BlueRock Therapeutics receives FDA Regenerative Medicine Advanced Therapy designation for Parkinson’s disease cell therapy candidate bemdaneprocel

Bayer AG and BlueRock Therapeutics LP, a clinical stage cell therapy company and wholly owned independently operated subsidiary of Bayer AG, announced today that BlueRock’s investigational cell therapy bemdaneprocel for the treatment of Parkinson’s disease has been granted Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA). “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,” said Seth Ettenberg, President and CEO of BlueRock Therapeutics. “Now with this RMAT designation in hand, we look forward to closely collaborating with the FDA to ready this program for phase II clinical studies.” Bemdaneprocel is the most clinically advanced investigational cell therapy in the U.S. for treating Parkinson’s disease. Phase I clinical trial data announced in March demonstrated that at 18 months it was well tolerated with no major safety issues. In addition, an observed increase in the F-DOPA PET imaging signal after stopping immune suppression therapy at 12 months, as defined in the study’s protocol, demonstrates that the transplanted cells survive and engraft in the brain. Managed by the FDA’s Center for Biologics, Evaluation and Research (CBER), the RMAT program is dedicated to investigational regenerative medicines, including cell therapies, intended to treat, modify, reverse, or cure serious diseases. Investigational therapies that receive RMAT designation are eligible to receive expedited development review and development planning guidance from senior CBER managers. RMAT also creates a pathway for early discussions about potential surrogate endpoints and ways to support accelerated approval and satisfy post approval requirements. “The RMAT designation for bemdaneprocel underscores the potential of this candidate to fundamentally change the way we think about Parkinson’s disease care,” said Christian Rommel, PhD, Head of Research and Development at Bayer’s Pharmaceuticals Division. “We are driven by our commitment to deliver breakthrough innovation for patients and are proud and excited to see bemdaneprocel continuing to clear hurdles in the development process.” About bemdaneprocel (BRT-DA01) and the Phase I Trial 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. 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 reform neural networks that have been severely affected by Parkinson’s and restore motor and non-motor function to patients. Bemdaneprocel has not been approved for treatment of any disease or medical condition by any health authority. The phase I study is a multi-center, multi-site, open-label, non-randomized, non-controlled study. Twelve (12) subjects diagnosed with Parkinson’s disease received surgical transplantation of 1 of 2 different dose levels of bemdaneprocel cells to the post-commissural putamen bilaterally, and administration of a 1-year immunosuppression regimen. Cohort A (5 subjects) received a dose of 0.9 million cells per putamen. Cohort B (7 subjects) received 2.7 million cells per putamen. Safety and tolerability were assessed at 1 year as the primary endpoint, along with evidence of cell survival and motor effects. The feasibility of transplantation was also assessed. Assessments were repeated at 18 months. All assessments will continue over 2 years.