-motor function.
Phase I Clinical Trial Results
Data from the Phase I clinical trial of bemdaneprocel, announced in March, demonstrated promising results regarding safety and tolerability. The open-label, non-randomized, non-controlled study involved 12 participants with Parkinson's disease who received surgical transplantation of bemdaneprocel cells. The primary endpoint was safety and tolerability at 1 year, with assessments repeated at 18 months.
The trial found that bemdaneprocel was well-tolerated with no major safety issues reported through 18 months. Furthermore, an observed increase in the F-DOPA PET imaging signal after stopping immune suppression therapy at 12 months suggested that the transplanted cells survived and engrafted in the brain.
Seth Ettenberg, President and CEO of BlueRock Therapeutics, commented, “We are excited about the positive data from the bemdaneprocel phase I clinical trial and believe it has great potential to help patients living with Parkinson's disease regain functions they have lost to the disease.”
Parkinson's Disease: An Unmet Medical Need
Parkinson's disease is a progressive neurodegenerative disorder affecting more than 10 million people worldwide, including approximately one million in the United States. The disease is characterized by the loss of dopamine-producing neurons in the brain, leading to motor symptoms such as tremors, rigidity, and slowness of movement. Current treatments primarily focus on managing symptoms, and their effectiveness decreases over time. There is currently no cure for Parkinson's disease, highlighting the urgent need for new therapeutic approaches.
Ongoing Development and Future Directions
With the RMAT designation, BlueRock Therapeutics plans to collaborate closely with the FDA to prepare bemdaneprocel for Phase II clinical studies. The company aims to further evaluate the safety and efficacy of bemdaneprocel in a larger patient population and to assess its potential to modify the course of Parkinson's disease.
